- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03213678
Samotolisib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With TSC or PI3K/MTOR Mutations (A Pediatric MATCH Treatment Trial)
NCI-COG Pediatric MATCH (Molecular Analysis For Therapy Choice)- Phase 2 Subprotocol of LY3023414 in Patients With Solid Tumors
Study Overview
Status
Conditions
- Malignant Glioma
- Rhabdoid Tumor
- Advanced Malignant Solid Neoplasm
- Refractory Malignant Solid Neoplasm
- Recurrent Ependymoma
- Recurrent Ewing Sarcoma
- Recurrent Hepatoblastoma
- Recurrent Langerhans Cell Histiocytosis
- Recurrent Malignant Germ Cell Tumor
- Recurrent Malignant Solid Neoplasm
- Recurrent Medulloblastoma
- Recurrent Neuroblastoma
- Recurrent Non-Hodgkin Lymphoma
- Recurrent Osteosarcoma
- Recurrent Peripheral Primitive Neuroectodermal Tumor
- Recurrent Rhabdomyosarcoma
- Recurrent Soft Tissue Sarcoma
- Refractory Langerhans Cell Histiocytosis
- Refractory Malignant Germ Cell Tumor
- Refractory Neuroblastoma
- Refractory Non-Hodgkin Lymphoma
- Wilms Tumor
- Recurrent Glioma
- Ann Arbor Stage III Non-Hodgkin Lymphoma
- Ann Arbor Stage IV Non-Hodgkin Lymphoma
- Recurrent Primary Central Nervous System Neoplasm
- Refractory Primary Central Nervous System Neoplasm
- Stage III Osteosarcoma AJCC v7
- Stage III Soft Tissue Sarcoma AJCC v7
- Stage IV Osteosarcoma AJCC v7
- Stage IV Soft Tissue Sarcoma AJCC v7
- Stage IVA Osteosarcoma AJCC v7
- Stage IVB Osteosarcoma AJCC v7
Detailed Description
PRIMARY OBJECTIVE:
I. To determine the objective response rate (ORR; complete response + partial response) in pediatric patients treated with samotolisib (LY3023414) with advanced solid tumors, non-Hodgkin lymphomas or central nervous system (CNS) tumors that harbor TSC loss of function mutations, and/or other PI3K/MTOR activating mutations.
SECONDARY OBJECTIVES:
I. To estimate the progression free survival in pediatric patients treated with LY3023414 with advanced solid tumors, non-Hodgkin lymphomas or CNS tumors that harbor TSC loss of function mutations, and/or other PI3K/MTOR activating mutations.
II. To obtain information about the tolerability of LY3023414 in children with relapsed or refractory cancer.
III. To characterize the pharmacokinetics of LY3023414 in children with recurrent or refractory cancer.
EXPLORATORY OBJECTIVES:
I. To increase knowledge of the genomic landscape of relapsed pediatric solid tumors and lymphomas and identify potential predictive biomarkers (other than the genomic alteration for which study treatment was assigned) using additional genomic, transcriptomic, and proteomic testing platforms.
II. To explore approaches to profiling changes in tumor genomics over time through evaluation of circulating tumor deoxyribonucleic acid (DNA).
III. To evaluate the frequency and mechanism of biallelic loss of function, and evaluate the expression of TSC1, TSC2, and PTEN in subjects who enroll with a loss of function mutation in one of these genes.
OUTLINE: This is a dose-escalation study.
Patients receive samotolisib orally (PO) twice daily (BID) on days 1-28. Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unexpected toxicity. Patients undergo an x-ray, computed tomography (CT), magnetic resonance imaging (MRI), fludeoxyglucose F-18 (FDG)-position emission tomography (FDG-PET), and blood sample collection on study.
After completion of study treatment, patients are followed up periodically.
Study Type
Enrollment (Actual)
Phase
- Phase 2
Contacts and Locations
Study Locations
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San Juan, Puerto Rico, 00926
- University Pediatric Hospital
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San Juan, Puerto Rico, 00912
- San Jorge Children's Hospital
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Alabama
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Birmingham, Alabama, United States, 35233
- Children's Hospital of Alabama
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Alaska
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Anchorage, Alaska, United States, 99508
- Providence Alaska Medical Center
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Arizona
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Mesa, Arizona, United States, 85202
- Banner Children's at Desert
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Tucson, Arizona, United States, 85719
- Banner University Medical Center - Tucson
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Arkansas
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Little Rock, Arkansas, United States, 72202-3591
- Arkansas Children's Hospital
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California
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Downey, California, United States, 90242
- Kaiser Permanente Downey Medical Center
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Duarte, California, United States, 91010
- City of Hope Comprehensive Cancer Center
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Loma Linda, California, United States, 92354
- Loma Linda University Medical Center
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Long Beach, California, United States, 90806
- Miller Children's and Women's Hospital Long Beach
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Los Angeles, California, United States, 90027
- Children's Hospital Los Angeles
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Los Angeles, California, United States, 90095
- Mattel Children's Hospital UCLA
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Madera, California, United States, 93636
- Valley Children's Hospital
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Oakland, California, United States, 94609
- UCSF Benioff Children's Hospital Oakland
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Oakland, California, United States, 94611
- Kaiser Permanente-Oakland
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Sacramento, California, United States, 95817
- University of California Davis Comprehensive Cancer Center
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San Francisco, California, United States, 94158
- UCSF Medical Center-Mission Bay
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Colorado
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Aurora, Colorado, United States, 80045
- Children's Hospital Colorado
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Denver, Colorado, United States, 80218
- Rocky Mountain Hospital for Children-Presbyterian Saint Luke's Medical Center
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Connecticut
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New Haven, Connecticut, United States, 06520
- Yale University
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Delaware
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Wilmington, Delaware, United States, 19803
- Alfred I duPont Hospital for Children
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District of Columbia
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Washington, District of Columbia, United States, 20010
- Children's National Medical Center
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Washington, District of Columbia, United States, 20007
- MedStar Georgetown University Hospital
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Florida
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Gainesville, Florida, United States, 32610
- University of Florida Health Science Center - Gainesville
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Jacksonville, Florida, United States, 32207
- Nemours Children's Clinic-Jacksonville
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Miami, Florida, United States, 33155
- Nicklaus Children's Hospital
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Miami, Florida, United States, 33136
- University of Miami Miller School of Medicine-Sylvester Cancer Center
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Orlando, Florida, United States, 32827
- Nemours Children's Hospital
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Orlando, Florida, United States, 32803
- AdventHealth Orlando
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Orlando, Florida, United States, 32806
- Arnold Palmer Hospital for Children
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Pensacola, Florida, United States, 32504
- Nemours Children's Clinic - Pensacola
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Saint Petersburg, Florida, United States, 33701
- Johns Hopkins All Children's Hospital
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Tampa, Florida, United States, 33607
- Saint Joseph's Hospital/Children's Hospital-Tampa
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Georgia
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Atlanta, Georgia, United States, 30322
- Children's Healthcare of Atlanta - Egleston
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Savannah, Georgia, United States, 31404
- Memorial Health University Medical Center
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Idaho
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Boise, Idaho, United States, 83712
- Saint Luke's Cancer Institute - Boise
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Illinois
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Chicago, Illinois, United States, 60637
- University of Chicago Comprehensive Cancer Center
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Maywood, Illinois, United States, 60153
- Loyola University Medical Center
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Peoria, Illinois, United States, 61637
- Saint Jude Midwest Affiliate
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Springfield, Illinois, United States, 62702
- Southern Illinois University School of Medicine
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Indiana
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Indianapolis, Indiana, United States, 46202
- Riley Hospital for Children
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Iowa
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Des Moines, Iowa, United States, 50309
- Blank Children's Hospital
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Iowa City, Iowa, United States, 52242
- University of Iowa/Holden Comprehensive Cancer Center
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Kentucky
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Louisville, Kentucky, United States, 40202
- Norton Children's Hospital
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Louisiana
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New Orleans, Louisiana, United States, 70121
- Ochsner Medical Center Jefferson
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New Orleans, Louisiana, United States, 70118
- Children's Hospital New Orleans
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Maine
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Bangor, Maine, United States, 04401
- Eastern Maine Medical Center
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Maryland
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Baltimore, Maryland, United States, 21287
- Johns Hopkins University/Sidney Kimmel Cancer Center
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Baltimore, Maryland, United States, 21215
- Sinai Hospital of Baltimore
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Massachusetts
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Boston, Massachusetts, United States, 02215
- Dana-Farber Cancer Institute
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Boston, Massachusetts, United States, 02114
- Massachusetts General Hospital Cancer Center
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Michigan
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Ann Arbor, Michigan, United States, 48109
- C S Mott Children's Hospital
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East Lansing, Michigan, United States, 48824
- Michigan State University Clinical Center
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Grand Rapids, Michigan, United States, 49503
- Helen DeVos Children's Hospital at Spectrum Health
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Kalamazoo, Michigan, United States, 49007
- Bronson Methodist Hospital
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Minnesota
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Minneapolis, Minnesota, United States, 55404
- Children's Hospitals and Clinics of Minnesota - Minneapolis
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Minneapolis, Minnesota, United States, 55455
- University of Minnesota/Masonic Cancer Center
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Mississippi
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Jackson, Mississippi, United States, 39216
- University of Mississippi Medical Center
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Missouri
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Kansas City, Missouri, United States, 64108
- Children's Mercy Hospitals and Clinics
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Saint Louis, Missouri, United States, 63110
- Washington University School of Medicine
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Saint Louis, Missouri, United States, 63141
- Mercy Hospital Saint Louis
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Saint Louis, Missouri, United States, 63104
- Cardinal Glennon Children's Medical Center
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Nebraska
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Omaha, Nebraska, United States, 68198
- University of Nebraska Medical Center
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Omaha, Nebraska, United States, 68114
- Children's Hospital and Medical Center of Omaha
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Nevada
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Las Vegas, Nevada, United States, 89144
- Summerlin Hospital Medical Center
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Las Vegas, Nevada, United States, 89109
- Sunrise Hospital and Medical Center
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Las Vegas, Nevada, United States, 89135
- Alliance for Childhood Diseases/Cure 4 the Kids Foundation
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Las Vegas, Nevada, United States, 89102
- University Medical Center of Southern Nevada
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New Jersey
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Hackensack, New Jersey, United States, 07601
- Hackensack University Medical Center
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Morristown, New Jersey, United States, 07960
- Morristown Medical Center
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New Brunswick, New Jersey, United States, 08901
- Saint Peter's University Hospital
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New York
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Albany, New York, United States, 12208
- Albany Medical Center
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Bronx, New York, United States, 10467
- Montefiore Medical Center - Moses Campus
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Buffalo, New York, United States, 14263
- Roswell Park Cancer Institute
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New Hyde Park, New York, United States, 11040
- The Steven and Alexandra Cohen Children's Medical Center of New York
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New York, New York, United States, 10065
- Memorial Sloan Kettering Cancer Center
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New York, New York, United States, 10032
- NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center
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New York, New York, United States, 10065
- NYP/Weill Cornell Medical Center
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Rochester, New York, United States, 14642
- University of Rochester
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Syracuse, New York, United States, 13210
- State University of New York Upstate Medical University
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Valhalla, New York, United States, 10595
- New York Medical College
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North Carolina
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Asheville, North Carolina, United States, 28801
- Mission Hospital
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Charlotte, North Carolina, United States, 28203
- Carolinas Medical Center/Levine Cancer Institute
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Charlotte, North Carolina, United States, 28204
- Novant Health Presbyterian Medical Center
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Durham, North Carolina, United States, 27710
- Duke University Medical Center
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North Dakota
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Fargo, North Dakota, United States, 58122
- Sanford Broadway Medical Center
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Ohio
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Cincinnati, Ohio, United States, 45229
- Cincinnati Children's Hospital Medical Center
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Cleveland, Ohio, United States, 44195
- Cleveland Clinic Foundation
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Cleveland, Ohio, United States, 44106
- Rainbow Babies and Childrens Hospital
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Columbus, Ohio, United States, 43205
- Nationwide Children's Hospital
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Dayton, Ohio, United States, 45404
- Dayton Children's Hospital
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Toledo, Ohio, United States, 43606
- ProMedica Toledo Hospital/Russell J Ebeid Children's Hospital
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Oklahoma
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Oklahoma City, Oklahoma, United States, 73104
- University of Oklahoma Health Sciences Center
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Oregon
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Portland, Oregon, United States, 97239
- Oregon Health and Science University
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Portland, Oregon, United States, 97227
- Legacy Emanuel Children's Hospital
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Pennsylvania
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Danville, Pennsylvania, United States, 17822
- Geisinger Medical Center
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Philadelphia, Pennsylvania, United States, 19104
- Children's Hospital of Philadelphia
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Pittsburgh, Pennsylvania, United States, 15224
- Children's Hospital of Pittsburgh of UPMC
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South Carolina
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Columbia, South Carolina, United States, 29203
- Prisma Health Richland Hospital
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Greenville, South Carolina, United States, 29605
- BI-LO Charities Children's Cancer Center
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South Dakota
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Sioux Falls, South Dakota, United States, 57117-5134
- Sanford USD Medical Center - Sioux Falls
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Tennessee
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Knoxville, Tennessee, United States, 37916
- East Tennessee Childrens Hospital
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Memphis, Tennessee, United States, 38105
- Saint Jude Children's Research Hospital
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Nashville, Tennessee, United States, 37232
- Vanderbilt University/Ingram Cancer Center
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Nashville, Tennessee, United States, 37203
- The Children's Hospital at TriStar Centennial
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Texas
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Austin, Texas, United States, 78723
- Dell Children's Medical Center of Central Texas
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Dallas, Texas, United States, 75390
- UT Southwestern/Simmons Cancer Center-Dallas
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Dallas, Texas, United States, 75230
- Medical City Dallas Hospital
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Fort Worth, Texas, United States, 76104
- Cook Children's Medical Center
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Houston, Texas, United States, 77030
- M D Anderson Cancer Center
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Houston, Texas, United States, 77030
- Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center
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San Antonio, Texas, United States, 78207
- Children's Hospital of San Antonio
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San Antonio, Texas, United States, 78229
- Methodist Children's Hospital of South Texas
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Temple, Texas, United States, 76508
- Scott and White Memorial Hospital
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Utah
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Salt Lake City, Utah, United States, 84113
- Primary Children's Hospital
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Vermont
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Burlington, Vermont, United States, 05405
- University of Vermont and State Agricultural College
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Virginia
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Norfolk, Virginia, United States, 23507
- Children's Hospital of The King's Daughters
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Richmond, Virginia, United States, 23298
- Virginia Commonwealth University/Massey Cancer Center
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Washington
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Seattle, Washington, United States, 98105
- Seattle Children's Hospital
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Spokane, Washington, United States, 99204
- Providence Sacred Heart Medical Center and Children's Hospital
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Tacoma, Washington, United States, 98405
- Mary Bridge Children's Hospital and Health Center
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Tacoma, Washington, United States, 98431
- Madigan Army Medical Center
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West Virginia
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Morgantown, West Virginia, United States, 26506
- West Virginia University Healthcare
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Wisconsin
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Madison, Wisconsin, United States, 53792
- University of Wisconsin Carbone Cancer Center
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Marshfield, Wisconsin, United States, 54449
- Marshfield Medical Center-Marshfield
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Milwaukee, Wisconsin, United States, 53226
- Children's Hospital of Wisconsin
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
Inclusion Criteria:
- Patient must have enrolled onto APEC1621SC and must have been given a treatment assignment to Molecular Analysis for Therapy Choice (MATCH) to APEC1621D based on the presence of an actionable mutation as defined in APEC1621SC; note that treatment assignment may be to primary cohort A for patients with TSC1 or TSC2 loss of function mutations or primary cohort B for patients with other PI3K/MTOR pathway mutations
- Patients accruing to dose level 1 must have a body surface area >= 0.52 m^2 at the time of study enrollment; patients accruing to dose level 2 must have a body surface area >= 0.37 m^2 at the time of study enrollment; patients accruing to dose level -1 must have a body surface area >= 0.75 m^2 at the time of study enrollment
Patients must have radiographically measurable disease at the time of study enrollment; patients with neuroblastoma who do not have measurable disease but have metaiodobenzylguanidine (MIBG) positive (+) evaluable disease are eligible; measurable disease in patients with CNS involvement is defined as any lesion that is at minimum 10 mm in one dimension on standard magnetic resonance imaging (MRI) or computed tomography (CT)
Note: The following do not qualify as measurable disease:
- Malignant fluid collections (e.g., ascites, pleural effusions)
- Bone marrow infiltration except that detected by MIBG scan for neuroblastoma
- Lesions only detected by nuclear medicine studies (e.g., bone, gallium or positron emission tomography [PET] scans) except as noted for neuroblastoma
- Elevated tumor markers in plasma or cerebrospinal fluid (CSF)
- Previously radiated lesions that have not demonstrated clear progression post radiation
- Leptomeningeal lesions that do not meet the measurement requirements for Response Evaluation Criteria in Solid Tumors (RECIST) 1.1
- Bone lesions without an associated soft tissue mass >= 10 mm in greatest diameter; bone lesions with an associated soft tissue mass >= 10 mm in greatest diameter imaged by CT or MRI are considered measurable
- Karnofsky >= 50% for patients > 16 years of age and Lansky >= 50 for patients =< 16 years of age; Note: neurologic deficits in patients with CNS tumors must have been stable for at least 7 days prior to study enrollment; patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score
Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment; if after the required timeframe, the numerical eligibility criteria are met, e.g. blood count criteria, the patient is considered to have recovered adequately
- Cytotoxic chemotherapy or other anti-cancer agents known to be myelosuppressive; >= 21 days after the last dose of cytotoxic or myelosuppressive chemotherapy (42 days if prior nitrosourea)
- Anti-cancer agents not known to be myelosuppressive (e.g. not associated with reduced platelet or absolute neutrophil count [ANC] counts): >= 7 days after the last dose of agent
- Antibodies: >= 21 days must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to grade =< 1
- Corticosteroids: if used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
- Hematopoietic growth factors: >= 14 days after the last dose of a long-acting growth factor (e.g. pegfilgrastim) or 7 days for short-acting growth factor; for growth factors that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur; the duration of this interval must be discussed with the study chair and the study-assigned research coordinator
- Interleukins, interferons and cytokines (other than hematopoietic growth factors): >= 21 days after the completion of interleukins, interferon or cytokines (other than hematopoietic growth factors)
Stem cell Infusions (with or without total body irradiation [TBI]):
- Allogeneic (non-autologous) bone marrow or stem cell transplant, or any stem cell infusion including donor lymphocyte infusion (DLI) or boost infusion: >= 84 days after infusion and no evidence of graft versus host disease (GVHD)
- Autologous stem cell infusion including boost infusion: >= 42 days
- Cellular therapy: >= 42 days after the completion of any type of cellular therapy (e.g. modified T cells, natural killer [NK] cells, dendritic cells, etc.)
Radiation therapy (XRT)/external beam irradiation including protons: >= 14 days after local XRT; >= 150 days after TBI, craniospinal XRT or if radiation to >= 50% of the pelvis; >= 42 days if other substantial bone marrow (BM) radiation
- Note: radiation may not be delivered to "measurable disease" tumor site(s) being used to follow response to subprotocol treatment
- Radiopharmaceutical therapy (e.g., radiolabeled antibody, iobenguane I-131 [131I-MIBG]): >= 42 days after systemically administered radiopharmaceutical therapy
- Patients must not have received prior exposure to LY3023414
- Patients must not have received prior exposure to an agent specifically directed at the PI3K/MTOR pathway (a PI3K inhibitor, an AKT inhibitor, an MTOR inhibitor, including rapalogs, or a combined PI3K/MTOR inhibitor)
For patients with solid tumors without known bone marrow involvement:
- Peripheral absolute neutrophil count (ANC) >= 1000/mm^3
- Platelet count >= 100,000/mm^3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment)
- Patients with known bone marrow metastatic disease will be eligible for study provided they meet the blood counts (may receive transfusions provided they are not known to be refractory to red cell or platelet transfusions); these patients will not be evaluable for hematologic toxicity
Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 ml/min/1.73 m^2 or a serum creatinine based on age/gender as follows:
- Age: 1 to < 2 years; maximum serum creatinine (mg/dL): male 0.6; female 0.6
- Age: 2 to < 6 years; maximum serum creatinine (mg/dL): male 0.8; female 0.8
- Age: 6 to < 10 years; maximum serum creatinine (mg/dL): male 1; female 1
- Age: 10 to < 13 years; maximum serum creatinine (mg/dL): male 1.2; female 1.2
- Age: 13 to < 16 years; maximum serum creatinine (mg/dL): male 1.5; female 1.4
- Age: >= 16 years; maximum serum creatinine (mg/dL): male 1.7; female 1.4
- Bilirubin (sum of conjugated + unconjugated) =< 1.5 x upper limit of normal (ULN) for age
- Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 135 U/L; (for the purpose of this study, the ULN for SGPT is 45 U/L)
- Serum albumin >= 2 g/dL
- Patients must have a normal blood sugar level for age; if an initial random draw (i.e. non-fasting) blood glucose value is out of range, it is acceptable to repeat this test as a fasting draw
- Patients must have a serum triglyceride level =< 300 mg/dL and serum cholesterol level =< 300 mg/dL; if an initial random draw (i.e. non-fasting) is out of range, it is acceptable to repeat this test as a fasting draw
- Patients with seizure disorder may be enrolled if on anticonvulsants and well controlled
- Nervous system disorders (by Common Terminology Criteria for Adverse Events version 5.0 [CTCAE V 5.0]) resulting from prior therapy must be =< grade 2, with the exception of decreased tendon reflex (DTR); any grade of DTR is eligible
- Corrected QT (QTc) interval =< 480 milliseconds
- Patients must be able to swallow intact tablets
- All patients and/or their parents or legally authorized representatives must sign a written informed consent; assent, when appropriate, will be obtained according to institutional guidelines
Exclusion Criteria:
- Pregnant or breast-feeding women will not be entered on this study; pregnancy tests must be obtained in girls who are post-menarchal; males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method while receiving study treatment and for 3 months after the last dose of LY3023414
Concomitant medications
- Corticosteroids: patients receiving corticosteroids who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible; if used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
- Investigational drugs: patients who are currently receiving another investigational drug are not eligible
- Anti-cancer agents: patients who are currently receiving other anti-cancer agents are not eligible
- Anti-GVHD agents post-transplant: patients who are receiving cyclosporine, tacrolimus or other agents to prevent graft-versus-host disease post bone marrow transplant are not eligible for this trial
- Patients who have an uncontrolled infection are not eligible
- Patients who have insulin dependent diabetes are not eligible
- Patients who have received a prior solid organ transplantation are not eligible
- Patients with subependymal giant cell astrocytomas (SEGAs) are not eligible
- Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: Treatment (samotolisib)
Patients receive samotolisib PO BID on days 1-28.
Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unexpected toxicity.
Patients undergo an x-ray, CT, MRI, FDG-PET, and blood sample collection on study.
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Undergo MRI
Other Names:
Undergo CT
Other Names:
Given PO
Other Names:
Undergo blood specimen collection
Other Names:
Undergo FDG-PET
Other Names:
Undergo x-ray
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Response rate
Time Frame: Up to 2 years
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Response rates will be calculated as the percent of evaluable patients who are responders, and confidence intervals will be constructed using the Wilson score interval method.
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Up to 2 years
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Percentage of patients experiencing grade 3 or 4 adverse events
Time Frame: From enrollment to the end of treatment, up to 2 years
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Will be graded by National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0.
Any eligible patient who receives at least one dose of protocol therapy will be considered in the evaluation of toxicity.
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From enrollment to the end of treatment, up to 2 years
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Progression free survival (PFS)
Time Frame: From the initiation of protocol treatment to the occurrence of any of the following events: disease progression or disease recurrence or death from any cause, assessed up to 2 years
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PFS along with the confidence intervals will be estimated using the Kaplan-Meier method.
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From the initiation of protocol treatment to the occurrence of any of the following events: disease progression or disease recurrence or death from any cause, assessed up to 2 years
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Pharmacokinetic (PK) parameters
Time Frame: At baseline, 30 minutes, 1, 2, 4, 4, 6-8 hours, and 24 hours after the morning dose on day 1 course 1, pre-dose and at 1-2 hours after the morning dose on day 15 course 1
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A descriptive analysis of pharmacokinetic (PK) parameters will be performed to define systemic exposure, drug clearance, and other pharmacokinetic parameters.
The PK parameters will be summarized with simple summary statistics, including means, medians, ranges, and standard deviations (if numbers and distribution permit).
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At baseline, 30 minutes, 1, 2, 4, 4, 6-8 hours, and 24 hours after the morning dose on day 1 course 1, pre-dose and at 1-2 hours after the morning dose on day 15 course 1
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Other Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Potential predictive biomarker identification using additional genomic, transcriptomic, and proteomic testing platforms
Time Frame: Up to 3 years
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A descriptive analysis will be performed and will be summarized with simple summary statistics.
All of these analyses will be descriptive in nature.
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Up to 3 years
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Biallelic loss of function frequency and mechanism
Time Frame: Up to 3 years
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A descriptive analysis will be performed and will be summarized with simple summary statistics.
All of these analyses will be descriptive in nature.
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Up to 3 years
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Change in tumor genomic profile
Time Frame: Baseline up to 3 years
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A descriptive analysis will be performed and will be summarized with simple summary statistics.
All of these analyses will be descriptive in nature.
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Baseline up to 3 years
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TSC1, TSC2, and PTEN expression levels
Time Frame: Up to 3 years
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A descriptive analysis will be performed and will be summarized with simple summary statistics.
All of these analyses will be descriptive in nature.
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Up to 3 years
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Collaborators and Investigators
Sponsor
Investigators
- Principal Investigator: Theodore W Laetsch, Children's Oncology Group
Publications and helpful links
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Pathologic Processes
- Nervous System Diseases
- Respiratory Tract Diseases
- Immune System Diseases
- Neoplasms, Connective and Soft Tissue
- Neoplasms by Histologic Type
- Lymphoproliferative Disorders
- Lymphatic Diseases
- Immunoproliferative Disorders
- Lung Diseases
- Urologic Neoplasms
- Urogenital Neoplasms
- Neoplasms by Site
- Kidney Diseases
- Urologic Diseases
- Neoplasms, Glandular and Epithelial
- Disease Attributes
- Genetic Diseases, Inborn
- Neoplasms, Neuroepithelial
- Neoplasms, Nerve Tissue
- Kidney Neoplasms
- Neoplastic Syndromes, Hereditary
- Neoplasms, Complex and Mixed
- Neoplasms, Bone Tissue
- Neoplasms, Connective Tissue
- Lung Diseases, Interstitial
- Neoplasms, Muscle Tissue
- Myosarcoma
- Female Urogenital Diseases
- Female Urogenital Diseases and Pregnancy Complications
- Urogenital Diseases
- Male Urogenital Diseases
- Neoplasms
- Sarcoma
- Lymphoma
- Neoplasms, Germ Cell and Embryonal
- Recurrence
- Lymphoma, Non-Hodgkin
- Glioma
- Sarcoma, Ewing
- Nervous System Neoplasms
- Central Nervous System Neoplasms
- Ependymoma
- Medulloblastoma
- Rhabdoid Tumor
- Osteosarcoma
- Neuroblastoma
- Rhabdomyosarcoma
- Neuroectodermal Tumors
- Neuroectodermal Tumors, Primitive
- Wilms Tumor
- Neuroectodermal Tumors, Primitive, Peripheral
- Hepatoblastoma
- Histiocytosis, Langerhans-Cell
- Histiocytosis
Other Study ID Numbers
- NCI-2017-01249 (Registry Identifier: CTRP (Clinical Trial Reporting Program))
- U10CA180886 (U.S. NIH Grant/Contract)
- APEC1621D (Other Identifier: CTEP)
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
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.
Clinical Trials on Malignant Glioma
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City of Hope Medical CenterNational Cancer Institute (NCI); Food and Drug Administration (FDA)Active, not recruitingRecurrent Glioblastoma | Recurrent Malignant Glioma | Refractory Malignant Glioma | Recurrent WHO Grade III Glioma | Recurrent WHO Grade II Glioma | Refractory Glioblastoma | Refractory WHO Grade II Glioma | Refractory WHO Grade III GliomaUnited States
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Henry FriedmanCompletedGrade IV Malignant GliomaUnited States
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Annick DesjardinsAmgenTerminated
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Kentuckiana Cancer InstituteEisai Inc.UnknownNewly Diagnosed Supratentorial Malignant GliomaUnited States
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AmgenCompletedGlioblastoma or Malignant GliomaUnited States, Australia, Netherlands, Germany, Spain, France
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Duke UniversityMonteris MedicalWithdrawnGlioblastoma | Malignant Glioma of BrainUnited States
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CellabMEDRecruitingRecurrent Malignant GliomaKorea, Republic of
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University of ChicagoRecruitingMalignant Glioma of BrainUnited States
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Angiochem IncCompletedRecurrent or Progressive Malignant GliomaUnited States
Clinical Trials on Magnetic Resonance Imaging
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M.D. Anderson Cancer CenterNational Cancer Institute (NCI)CompletedHematopoietic and Lymphoid Cell Neoplasm | Malignant Solid NeoplasmUnited States
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M.D. Anderson Cancer CenterNational Cancer Institute (NCI)Recruiting
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M.D. Anderson Cancer CenterActive, not recruitingProstate Adenocarcinoma | Prostate CarcinomaUnited States
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OHSU Knight Cancer InstituteOregon Health and Science UniversityRecruitingIntracranial NeoplasmUnited States
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University of California, San FranciscoTerminatedAnatomic Stage I Breast Cancer AJCC v8 | Anatomic Stage IA Breast Cancer AJCC v8 | Anatomic Stage IB Breast Cancer AJCC v8 | Anatomic Stage II Breast Cancer AJCC v8 | Anatomic Stage IIA Breast Cancer AJCC v8 | Anatomic Stage IIB Breast Cancer AJCC v8 | Anatomic Stage III Breast Cancer AJCC v8 | Anatomic... and other conditionsUnited States
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Wake Forest University Health SciencesNational Cancer Institute (NCI)CompletedStage IIIA Breast Cancer | Stage IIIB Breast Cancer | Stage IA Breast Cancer | Stage IB Breast Cancer | Stage IIA Breast Cancer | Stage IIB Breast Cancer | Stage IIIC Breast Cancer | Healthy SubjectUnited States
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Stanford UniversityTerminatedLaryngeal Neoplasms | Head and Neck Cancers | Larynx CancerUnited States
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Vanderbilt-Ingram Cancer CenterNational Cancer Institute (NCI)CompletedBreast CancerUnited States
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Abramson Cancer Center of the University of PennsylvaniaCompletedBrain TumorUnited States
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M.D. Anderson Cancer CenterNational Cancer Institute (NCI)TerminatedThoracic Spine NeoplasmUnited States