A Study of the Drugs Selumetinib Versus Carboplatin/Vincristine in Patients With Neurofibromatosis and Low-Grade Glioma

April 17, 2024 updated by: National Cancer Institute (NCI)

A Phase 3 Randomized Study of Selumetinib Versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG)

This phase III trial studies if selumetinib works just as well as the standard treatment with carboplatin/vincristine (CV) for subjects with NF1-associated low grade glioma (LGG), and to see if selumetinib is better than CV in improving vision in subjects with LGG of the optic pathway (vision nerves). Selumetinib is a drug that works by blocking some enzymes that low-grade glioma tumor cells need for their growth. This results in killing tumor cells. Drugs used as chemotherapy, such as carboplatin and vincristine, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. It is not yet known whether selumetinib works better in treating patients with NF1-associated low-grade glioma compared to standard therapy with carboplatin and vincristine.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

PRIMARY OBJECTIVES:

I. To determine whether the efficacy of treatment with selumetinib sulfate (selumetinib) as measured by event-free survival (EFS) is non-inferior to treatment with carboplatin/vincristine sulfate (vincristine) (CV) in previously untreated neurofibromatosis type 1 (NF1)-associated low-grade glioma (LGG).

II. To determine whether visual acuity (VA) using Teller acuity cards (TAC), in patients with NF1-associated LGG within the optic pathway, is better in those treated with selumetinib compared to CV.

SECONDARY OBJECTIVES:

I. To estimate tumor response rates and overall survival (OS) in each treatment regimen in previously untreated NF1-associated LGG.

II. To evaluate VA outcomes utilizing HOTV letter acuity testing in previously untreated NF1-associated LGG within the optic pathway in patients who are old enough to perform visual acuity testing utilizing HOTV (a recognition acuity measure).

III. To describe the improvement in motor function as measured by the Vineland scale in patients with previously untreated NF1-associated LGG that have documented motor deficits at enrollment.

IV. To prospectively evaluate and compare the quality of life among patients treated with selumetinib or CV.

V. To prospectively evaluate and compare the cognitive, social, emotional, and behavioral functioning of patients with NF1-associated LGG treated with either selumetinib or CV.

EXPLORATORY OBJECTIVES:

I. To evaluate optical coherence tomography (OCT) measures of retinal axon and ganglion cell thickness as a marker of treatment response in previously untreated NF1-associated LGG within the optic pathway.

II. To compare novel, semi-automated volumetric magnetic resonance imaging (MRI) measures to traditional measurements of treatment response (bi-dimensional MRI measurements) in NF1-associated optic pathway tumors.

III. To obtain paired blood and tumor tissue to be banked for future NF1-LGG biology studies involving comprehensive molecular analysis, including but not limited to whole exome and ribonucleic acid (RNA) sequencing.

OUTLINE: Patients are randomized to 1 of 2 arms.

ARM I:

INDUCTION: Patients receive carboplatin intravenously (IV) over 60 minutes on days 1, 8, 15, 22, 43, 50, 57, and 64 and vincristine IV or IV push over 1 minute on days 1, 8, 15, 22, 29, 36, 43, 50, 57, and 64 in the absence of disease progression or unacceptable toxicity. Patients also undergo MRI during screening and on study.

MAINTENANCE: Patients receive carboplatin IV over 60 minutes on days 1, 8, 15, and 22 and vincristine IV or IV push over 1 minute on days 1, 8, and 15. Treatment repeats every 6 weeks for 8 cycles in the absence of disease progression or unacceptable toxicity. Patients also undergo MRI on study and during follow-up.

ARM II: Patients receive selumetinib sulfate orally (PO) twice daily (BID) on days 1-28. Treatment is continuous and repeats every 28 days for 27 cycles in the absence of disease progression or unacceptable toxicity. Patients also undergo MRI throughout the trial.

After completion of study treatment, patients are followed up with MRIs and physical exams every 3 months for 1 year, every 6 months for 2 years, and then once yearly for up to 10 years.

Study Type

Interventional

Enrollment (Estimated)

290

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Nova Scotia
      • Halifax, Nova Scotia, Canada, B3K 6R8
        • Recruiting
        • IWK Health Centre
        • Contact:
        • Principal Investigator:
          • Craig Erker
    • Quebec
      • Montreal, Quebec, Canada, H3H 1P3
        • Recruiting
        • The Montreal Children's Hospital of the MUHC
        • Contact:
        • Principal Investigator:
          • Stephanie Mourad
      • Montreal, Quebec, Canada, H3T 1C5
        • Recruiting
        • Centre Hospitalier Universitaire Sainte-Justine
        • Principal Investigator:
          • Monia Marzouki
        • Contact:
      • Sherbrooke, Quebec, Canada, J1H 5N4
        • Recruiting
        • Centre Hospitalier Universitaire de Sherbrooke-Fleurimont
        • Contact:
        • Principal Investigator:
          • Josee Brossard
  • Puerto Rico
      • Caguas, Puerto Rico, 00726
        • Suspended
        • HIMA San Pablo Oncologic Hospital
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233
        • Suspended
        • Children's Hospital of Alabama
    • Arizona
      • Mesa, Arizona, United States, 85202
        • Recruiting
        • Banner Children's at Desert
        • Contact:
          • Site Public Contact
          • Phone Number: 480-412-3100
        • Principal Investigator:
          • Joseph C. Torkildson
      • Phoenix, Arizona, United States, 85016
        • Recruiting
        • Phoenix Childrens Hospital
        • Contact:
          • Site Public Contact
          • Phone Number: 602-546-0920
        • Principal Investigator:
          • Lindsey M. Hoffman
    • Arkansas
      • Little Rock, Arkansas, United States, 72202-3591
        • Recruiting
        • Arkansas Children's Hospital
        • Contact:
          • Site Public Contact
          • Phone Number: 501-364-7373
        • Principal Investigator:
          • David L. Becton
    • California
      • Loma Linda, California, United States, 92354
        • Recruiting
        • Loma Linda University Medical Center
        • Contact:
          • Site Public Contact
          • Phone Number: 909-558-4050
        • Principal Investigator:
          • Albert Kheradpour
      • Los Angeles, California, United States, 90027
        • Recruiting
        • Children's Hospital Los Angeles
        • Contact:
          • Site Public Contact
          • Phone Number: 323-361-4110
        • Principal Investigator:
          • Nathan J. Robison
      • Oakland, California, United States, 94611
        • Recruiting
        • Kaiser Permanente-Oakland
        • Contact:
          • Site Public Contact
          • Phone Number: 877-642-4691
          • Email: Kpoct@kp.org
        • Principal Investigator:
          • Aarati V. Rao
      • Orange, California, United States, 92868
        • Recruiting
        • Children's Hospital of Orange County
        • Contact:
        • Principal Investigator:
          • Elyssa M. Rubin
      • Palo Alto, California, United States, 94304
        • Recruiting
        • Lucile Packard Children's Hospital Stanford University
        • Contact:
        • Principal Investigator:
          • Jay Michael S. Balagtas
      • San Diego, California, United States, 92123
        • Recruiting
        • Rady Children's Hospital - San Diego
        • Contact:
          • Site Public Contact
          • Phone Number: 858-966-5934
        • Principal Investigator:
          • William D. Roberts
      • San Diego, California, United States, 92134
        • Recruiting
        • Naval Medical Center -San Diego
        • Contact:
          • Site Public Contact
          • Phone Number: 619-532-8712
        • Principal Investigator:
          • Yoko T. Udaka
      • San Francisco, California, United States, 94158
        • Suspended
        • UCSF Medical Center-Mission Bay
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Recruiting
        • Children's Hospital Colorado
        • Contact:
        • Principal Investigator:
          • Nicholas K. Foreman
    • Connecticut
      • Hartford, Connecticut, United States, 06106
        • Recruiting
        • Connecticut Children's Medical Center
        • Contact:
          • Site Public Contact
          • Phone Number: 860-545-9981
        • Principal Investigator:
          • Michael S. Isakoff
      • New Haven, Connecticut, United States, 06520
        • Recruiting
        • Yale University
        • Principal Investigator:
          • Asher M. Marks
        • Contact:
    • Delaware
      • Wilmington, Delaware, United States, 19803
        • Recruiting
        • Alfred I duPont Hospital for Children
        • Contact:
        • Principal Investigator:
          • Scott M. Bradfield
    • District of Columbia
      • Washington, District of Columbia, United States, 20010
        • Recruiting
        • Children's National Medical Center
        • Principal Investigator:
          • Jeffrey S. Dome
        • Contact:
    • Florida
      • Gainesville, Florida, United States, 32610
        • Recruiting
        • University of Florida Health Science Center - Gainesville
        • Contact:
        • Principal Investigator:
          • William B. Slayton
      • Hollywood, Florida, United States, 33021
        • Recruiting
        • Memorial Regional Hospital/Joe DiMaggio Children's Hospital
        • Contact:
          • Site Public Contact
          • Phone Number: 954-265-1847
          • Email: OHR@mhs.net
        • Principal Investigator:
          • Iftikhar Hanif
      • Jacksonville, Florida, United States, 32207
        • Recruiting
        • Nemours Children's Clinic-Jacksonville
        • Contact:
        • Principal Investigator:
          • Scott M. Bradfield
      • Miami, Florida, United States, 33155
        • Recruiting
        • Nicklaus Children's Hospital
        • Contact:
          • Site Public Contact
          • Phone Number: 888-624-2778
        • Principal Investigator:
          • Ziad A. Khatib
      • Orlando, Florida, United States, 32806
        • Recruiting
        • Arnold Palmer Hospital for Children
        • Contact:
        • Principal Investigator:
          • Amy A. Smith
      • Orlando, Florida, United States, 32827
        • Recruiting
        • Nemours Children's Hospital
        • Contact:
        • Principal Investigator:
          • Scott M. Bradfield
      • Orlando, Florida, United States, 32803
        • Recruiting
        • AdventHealth Orlando
        • Contact:
        • Principal Investigator:
          • Fouad M. Hajjar
      • Pensacola, Florida, United States, 32504
        • Recruiting
        • Sacred Heart Hospital
        • Contact:
        • Principal Investigator:
          • Jeffrey H. Schwartz
      • Saint Petersburg, Florida, United States, 33701
        • Recruiting
        • Johns Hopkins All Children's Hospital
        • Contact:
        • Principal Investigator:
          • Stacie L. Stapleton
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Recruiting
        • Children's Healthcare of Atlanta - Egleston
        • Contact:
        • Principal Investigator:
          • Jason R. Fangusaro
    • Hawaii
      • Honolulu, Hawaii, United States, 96826
        • Recruiting
        • Kapiolani Medical Center for Women and Children
        • Contact:
          • Site Public Contact
          • Phone Number: 808-983-6090
        • Principal Investigator:
          • Wade T. Kyono
    • Idaho
      • Boise, Idaho, United States, 83712
        • Recruiting
        • Saint Luke's Cancer Institute - Boise
        • Principal Investigator:
          • Martha M. Pacheco
        • Contact:
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Recruiting
        • Lurie Children's Hospital-Chicago
        • Contact:
          • Site Public Contact
          • Phone Number: 773-880-4562
        • Principal Investigator:
          • Angela J. Waanders
      • Chicago, Illinois, United States, 60637
        • Recruiting
        • University of Chicago Comprehensive Cancer Center
        • Contact:
        • Principal Investigator:
          • Wendy S. Darlington
      • Chicago, Illinois, United States, 60612
        • Recruiting
        • University of Illinois
        • Contact:
          • Site Public Contact
          • Phone Number: 312-355-3046
        • Principal Investigator:
          • Dipti S. Dighe
      • Springfield, Illinois, United States, 62702
        • Recruiting
        • Southern Illinois University School of Medicine
        • Contact:
          • Site Public Contact
          • Phone Number: 217-545-7929
        • Principal Investigator:
          • Gregory P. Brandt
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Recruiting
        • Riley Hospital for Children
        • Contact:
          • Site Public Contact
          • Phone Number: 800-248-1199
        • Principal Investigator:
          • Sandeep Batra
      • Indianapolis, Indiana, United States, 46260
        • Recruiting
        • Ascension Saint Vincent Indianapolis Hospital
        • Contact:
        • Principal Investigator:
          • Jessica F. Goodman
    • Iowa
      • Des Moines, Iowa, United States, 50309
        • Recruiting
        • Blank Children's Hospital
        • Contact:
        • Principal Investigator:
          • Samantha L. Mallory
      • Iowa City, Iowa, United States, 52242
        • Recruiting
        • University of Iowa/Holden Comprehensive Cancer Center
        • Contact:
          • Site Public Contact
          • Phone Number: 800-237-1225
        • Principal Investigator:
          • David S. Dickens
    • Kentucky
      • Lexington, Kentucky, United States, 40536
        • Recruiting
        • University of Kentucky/Markey Cancer Center
        • Contact:
          • Site Public Contact
          • Phone Number: 859-257-3379
        • Principal Investigator:
          • James T. Badgett
      • Louisville, Kentucky, United States, 40202
        • Recruiting
        • Norton Children's Hospital
        • Contact:
        • Principal Investigator:
          • Ashok B. Raj
    • Louisiana
      • New Orleans, Louisiana, United States, 70118
        • Recruiting
        • Children's Hospital New Orleans
        • Contact:
        • Principal Investigator:
          • Lolie C. Yu
      • New Orleans, Louisiana, United States, 70121
        • Recruiting
        • Ochsner Medical Center Jefferson
        • Contact:
        • Principal Investigator:
          • Craig Lotterman
    • Maine
      • Bangor, Maine, United States, 04401
        • Recruiting
        • Eastern Maine Medical Center
        • Contact:
          • Site Public Contact
          • Phone Number: 207-973-4274
        • Principal Investigator:
          • Daniel L. Callaway
      • Scarborough, Maine, United States, 04074
        • Recruiting
        • Maine Children's Cancer Program
        • Contact:
        • Principal Investigator:
          • Stanley Chaleff
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Recruiting
        • Johns Hopkins University/Sidney Kimmel Cancer Center
        • Contact:
        • Principal Investigator:
          • Kenneth J. Cohen
      • Bethesda, Maryland, United States, 20889-5600
        • Active, not recruiting
        • Walter Reed National Military Medical Center
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Recruiting
        • Massachusetts General Hospital Cancer Center
        • Contact:
          • Site Public Contact
          • Phone Number: 877-726-5130
        • Principal Investigator:
          • David H. Ebb
      • Boston, Massachusetts, United States, 02215
        • Recruiting
        • Dana-Farber Cancer Institute
        • Contact:
          • Site Public Contact
          • Phone Number: 877-442-3324
        • Principal Investigator:
          • Susan N. Chi
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • Recruiting
        • C S Mott Children's Hospital
        • Contact:
          • Site Public Contact
          • Phone Number: 800-865-1125
        • Principal Investigator:
          • Andrea T. Franson
      • Detroit, Michigan, United States, 48201
        • Suspended
        • Wayne State University/Karmanos Cancer Institute
      • Detroit, Michigan, United States, 48201
      • Grand Rapids, Michigan, United States, 49503
        • Recruiting
        • Helen DeVos Children's Hospital at Spectrum Health
        • Principal Investigator:
          • Kathleen J. Yost
        • Contact:
      • Royal Oak, Michigan, United States, 48073
        • Recruiting
        • Beaumont Children's Hospital-Royal Oak
        • Contact:
          • Site Public Contact
          • Phone Number: 248-551-7695
        • Principal Investigator:
          • Laura K. Gowans
    • Minnesota
      • Minneapolis, Minnesota, United States, 55404
        • Recruiting
        • Children's Hospitals and Clinics of Minnesota - Minneapolis
        • Principal Investigator:
          • Michael K. Richards
        • Contact:
      • Minneapolis, Minnesota, United States, 55455
        • Recruiting
        • University of Minnesota/Masonic Cancer Center
        • Contact:
          • Site Public Contact
          • Phone Number: 612-624-2620
        • Principal Investigator:
          • Christopher L. Moertel
      • Rochester, Minnesota, United States, 55905
        • Recruiting
        • Mayo Clinic in Rochester
        • Contact:
          • Site Public Contact
          • Phone Number: 855-776-0015
        • Principal Investigator:
          • Jonathan D. Schwartz
    • Mississippi
      • Jackson, Mississippi, United States, 39216
        • Recruiting
        • University of Mississippi Medical Center
        • Principal Investigator:
          • Betty L. Herrington
        • Contact:
          • Site Public Contact
          • Phone Number: 601-815-6700
    • Missouri
      • Columbia, Missouri, United States, 65201
      • Kansas City, Missouri, United States, 64108
        • Recruiting
        • Children's Mercy Hospitals and Clinics
        • Contact:
          • Site Public Contact
          • Phone Number: 816-302-6808
          • Email: rryan@cmh.edu
        • Principal Investigator:
          • Keith J. August
      • Saint Louis, Missouri, United States, 63110
        • Recruiting
        • Washington University School of Medicine
        • Contact:
        • Principal Investigator:
          • Andrew S. Cluster
      • Saint Louis, Missouri, United States, 63104
        • Recruiting
        • Cardinal Glennon Children's Medical Center
        • Contact:
          • Site Public Contact
          • Phone Number: 314-268-4000
        • Principal Investigator:
          • William S. Ferguson
    • Nebraska
      • Omaha, Nebraska, United States, 68114
        • Recruiting
        • Children's Hospital and Medical Center of Omaha
        • Contact:
          • Site Public Contact
          • Phone Number: 402-955-3949
        • Principal Investigator:
          • Jill C. Beck
      • Omaha, Nebraska, United States, 68198
        • Recruiting
        • University of Nebraska Medical Center
        • Contact:
        • Principal Investigator:
          • Jill C. Beck
    • New Hampshire
      • Lebanon, New Hampshire, United States, 03756
        • Recruiting
        • Dartmouth Hitchcock Medical Center/Dartmouth Cancer Center
        • Principal Investigator:
          • Angela Ricci
        • Contact:
    • New Jersey
      • Morristown, New Jersey, United States, 07960
        • Recruiting
        • Morristown Medical Center
        • Contact:
          • Site Public Contact
          • Phone Number: 973-971-5900
        • Principal Investigator:
          • Kathryn L. Laurie
      • New Brunswick, New Jersey, United States, 08903
        • Recruiting
        • Rutgers Cancer Institute of New Jersey-Robert Wood Johnson University Hospital
        • Principal Investigator:
          • Scott Moerdler
        • Contact:
          • Site Public Contact
          • Phone Number: 732-235-8675
    • New Mexico
      • Albuquerque, New Mexico, United States, 87102
        • Recruiting
        • University of New Mexico Cancer Center
        • Contact:
        • Principal Investigator:
          • Jessica M. Valdez
      • Albuquerque, New Mexico, United States, 87106
        • Recruiting
        • Presbyterian Hospital
        • Contact:
        • Principal Investigator:
          • Xiaxin Li
    • New York
      • Albany, New York, United States, 12208
        • Recruiting
        • Albany Medical Center
        • Contact:
          • Site Public Contact
          • Phone Number: 518-262-5513
        • Principal Investigator:
          • Lauren R. Weintraub
      • Buffalo, New York, United States, 14263
        • Recruiting
        • Roswell Park Cancer Institute
        • Contact:
        • Principal Investigator:
          • Matthew J. Barth
      • New Hyde Park, New York, United States, 11040
        • Recruiting
        • The Steven and Alexandra Cohen Children's Medical Center of New York
        • Contact:
          • Site Public Contact
          • Phone Number: 718-470-3460
        • Principal Investigator:
          • Mark P. Atlas
      • New York, New York, United States, 10016
        • Recruiting
        • Laura and Isaac Perlmutter Cancer Center at NYU Langone
        • Contact:
        • Principal Investigator:
          • Sharon L. Gardner
      • New York, New York, United States, 10065
        • Active, not recruiting
        • Memorial Sloan Kettering Cancer Center
      • Rochester, New York, United States, 14642
        • Recruiting
        • University of Rochester
        • Principal Investigator:
          • David N. Korones
        • Contact:
          • Site Public Contact
          • Phone Number: 585-275-5830
      • Syracuse, New York, United States, 13210
        • Recruiting
        • State University of New York Upstate Medical University
        • Contact:
          • Site Public Contact
          • Phone Number: 315-464-5476
        • Principal Investigator:
          • Philip M. Monteleone
      • Valhalla, New York, United States, 10595
        • Recruiting
        • New York Medical College
        • Contact:
          • Site Public Contact
          • Phone Number: 914-594-3794
        • Principal Investigator:
          • Jessica C. Hochberg
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27599
        • Recruiting
        • UNC Lineberger Comprehensive Cancer Center
        • Contact:
        • Principal Investigator:
          • David E. Kram
      • Charlotte, North Carolina, United States, 28203
        • Recruiting
        • Carolinas Medical Center/Levine Cancer Institute
        • Contact:
          • Site Public Contact
          • Phone Number: 800-804-9376
        • Principal Investigator:
          • Joel A. Kaplan
      • Durham, North Carolina, United States, 27710
        • Recruiting
        • Duke University Medical Center
        • Principal Investigator:
          • Jessica M. Sun
        • Contact:
          • Site Public Contact
          • Phone Number: 888-275-3853
      • Greenville, North Carolina, United States, 27834
        • Recruiting
        • East Carolina University
        • Contact:
        • Principal Investigator:
          • Andrea R. Whitfield
      • Winston-Salem, North Carolina, United States, 27157
        • Recruiting
        • Wake Forest University Health Sciences
        • Contact:
          • Site Public Contact
          • Phone Number: 336-713-6771
        • Principal Investigator:
          • Thomas W. McLean
    • North Dakota
      • Fargo, North Dakota, United States, 58122
    • Ohio
      • Akron, Ohio, United States, 44308
        • Recruiting
        • Children's Hospital Medical Center of Akron
        • Contact:
          • Site Public Contact
          • Phone Number: 330-543-3193
        • Principal Investigator:
          • Erin Wright
      • Cincinnati, Ohio, United States, 45229
        • Recruiting
        • Cincinnati Children's Hospital Medical Center
        • Contact:
        • Principal Investigator:
          • Peter M. de Blank
      • Cleveland, Ohio, United States, 44106
        • Recruiting
        • Rainbow Babies and Childrens Hospital
        • Contact:
          • Site Public Contact
          • Phone Number: 216-844-5437
        • Principal Investigator:
          • Duncan S. Stearns
      • Columbus, Ohio, United States, 43205
      • Dayton, Ohio, United States, 45404
        • Recruiting
        • Dayton Children's Hospital
        • Contact:
          • Site Public Contact
          • Phone Number: 800-228-4055
        • Principal Investigator:
          • Mukund G. Dole
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73104
        • Recruiting
        • University of Oklahoma Health Sciences Center
        • Contact:
        • Principal Investigator:
          • Rene Y. McNall-Knapp
    • Oregon
      • Portland, Oregon, United States, 97227
        • Recruiting
        • Legacy Emanuel Children's Hospital
        • Contact:
          • Site Public Contact
          • Phone Number: 503-413-2560
        • Principal Investigator:
          • Janice F. Olson
      • Portland, Oregon, United States, 97239
        • Recruiting
        • Oregon Health and Science University
        • Contact:
        • Principal Investigator:
          • Linda C. Stork
    • Pennsylvania
      • Danville, Pennsylvania, United States, 17822
        • Recruiting
        • Geisinger Medical Center
        • Contact:
        • Principal Investigator:
          • Jagadeesh Ramdas
      • Hershey, Pennsylvania, United States, 17033
        • Recruiting
        • Penn State Children's Hospital
        • Contact:
          • Site Public Contact
          • Phone Number: 717-531-6012
        • Principal Investigator:
          • Lisa M. McGregor
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • Children's Hospital of Philadelphia
        • Principal Investigator:
          • Michael J. Fisher
        • Contact:
      • Philadelphia, Pennsylvania, United States, 19134
        • Recruiting
        • Saint Christopher's Hospital for Children
        • Contact:
          • Site Public Contact
          • Phone Number: 215-427-8991
        • Principal Investigator:
          • Gregory E. Halligan
      • Pittsburgh, Pennsylvania, United States, 15224
        • Recruiting
        • Children's Hospital of Pittsburgh of UPMC
        • Contact:
        • Principal Investigator:
          • James T. Felker
    • Rhode Island
      • Providence, Rhode Island, United States, 02903
        • Recruiting
        • Rhode Island Hospital
        • Contact:
          • Site Public Contact
          • Phone Number: 401-444-1488
        • Principal Investigator:
          • Jennifer J. Welch
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • Recruiting
        • Medical University of South Carolina
        • Contact:
        • Principal Investigator:
          • Jacqueline M. Kraveka
      • Columbia, South Carolina, United States, 29203
        • Recruiting
        • Prisma Health Richland Hospital
        • Contact:
          • Site Public Contact
          • Phone Number: 864-241-6251
        • Principal Investigator:
          • Stuart L. Cramer
      • Greenville, South Carolina, United States, 29605
        • Recruiting
        • BI-LO Charities Children's Cancer Center
        • Contact:
          • Site Public Contact
          • Phone Number: 864-241-6251
        • Principal Investigator:
          • Aniket Saha
    • Tennessee
      • Knoxville, Tennessee, United States, 37916
        • Recruiting
        • East Tennessee Childrens Hospital
        • Contact:
          • Site Public Contact
          • Phone Number: 865-541-8266
        • Principal Investigator:
          • Susan E. Spiller
      • Memphis, Tennessee, United States, 38105
        • Recruiting
        • Saint Jude Children's Research Hospital
        • Contact:
        • Principal Investigator:
          • Ibrahim A. Qaddoumi
      • Nashville, Tennessee, United States, 37232
        • Recruiting
        • Vanderbilt University/Ingram Cancer Center
        • Contact:
          • Site Public Contact
          • Phone Number: 800-811-8480
        • Principal Investigator:
          • Devang J. Pastakia
    • Texas
      • Austin, Texas, United States, 78723
        • Recruiting
        • Dell Children's Medical Center of Central Texas
        • Contact:
        • Principal Investigator:
          • Shannon M. Cohn
      • Dallas, Texas, United States, 75390
        • Recruiting
        • UT Southwestern/Simmons Cancer Center-Dallas
        • Contact:
        • Principal Investigator:
          • Laura J. Klesse
      • El Paso, Texas, United States, 79905
        • Recruiting
        • El Paso Children's Hospital
        • Contact:
        • Principal Investigator:
          • Benjamin Carcamo
      • Fort Worth, Texas, United States, 76104
      • Houston, Texas, United States, 77030
        • Recruiting
        • M D Anderson Cancer Center
        • Contact:
        • Principal Investigator:
          • Najat C. Daw
      • Houston, Texas, United States, 77030
        • Recruiting
        • Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center
        • Contact:
          • Site Public Contact
          • Phone Number: 713-798-1354
          • Email: burton@bcm.edu
        • Principal Investigator:
          • Surya P. Rednam
      • Lubbock, Texas, United States, 79410
        • Recruiting
        • Covenant Children's Hospital
        • Principal Investigator:
          • Kishor M. Bhende
        • Contact:
      • Lubbock, Texas, United States, 79415
        • Recruiting
        • UMC Cancer Center / UMC Health System
        • Contact:
          • Site Public Contact
          • Phone Number: 806-775-8590
        • Principal Investigator:
          • Erin K. Barr
      • San Antonio, Texas, United States, 78207
        • Recruiting
        • Children's Hospital of San Antonio
        • Contact:
        • Principal Investigator:
          • Timothy C. Griffin
      • San Antonio, Texas, United States, 78229
        • Recruiting
        • University of Texas Health Science Center at San Antonio
        • Contact:
        • Principal Investigator:
          • Shafqat Shah
      • San Antonio, Texas, United States, 78229
        • Recruiting
        • Methodist Children's Hospital of South Texas
        • Contact:
        • Principal Investigator:
          • Jose M. Esquilin
    • Utah
      • Salt Lake City, Utah, United States, 84113
        • Recruiting
        • Primary Children's Hospital
        • Contact:
          • Site Public Contact
          • Phone Number: 801-585-5270
        • Principal Investigator:
          • Priya Chan
    • Vermont
      • Burlington, Vermont, United States, 05405
        • Recruiting
        • University of Vermont and State Agricultural College
        • Contact:
          • Site Public Contact
          • Phone Number: 802-656-8990
          • Email: rpo@uvm.edu
        • Principal Investigator:
          • Jessica L. Heath
    • Virginia
      • Norfolk, Virginia, United States, 23507
        • Recruiting
        • Children's Hospital of The King's Daughters
        • Contact:
        • Principal Investigator:
          • Eric J. Lowe
      • Richmond, Virginia, United States, 23298
        • Recruiting
        • Virginia Commonwealth University/Massey Cancer Center
        • Contact:
        • Principal Investigator:
          • Gita V. Massey
    • Washington
      • Seattle, Washington, United States, 98105
        • Recruiting
        • Seattle Children's Hospital
        • Contact:
          • Site Public Contact
          • Phone Number: 866-987-2000
        • Principal Investigator:
          • Sarah E. Leary
      • Spokane, Washington, United States, 99204
        • Recruiting
        • Providence Sacred Heart Medical Center and Children's Hospital
        • Contact:
        • Principal Investigator:
          • Judy L. Felgenhauer
      • Tacoma, Washington, United States, 98405
        • Recruiting
        • Mary Bridge Children's Hospital and Health Center
        • Contact:
        • Principal Investigator:
          • Robert G. Irwin
      • Tacoma, Washington, United States, 98431
        • Recruiting
        • Madigan Army Medical Center
        • Principal Investigator:
          • Melissa A. Forouhar
        • Contact:
    • Wisconsin
      • Madison, Wisconsin, United States, 53792
        • Recruiting
        • University of Wisconsin Carbone Cancer Center
        • Principal Investigator:
          • Kenneth B. De Santes
        • Contact:
      • Milwaukee, Wisconsin, United States, 53226
        • Recruiting
        • Children's Hospital of Wisconsin
        • Contact:
        • Principal Investigator:
          • Sarah Rumler

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 21 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Patients must be >= 2 years and =< 21 years at the time of enrollment
  • Patients must have a body surface area (BSA) of >= 0.5 m^2 at enrollment
  • Patients must have neurofibromatosis type 1 (NF1) based on clinical criteria and/or germline genetic testing
  • Patients must be newly diagnosed or have previously diagnosed NF-1 associated LGG that has not been treated with any modality other than surgery

  • For patients with optic pathway gliomas (OPGs):

    • Newly-diagnosed patients with OPG are eligible if there are neurologic symptoms (including visual dysfunction, as defined below) or other exam findings associated with the tumor
    • Previously-diagnosed patients with OPG are eligible if they have new or worsening neurologic symptoms (including visual dysfunction, as defined below) or have tumor growth

    • For both newly-diagnosed and previously-diagnosed OPG, the patient may be eligible, irrespective of whether there has been tumor growth or other neurological symptoms or worsening, if they meet at least one of the following visual criteria:

      • Visual worsening, defined as worsening of visual acuity (VA) or visual fields (VF) documented within the past year (by examination or history); OR
      • Significant visual dysfunction (defined as VA worse than normal for age by 0.6 logMAR [20/80, 6/24, or 2.5/10] or more in one or both eyes)

  • For patients with LGG in other locations (i.e., not OPGs):

    • Newly-diagnosed patients with LGG are eligible if there are neurologic symptoms or other exam findings associated with the tumor

      • NOTE: Newly-diagnosed patients with LGG without associated neurologic symptoms or exam findings are not eligible
    • Previously-diagnosed patients with LGG are eligible if they have new or worsening neurologic symptoms or have tumor growth
  • Although not required, if a biopsy/tumor resection is performed, eligible histologies will include all tumors considered LGG or low-grade astrocytoma (World Health Organization [WHO] grade I and II) by 5th edition WHO classification of central nervous system (CNS) tumors with the exception of subependymal giant cell astrocytoma
  • Patients must have two-dimensional measurable tumor >= 1 cm^2
  • Patients with metastatic disease or multiple independent primary LGGs are allowed on study

  • Creatinine clearance or radioisotope glomerular filtration Rate (GFR) >= 70 mL/min/1.73 m^2 OR a serum creatinine based on age/gender (within 7 days prior to enrollment) as follows:

    • Age; maximum serum creatinine (mg/dL)
    • 2 to < 6 years; 0.8 (male) and 0.8 (female)
    • 6 to < 10 years; 1 (male) and 1 (female)
    • 10 to < 13 years; 1.2 (male) and 1.2 (female)
    • 13 to < 16 years; 1.5 (male) and 1.4 (female)
    • >= 16 years; 1.7 (male) and 1.4 (female)
  • Total bilirubin =< 1.5 x upper limit of normal (ULN) for age (within 7 days prior to enrollment) (children with a diagnosis of Gilbert's syndrome will be allowed on study regardless of their total and indirect [unconjugated] bilirubin levels as long as their direct [conjugated] bilirubin is < 3.1 mg/dL)
  • Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 3 x upper limit of normal (ULN) = 135 U/L (within 7 days prior to enrollment). For the purpose of this study, the ULN for SGPT is 45 U/L
  • Albumin >= 2 g/dL (within 7 days prior to enrollment)
  • Left ventricular ejection fraction (LVEF) >= 53% (or institutional normal; if the LVEF result is given as a range of values, then the upper value of the range will be used) by echocardiogram (within 4 weeks prior to enrollment)
  • Corrected QT (QTc) interval =< 450 msec by electrocardiography (EKG) (within 4 weeks prior to enrollment)
  • Absolute neutrophil count >= 1,000/uL (unsupported) (within 7 days prior to enrollment)
  • Platelets >= 100,000/uL (unsupported) (within 7 days prior to enrollment)
  • Hemoglobin >= 8 g/dL (may be supported) (within 7 days prior to enrollment)
  • Patients with a known seizure disorder should be stable and should have not experienced a significant increase in seizure frequency within 2 weeks prior to enrollment

  • Patients 2-17 years of age must have a blood pressure that is =< 95th percentile for age, height, and gender at the time of enrollment. Patients >= 18 years of age must have a blood pressure =< 130/80 mmHg at the time of enrollment (with or without the use of antihypertensive medications).

    • Note: Adequate blood pressure can be achieved using medication for the treatment of hypertension
  • All patients must have ophthalmology toxicity assessments performed within 4 weeks prior to enrollment
  • For all patients, an MRI of the brain (with orbital cuts for optic pathway tumors) and/or spine (depending on the site(s) of primary disease) with and without contrast must be performed within 4 weeks prior to enrollment

  • For patients who undergo a surgery on the target tumor (not required), a pre- and post-operative* MRI of the brain (with orbital cuts for optic pathway tumors) or spine (depending on the site(s) of primary disease) with and without contrast must also be performed within 4 weeks prior to enrollment

    • The post-operative MRIs should be performed ideally within 48 hours after surgery if possible
  • Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1, or 2. Use Karnofsky for patients > 16 years of age and Lansky for patients =< 16 years of age
  • Patients must have the ability to swallow whole capsules
  • Patients must have receptive and expressive language skills in English or Spanish to complete the quality of life (QOL) and neurocognitive assessments
  • All patients and/or their parents or legal guardians must sign a written informed consent.
  • All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met.

Exclusion Criteria:

  • Patients must not have received any prior tumor-directed therapy including chemotherapy, radiation therapy, immunotherapy, or bone marrow transplant. Prior surgical intervention is permitted
  • Patients with a concurrent malignancy or history of treatment (other than surgery) for another tumor within the last year are ineligible
  • Patients may not be receiving any other investigational agents
  • Patients with any serious medical or psychiatric illness/ condition, including substance use disorders likely in the judgement of the investigator to interfere or limit compliance with study requirements/treatment are not eligible
  • Patients who, in the opinion of the investigator, are not able to comply with the study procedures are not eligible
  • Female patients who are pregnant are not eligible since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required for female patients of childbearing potential
  • Lactating females who plan to breastfeed their infants are not eligible

  • Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation and for 12 weeks after stopping study therapy are not eligible

    • Note: Women of child-bearing potential and males with sexual partners who are pregnant or who could become pregnant (i.e., women of child-bearing potential) should use effective methods of contraception for the duration of the study and for 12 weeks after stopping study therapy to avoid pregnancy and/or potential adverse effects on the developing embryo

  • Cardiac conditions:

    • Known genetic disorder that increases risk for coronary artery disease. Note: The presence of dyslipidemia in a family with a history of myocardial infarction is not in itself an exclusion unless there is a known genetic disorder documented
    • Symptomatic heart failure
    • New York Heart Association (NYHA) class II-IV prior or current cardiomyopathy
    • Severe valvular heart disease
    • History of atrial fibrillation

  • Ophthalmologic conditions:

    • Current or past history of central serous retinopathy
    • Current or past history of retinal vein occlusion or retinal detachment

    • Patients with uncontrolled glaucoma

      • If checking pressure is clinically indicated, patients with intraocular pressure (IOP) > 22 mmHg or ULN adjusted by age are not eligible
    • Ophthalmological findings secondary to long-standing optic pathway glioma (such as visual loss, optic nerve pallor, or strabismus) or longstanding orbito-temporal plexiform neurofibroma (PN), such as visual loss, strabismus) will NOT be considered a significant abnormality for the purposes of the study

  • Treatments and/or medications patient is receiving that would make her/him ineligible, such as:

    • Supplementation with vitamin E greater than 100% of the daily recommended dose. Any multivitamin containing vitamin E must be stopped prior to study enrollment even if less than 100% of the daily recommended dosing for vitamin E

    • Surgery within 2 weeks prior to enrollment, with the exception of surgical placement for vascular access or cerebrospinal fluid (CSF) diverting procedures such as endoscopic third ventriculostomy (ETV) and ventriculo-peritoneal (VP) shunt.

      • Note: Patients must have healed from any prior surgery prior to enrollment
  • Patients who have an uncontrolled infection are not eligible

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Active Comparator: Arm I (carboplatin, vincristine)

INDUCTION: Patients receive carboplatin IV over 60 minutes on days 1, 8, 15, 22, 43, 50, 57, and 64 and vincristine IV or IV push over 1 minute on days 1, 8, 15, 22, 29, 36, 43, 50, 57, and 64 in the absence of disease progression or unacceptable toxicity. Patients also undergo MRI during screening and on study.

MAINTENANCE: Patients receive carboplatin IV over 60 minutes on days 1, 8, 15, and 22 and vincristine IV or IV push over 1 minute on days 1, 8, and 15. Treatment repeats every 6 weeks for 8 cycles in the absence of disease progression or unacceptable toxicity. Patients also undergo MRI on study and during follow-up.
Drug: Carboplatin
Given IV
Other Names:
  • Blastocarb
  • Carboplat
  • Carboplatin Hexal
  • Carboplatino
  • Carboplatinum
  • Carbosin
  • Carbosol
  • Carbotec
  • CBDCA
  • Displata
  • Ercar
  • JM-8
  • Nealorin
  • Novoplatinum
  • Paraplatin
  • Paraplatin AQ
  • Paraplatine
  • Platinwas
  • Ribocarbo
  • JM8
Other: Quality-of-Life Assessment
Ancillary studies
Other Names:
  • Quality of Life Assessment
Other: Questionnaire Administration
Ancillary studies
Procedure: Magnetic Resonance Imaging
Undergo MRI
Other Names:
  • MRI
  • Magnetic Resonance
  • Magnetic Resonance Imaging Scan
  • Medical Imaging, Magnetic Resonance / Nuclear Magnetic Resonance
  • MR
  • MR Imaging
  • MRI Scan
  • NMR Imaging
  • NMRI
  • Nuclear Magnetic Resonance Imaging
  • Magnetic Resonance Imaging (MRI)
  • sMRI
  • Magnetic resonance imaging (procedure)
  • MRIs
  • Structural MRI
Drug: Vincristine Sulfate
Given IV or IV push
Other Names:
  • Oncovin
  • Kyocristine
  • Leurocristine Sulfate
  • Leurocristine, sulfate
  • Vincasar
  • Vincosid
  • Vincrex
  • Vincristine, sulfate
Experimental: Arm II (selumetinib sulfate)
Patients receive selumetinib sulfate PO BID on days 1-28. Treatment is continuous and repeats every 28 days for 27 cycles in the absence of disease progression or unacceptable toxicity. Patients also undergo MRI throughout the trial.
Other: Quality-of-Life Assessment
Ancillary studies
Other Names:
  • Quality of Life Assessment
Other: Questionnaire Administration
Ancillary studies
Procedure: Magnetic Resonance Imaging
Undergo MRI
Other Names:
  • MRI
  • Magnetic Resonance
  • Magnetic Resonance Imaging Scan
  • Medical Imaging, Magnetic Resonance / Nuclear Magnetic Resonance
  • MR
  • MR Imaging
  • MRI Scan
  • NMR Imaging
  • NMRI
  • Nuclear Magnetic Resonance Imaging
  • Magnetic Resonance Imaging (MRI)
  • sMRI
  • Magnetic resonance imaging (procedure)
  • MRIs
  • Structural MRI
Drug: Selumetinib Sulfate
Given PO
Other Names:
  • AZD-6244 Hydrogen Sulfate
  • AZD6244 Hydrogen Sulfate
  • AZD6244 Hydrogen Sulphate
  • Koselugo
  • Selumetinib Sulphate

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Event-free survival (EFS)
Time Frame: From randomization to the first occurrence of any of the following events: clinical or radiographic disease progression, disease recurrence, second malignant neoplasm, or death from any cause, assessed up to 3 years after accrual completion
EFS is defined as time from randomization to the first occurrence of any of the following events: clinical or radiographic disease progression, disease recurrence, second malignant neoplasm, or death from any cause. Patients who are event-free will be censored at the time of last follow-up. Hazard ratio based on a stratified Cox proportional hazards model will be reported, along with a 90% confidence interval.
From randomization to the first occurrence of any of the following events: clinical or radiographic disease progression, disease recurrence, second malignant neoplasm, or death from any cause, assessed up to 3 years after accrual completion
Number of participants with visual acuity (VA) improvement per arm
Time Frame: Baseline and end of about 12 months of treatment
VA will be assessed using Teller acuity cards (TAC). A significant improvement in VA will be defined as a decrease of >= 0.2 logMAR (corrected for age) from baseline (pre-treatment baseline) to end of about 12 months of treatment. The primary analysis will be based on per subject outcome (rather than per eye).
Baseline and end of about 12 months of treatment

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Radiographic tumor response rate
Time Frame: Assessed up to 3 years after accrual completion
Tumors will be classified into complete response (CR), partial response (PR), stable disease (SD), and progressive disease (PD). Radiologic response rates will be summarized per arm and be tested for a difference between the two arms using an exact binomial test.
Assessed up to 3 years after accrual completion
Overall survival (OS)
Time Frame: From randomization until death from any cause or till the time of last follow-up for patients who are alive at the time of analysis, assessed up to 3 years after accrual completion
OS is defined as the time from randomization until death from any cause or till the time of last follow-up for patients who are alive at the time of analysis. Hazard ratio will be reported, along with a 90% confidence interval.
From randomization until death from any cause or till the time of last follow-up for patients who are alive at the time of analysis, assessed up to 3 years after accrual completion
Change in VA using HOTV letter acuity testing
Time Frame: Baseline and end of about 12 months of treatment
HOTV is a recognition acuity measure. It will be conducted on patients who are developmentally able to perform this testing.
Baseline and end of about 12 months of treatment
Change in motor function
Time Frame: Baseline and approximately 12 months of treatment
The Vineland-3 Motor Scale from the Comprehensive Parent Rating Form will be used to assess motor deficits. Change in Vineland motor scale from baseline to about 12 months of treatment will be compared between two treatment arms.
Baseline and approximately 12 months of treatment
Change in quality of life (QOL)
Time Frame: Baseline and 12 months of treatment
Will be measured by Pain and Hurt subscale score. QOL will be assessed by Pediatric Quality of Life (PedsQL) Generic and Brain Tumor modules. Analysis will be based on a 2-sample t-test comparing change in Pain and Hurt subscale score from baseline to 12 months for the two arms.
Baseline and 12 months of treatment
Change in quality of life (QOL)
Time Frame: Baseline and 12 months of treatment
Will be measured by Movement and Balance subscale score. QOL will be assessed by Pediatric Quality of Life (PedsQL) Generic and Brain Tumor modules. Analysis will be based on a 2-sample t-test comparing change in Movement and Balance subscale score from baseline to 12 months for the two arms.
Baseline and 12 months of treatment
Change in executive function
Time Frame: Baseline and 24 months of treatment
Will be measured by BRIEF Cognitive Regulation Index (CRI). Executive function will be measured by age-appropriate Behavior Rating Inventory of Executive Function (BRIEF) questionnaire. Analysis will be based on a 2-sample t-test comparing change in the designated score from baseline to 24 months for the two arms.
Baseline and 24 months of treatment
Change in neurocognitive function
Time Frame: Baseline and 24 months of treatment
Will be measured by Cogstate composite score. Neurocognitive function will be measured by a computerized battery (Cogstate) testing. Analysis will be based on a 2-sample t-test comparing change in Cogstate composite score from baseline to 24 months for the two arms.
Baseline and 24 months of treatment

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in circumpapillary retinal nerve fiber layer (cpRNFL) thickness by treatment arm
Time Frame: Baseline and 12 months
cpRNFL thickness is a measure of optical coherence tomography (OCT). This assessment will be conducted in a subgroup of consenting patients with optic pathway gliomas (OPGs) treated at select COG institutions with the expertise to utilize this technology. Analysis will be conducted on a per-eye basis.
Baseline and 12 months
cpRNFL thickness at baseline by visual acuity (VA) treatment response
Time Frame: Baseline and 12 months
Data will be analyzed on a per-eye basis. Children with OPGs will be classified into decline in VA versus (vs.) stable/improved VA, depending on VA treatment response at 12 months. cpRNFL thickness prior to treatment initiation will be compared.
Baseline and 12 months
cpRNFL thickness change over time by visual acuity (VA) treatment response
Time Frame: Baseline and 12 months
Data will be analyzed on a per-eye basis. Children with OPGs will be classified into decline in VA vs. stable/improved VA, depending on VA treatment response at 12 months. cpRNFL thickness change over time will be compared.
Baseline and 12 months
Change in macular ganglion cell - inner plexiform layer (GCIPL) thickness by treatment arm
Time Frame: Baseline and 12 months
GCIPL thickness is another measure of optical coherence tomography (OCT). This assessment will be conducted in a subgroup of consenting patients with optic pathway gliomas (OPGs) treated at select COG institutions with the expertise to utilize this technology. Analysis will be conducted on a per-eye basis.
Baseline and 12 months
GCIPL thickness at baseline by visual acuity (VA) treatment response
Time Frame: Baseline and 12 months
Data will be analyzed on a per-eye basis. Children with OPGs will be classified into decline in VA vs. stable/improved VA, depending on VA treatment response at 12 months. GCIPL thickness prior to treatment initiation will be compared.
Baseline and 12 months
GCIPL thickness change over time by visual acuity (VA) treatment response
Time Frame: Baseline and 12 months
Data will be analyzed on a per-eye basis. Children with OPGs will be classified into decline in VA vs. stable/improved VA, depending on VA treatment response at 12 months. GCIPL thickness change over time will be compared.
Baseline and 12 months
Novel semi-automated volumetric magnetic resonance imaging (MRI) measure at 3 months
Time Frame: 3 months on study
Optic pathway tumors will be classified into progressive disease (PD), stable disease (SD), partial response (PR), and complete response (CR) using novel semi-automated volumetric MRI measurements. This assessment will include patients with OPGs consenting to volumetric MRI study.
3 months on study
Percent change in tumor size between volumetric measure and traditional 2-dimentional (2-D) measure at 3 months
Time Frame: 3 months on study
This assessment includes participants with OPGs consenting to volumetric MRI study.
3 months on study
Novel semi-automated volumetric MRI measure at 6 months
Time Frame: 6 months on study
Optic pathway tumors will be classified into progressive disease (PD), stable disease (SD), partial response (PR), and complete response (CR) using novel semi-automated volumetric MRI measurements. This assessment will include patients with OPGs consenting to volumetric MRI study.
6 months on study
Percent change in tumor size between volumetric measure and traditional 2-dimentional (2-D) measure at 6 months
Time Frame: 6 months on study
This assessment includes participants with OPGs consenting to volumetric MRI study.
6 months on study
Novel semi-automated volumetric MRI measure at 9 months
Time Frame: 9 months on study
Optic pathway tumors will be classified into progressive disease (PD), stable disease (SD), partial response (PR), and complete response (CR) using novel semi-automated volumetric MRI measurements. This assessment will include patients with OPGs consenting to volumetric MRI study.
9 months on study
Percent change in tumor size between volumetric measure and traditional 2-dimentional (2-D) measure at 9 months
Time Frame: 9 months on study
This assessment includes participants with OPGs consenting to volumetric MRI study.
9 months on study
Novel semi-automated volumetric MRI measure at 12 months
Time Frame: 12 months on study
Optic pathway tumors will be classified into progressive disease (PD), stable disease (SD), partial response (PR), and complete response (CR) using novel semi-automated volumetric MRI measurements. This assessment will include patients with OPGs consenting to volumetric MRI study.
12 months on study
Percent change in tumor size between volumetric measure and traditional 2-dimentional (2-D) measure at 12 months
Time Frame: 12 months on study
This assessment includes participants with OPGs consenting to volumetric MRI study.
12 months on study
Novel semi-automated volumetric MRI measure at 15 months
Time Frame: 15 months on study
Optic pathway tumors will be classified into progressive disease (PD), stable disease (SD), partial response (PR), and complete response (CR) using novel semi-automated volumetric MRI measurements. This assessment will include patients with OPGs consenting to volumetric MRI study.
15 months on study
Percent change in tumor size between volumetric measure and traditional 2-dimentional (2-D) measure at 15 months
Time Frame: 15 months on study
This assessment includes participants with OPGs consenting to volumetric MRI study.
15 months on study
Tumor and blood banking
Time Frame: Up to 10 years
Up to 10 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

National Cancer Institute (NCI)

Investigators

  • Principal Investigator: Jason R Fangusaro, Children's Oncology Group

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 15, 2020

Primary Completion (Estimated)

May 1, 2027

Study Completion (Estimated)

May 1, 2027

Study Registration Dates

First Submitted

March 11, 2019

First Submitted That Met QC Criteria

March 11, 2019

First Posted (Actual)

March 12, 2019

Study Record Updates

Last Update Posted (Actual)

April 18, 2024

Last Update Submitted That Met QC Criteria

April 17, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NCI-2019-01396 (Registry Identifier: CTRP (Clinical Trial Reporting Program))
  • U10CA180886 (U.S. NIH Grant/Contract)
  • ACNS1831 (Other Identifier: CTEP)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

NCI is committed to sharing data in accordance with NIH policy. For more details on how clinical trial data is shared, access the link to the NIH data sharing policy page

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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