En undersøgelse med tovorafenib (DAY101) som en behandlingsmulighed for progressiv, recidiverende eller refraktær Langerhans cellehistiocytose
Fase 2 undersøgelse af Tovorafenib (DAY101) i recidiverende og refraktær Langerhans cellehistiocytose
This phase II trial tests the safety, side effects, best dose and activity of tovorafenib (DAY101) in treating patients with Langerhans cell histiocytosis that is growing, spreading, or getting worse (progressive), has come back (relapsed) after previous treatment, or does not respond to therapy (refractory). Langerhans cell histiocytosis is a type of disease that occurs when the body makes too many immature Langerhans cells (a type of white blood cell). When these cells build up, they can form tumors in certain tissues and organs including bones, skin, lungs and pituitary gland and can damage them. This tumor is more common in children and young adults. DAY101 may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Using DAY101 may be effective in treating patients with relapsed or refractory Langerhans cell histiocytosis.
Studieoversigt
Status
Intervention / Behandling
- Procedure: Bone Marrow Biopsy
- Procedure: Computed Tomography
- Procedure: Echocardiography Test
- Procedure: Multigated Acquisition Scan
- Procedure: Bone Marrow Aspiration
- Procedure: Lumbar Puncture
- Procedure: Biospecimen Collection
- Procedure: FDG-Positron Emission Tomography and Computed Tomography Scan
- Medicin: Tovorafenib
Detaljeret beskrivelse
PRIMARY OBJECTIVE:
I. To determine overall response rate (ORR) for children and young adults with relapsed or refractory BRAFV600E positive (cohort 1) and BRAFV600E negative (cohort 2) Langerhans cell histiocytosis (LCH) treated with tovorafenib (DAY101) after 2 cycles and must be maintained 4 weeks later.
SECONDARY OBJECTIVES:
I. To determine nature and severity of adverse events in patients treated with tovorafenib (DAY101) for relapsed or refractory LCH.
II. To describe event-free survival (EFS) at 1 year in children and young adults with relapsed and refractory LCH treated with tovorafenib (DAY 101) for up to 1 year.
III. To determine durability of response in children and young adults with relapsed or refractory LCH treated with tovorafenib (DAY101) following cessation of therapy in patients with complete response (CR) at 1 year.
IV. To describe progression-free (and relapse-free) survival (PFS) and overall survival (OS) in children and young adults with relapsed or refractory LCH treated with tovorafenib (DAY101) for up to 1 year.
EXPLORATORY OBJECTIVES:
I. To determine potential role of pathogenic tumor mutation in response to tovorafenib (DAY101), and to evaluate changes in bone marrow and peripheral blood cell populations carrying pathogenic mutations in response to tovorafenib (DAY101) therapy.
Ia. To define somatic mutations in LCH lesion biopsies; Ib. To determine impact of tovorafenib (DAY101) on bone marrow and blood BRAFV600E+ mononuclear cells; Ic. To determine impact of tovorafenib (DAY101) on cerebral spinal fluid and disease response; Id. To determine the performance of standardized immunohistochemical analysis of LCH lesion biopsies.
II. To compare performance of LCH-specific response criteria to Response Evaluation Criteria in Solid Tumors (RECIST) criteria.
III. To describe the pharmacokinetics of tovorafenib (DAY101) when administered to pediatric and young adult patients with relapse or refractory LCH.
OUTLINE: This is a dose escalation study of tovorafenib followed by a phase II trial.
Patients receive tovorafenib orally (PO) once weekly (QW) on days 1, 8, 15, and 22 of each cycle. Cycles repeat every 28 days for 12 cycles in the absence of disease progression or unacceptable toxicity. Patients also undergo multi-gated acquisition (MUGA) or echocardiography (ECHO) scans, and fludeoxyglucose F-18 (FDG)-positron emission tomography (PET) or computed tomography (CT) throughout the trial, and collection of blood and urine samples on study. Patients with suspicion of bone marrow and/or central nervous system involvement will also undergo bone marrow biopsy and aspiration and lumbar puncture on study and during follow up.
After completion of study treatment, patients are followed up at 28 days, months 3, 6, 9, and 12, and then at 2 years post cycle 12.
Undersøgelsestype
Tilmelding (Anslået)
Fase
- Fase 2
Kontakter og lokationer
Studiesteder
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New South Wales
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Hunter Regional Mail Centre, New South Wales, Australien, 2310
- Rekruttering
- John Hunter Children's Hospital
-
Kontakt:
- Site Public Contact
- Telefonnummer: (02) 4985 5180
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Ledende efterforsker:
- Kristy McCarthy
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Randwick, New South Wales, Australien, 2031
- Rekruttering
- Sydney Children's Hospital
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Ledende efterforsker:
- Draga Barbaric
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Kontakt:
- Site Public Contact
- Telefonnummer: (02) 9382-1721
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Queensland
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South Brisbane, Queensland, Australien, 4101
- Rekruttering
- Queensland Children's Hospital
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Ledende efterforsker:
- Steven A. Foresto
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Kontakt:
- Site Public Contact
- Telefonnummer: 61 7 3068 1111
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Victoria
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Parkville, Victoria, Australien, 3052
- Rekruttering
- Royal Children's Hospital
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Ledende efterforsker:
- Martin A. Campbell
-
Kontakt:
- Site Public Contact
- Telefonnummer: 61 3 9345 5656
- E-mail: Jordan.Hansford@rch.org.au
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Western Australia
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Perth, Western Australia, Australien, 6009
- Rekruttering
- Perth Children's Hospital
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Kontakt:
- Site Public Contact
- E-mail: helpdesk@childrensoncologygroup.org
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Ledende efterforsker:
- Michelle Ng
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Québec, Canada, G1V 4G2
- Rekruttering
- CHU de Quebec-Centre Hospitalier de l'Universite Laval (CHUL)
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Ledende efterforsker:
- Bruno Michon
-
Kontakt:
- Site Public Contact
- Telefonnummer: 418-525-4444
- E-mail: rechclinique@crchudequebec.ulaval.ca
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Alberta
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Edmonton, Alberta, Canada, T6G 2B7
- Rekruttering
- University of Alberta Hospital
-
Kontakt:
- Site Public Contact
- Telefonnummer: 780-407-8798
- E-mail: pedsoncologyresearch@ahs.ca
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Ledende efterforsker:
- Sarah J. McKillop
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British Columbia
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Vancouver, British Columbia, Canada, V6H 3V4
- Rekruttering
- British Columbia Children's Hospital
-
Kontakt:
- Site Public Contact
- Telefonnummer: 6477 604-875-2345
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Ledende efterforsker:
- Rebecca J. Deyell
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Nova Scotia
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Halifax, Nova Scotia, Canada, B3K 6R8
- Rekruttering
- IWK Health Centre
-
Kontakt:
- Site Public Contact
- Telefonnummer: 902-470-8520
- E-mail: Research@iwk.nshealth.ca
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Ledende efterforsker:
- Chelsea Ash
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Ontario
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Hamilton, Ontario, Canada, L8N 3Z5
- Suspenderet
- McMaster Children's Hospital at Hamilton Health Sciences
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London, Ontario, Canada, N6A 5W9
- Rekruttering
- Children's Hospital
-
Kontakt:
- Site Public Contact
- Telefonnummer: 519-685-8306
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Ledende efterforsker:
- Shayna M. Zelcer
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Toronto, Ontario, Canada, M5G 1X8
- Rekruttering
- Hospital for Sick Children
-
Kontakt:
- Site Public Contact
- Telefonnummer: 416-813-7654
- E-mail: ask.CRS@sickkids.ca
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Ledende efterforsker:
- Oussama A. Abla
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Quebec
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Montreal, Quebec, Canada, H3H 1P3
- Rekruttering
- The Montreal Children's Hospital of the MUHC
-
Kontakt:
- Site Public Contact
- Telefonnummer: 514-412-4445
- E-mail: info@thechildren.com
-
Ledende efterforsker:
- Stephanie Mourad
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Montreal, Quebec, Canada, H3T 1C5
- Rekruttering
- Centre Hospitalier Universitaire Sainte-Justine
-
Ledende efterforsker:
- Monia Marzouki
-
Kontakt:
- Site Public Contact
- Telefonnummer: 514-345-4931
- E-mail: yvan.samson@umontreal.ca
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Sherbrooke, Quebec, Canada, J1H 5N4
- Rekruttering
- Centre Hospitalier Universitaire de Sherbrooke-Fleurimont
-
Kontakt:
- Site Public Contact
- Telefonnummer: 819-820-6480
- E-mail: crcinformation.chus@ssss.gouv.qc.ca
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Ledende efterforsker:
- Stephanie Vairy
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-
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Alabama
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Birmingham, Alabama, Forenede Stater, 35233
- Rekruttering
- Children's Hospital of Alabama
-
Kontakt:
- Site Public Contact
- Telefonnummer: 205-638-9285
- E-mail: oncologyresearch@peds.uab.edu
-
Ledende efterforsker:
- Matthew A. Kutny
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Arkansas
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Little Rock, Arkansas, Forenede Stater, 72202-3591
- Rekruttering
- Arkansas Children's Hospital
-
Ledende efterforsker:
- Michael W. Bishop
-
Kontakt:
- Site Public Contact
- Telefonnummer: 501-364-7373
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California
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Downey, California, Forenede Stater, 90242
- Rekruttering
- Kaiser Permanente Downey Medical Center
-
Kontakt:
- Site Public Contact
- Telefonnummer: 626-564-3455
-
Ledende efterforsker:
- Robert M. Cooper
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Loma Linda, California, Forenede Stater, 92354
- Rekruttering
- Loma Linda University Medical Center
-
Kontakt:
- Site Public Contact
- Telefonnummer: 909-558-4050
-
Ledende efterforsker:
- Albert Kheradpour
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Long Beach, California, Forenede Stater, 90806
- Rekruttering
- Miller Children's and Women's Hospital Long Beach
-
Kontakt:
- Site Public Contact
- Telefonnummer: 562-933-5600
-
Ledende efterforsker:
- Jacqueline N. Casillas
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Los Angeles, California, Forenede Stater, 90027
- Rekruttering
- Children's Hospital Los Angeles
-
Kontakt:
- Site Public Contact
- Telefonnummer: 323-361-4110
-
Ledende efterforsker:
- Rebecca S. Parker
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Madera, California, Forenede Stater, 93636
- Aktiv, ikke rekrutterende
- Valley Children's Hospital
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Oakland, California, Forenede Stater, 94611
- Rekruttering
- Kaiser Permanente-Oakland
-
Kontakt:
- Site Public Contact
- Telefonnummer: 877-642-4691
- E-mail: Kpoct@kp.org
-
Ledende efterforsker:
- Aarati V. Rao
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Oakland, California, Forenede Stater, 94609
- Rekruttering
- UCSF Benioff Children's Hospital Oakland
-
Ledende efterforsker:
- Jennifer G. Michlitsch
-
Kontakt:
- Site Public Contact
- Telefonnummer: 510-428-3264
- E-mail: PedOncRschOAK@ucsf.edu
-
Orange, California, Forenede Stater, 92868
- Rekruttering
- Children's Hospital of Orange County
-
Kontakt:
- Site Public Contact
- Telefonnummer: 714-509-8646
- E-mail: oncresearch@choc.org
-
Ledende efterforsker:
- Elyssa M. Rubin
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Palo Alto, California, Forenede Stater, 94304
- Rekruttering
- Lucile Packard Children's Hospital Stanford University
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Kontakt:
- Site Public Contact
- Telefonnummer: 800-694-0012
- E-mail: ccto-office@stanford.edu
-
Ledende efterforsker:
- Jay Michael S. Balagtas
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San Francisco, California, Forenede Stater, 94158
- Rekruttering
- UCSF Medical Center-Mission Bay
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Kontakt:
- Site Public Contact
- Telefonnummer: 877-827-3222
- E-mail: cancertrials@ucsf.edu
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Ledende efterforsker:
- Natalie L. Wu
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Colorado
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Aurora, Colorado, Forenede Stater, 80045
- Rekruttering
- Children's Hospital Colorado
-
Kontakt:
- Site Public Contact
- Telefonnummer: 303-764-5056
- E-mail: josh.b.gordon@nsmtp.kp.org
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Ledende efterforsker:
- Timothy P. Garrington
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Denver, Colorado, Forenede Stater, 80218
- Rekruttering
- Rocky Mountain Hospital for Children-Presbyterian Saint Luke's Medical Center
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Kontakt:
- Site Public Contact
- Telefonnummer: 303-832-2344
- E-mail: PSGResearchSharedMailbox@HCAHealthcare.com
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Ledende efterforsker:
- Florence Choo
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Connecticut
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Hartford, Connecticut, Forenede Stater, 06106
- Rekruttering
- Connecticut Children's Medical Center
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Kontakt:
- Site Public Contact
- Telefonnummer: 860-545-9981
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Ledende efterforsker:
- Michael S. Isakoff
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New Haven, Connecticut, Forenede Stater, 06520
- Rekruttering
- Yale University
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Kontakt:
- Site Public Contact
- Telefonnummer: 203-785-5702
- E-mail: canceranswers@yale.edu
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Ledende efterforsker:
- Farzana Pashankar
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Delaware
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Wilmington, Delaware, Forenede Stater, 19803
- Rekruttering
- Alfred I duPont Hospital for Children
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Kontakt:
- Site Public Contact
- Telefonnummer: 302-651-5572
- E-mail: Allison.bruce@nemours.org
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Ledende efterforsker:
- Vibhuti Agarwal
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District of Columbia
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Washington D.C., District of Columbia, Forenede Stater, 20010
- Rekruttering
- Children's National Medical Center
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Ledende efterforsker:
- Jeffrey S. Dome
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Kontakt:
- Site Public Contact
- Telefonnummer: 202-476-2800
- E-mail: OncCRC_OnCall@childrensnational.org
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Florida
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Fort Myers, Florida, Forenede Stater, 33908
- Rekruttering
- Golisano Children's Hospital of Southwest Florida
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Kontakt:
- Site Public Contact
- Telefonnummer: 239-343-5333
- E-mail: molly.arnstrom@leehealth.org
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Ledende efterforsker:
- Emad K. Salman
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Gainesville, Florida, Forenede Stater, 32610
- Rekruttering
- UF Health Cancer Institute - Gainesville
-
Ledende efterforsker:
- Brian Stover
-
Kontakt:
- Site Public Contact
- Telefonnummer: 352-273-8010
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Hollywood, Florida, Forenede Stater, 33021
- Rekruttering
- Memorial Regional Hospital/Joe DiMaggio Children's Hospital
-
Kontakt:
- Site Public Contact
- Telefonnummer: 954-265-1847
- E-mail: OHR@mhs.net
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Ledende efterforsker:
- Iftikhar Hanif
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Jacksonville, Florida, Forenede Stater, 32207
- Rekruttering
- Nemours Children's Clinic-Jacksonville
-
Kontakt:
- Site Public Contact
- Telefonnummer: 302-651-5572
- E-mail: Allison.bruce@nemours.org
-
Ledende efterforsker:
- Vibhuti Agarwal
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Miami, Florida, Forenede Stater, 33155
- Rekruttering
- Nicklaus Children's Hospital
-
Kontakt:
- Site Public Contact
- Telefonnummer: 888-624-2778
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Ledende efterforsker:
- Ziad A. Khatib
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Miami, Florida, Forenede Stater, 33136
- Suspenderet
- University of Miami Miller School of Medicine-Sylvester Cancer Center
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Orlando, Florida, Forenede Stater, 32806
- Rekruttering
- Arnold Palmer Hospital for Children
-
Kontakt:
- Site Public Contact
- Telefonnummer: 321-841-5357
- E-mail: Jennifer.spinelli@orlandohealth.com
-
Ledende efterforsker:
- Zhongbo Hu
-
Orlando, Florida, Forenede Stater, 32827
- Rekruttering
- Nemours Children's Hospital
-
Kontakt:
- Site Public Contact
- Telefonnummer: 302-651-5572
- E-mail: Allison.bruce@nemours.org
-
Ledende efterforsker:
- Vibhuti Agarwal
-
Pensacola, Florida, Forenede Stater, 32504
- Rekruttering
- Nemours Children's Clinic - Pensacola
-
Kontakt:
- Site Public Contact
- E-mail: helpdesk@childrensoncologygroup.org
-
Ledende efterforsker:
- Jeffrey H. Schwartz
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St. Petersburg, Florida, Forenede Stater, 33701
- Rekruttering
- Johns Hopkins All Children's Hospital
-
Kontakt:
- Site Public Contact
- Telefonnummer: 727-767-4784
- E-mail: Ashley.Repp@jhmi.edu
-
Ledende efterforsker:
- Jennifer B. Dean
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Tampa, Florida, Forenede Stater, 33607
- Rekruttering
- Saint Joseph's Hospital/Children's Hospital-Tampa
-
Kontakt:
- Site Public Contact
- Telefonnummer: 813-357-0849
- E-mail: jennifer.manns@baycare.org
-
Ledende efterforsker:
- Don E. Eslin
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Georgia
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Atlanta, Georgia, Forenede Stater, 30329
- Rekruttering
- Children's Healthcare of Atlanta - Arthur M Blank Hospital
-
Ledende efterforsker:
- Kathryn S. Sutton
-
Kontakt:
- Site Public Contact
- Telefonnummer: 404-785-0232
- E-mail: Olivia.Floyd@choa.org
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Savannah, Georgia, Forenede Stater, 31404
- Rekruttering
- Memorial Health University Medical Center
-
Kontakt:
- Site Public Contact
- Telefonnummer: 912-350-7887
- E-mail: Lorraine.OHara@hcahealthcare.com
-
Ledende efterforsker:
- James N. Yarnall
-
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Hawaii
-
Honolulu, Hawaii, Forenede Stater, 96826
- Rekruttering
- Kapiolani Medical Center for Women and Children
-
Kontakt:
- Site Public Contact
- Telefonnummer: 808-983-6090
-
Ledende efterforsker:
- Wade T. Kyono
-
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Illinois
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Chicago, Illinois, Forenede Stater, 60611
- Rekruttering
- Lurie Children's Hospital-Chicago
-
Kontakt:
- Site Public Contact
- Telefonnummer: 773-880-4562
-
Ledende efterforsker:
- Joanna L. Weinstein
-
Chicago, Illinois, Forenede Stater, 60637
- Rekruttering
- University of Chicago Comprehensive Cancer Center
-
Kontakt:
- Site Public Contact
- Telefonnummer: 773-702-8222
- E-mail: cancerclinicaltrials@bsd.uchicago.edu
-
Ledende efterforsker:
- Perry C. Morocco
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Peoria, Illinois, Forenede Stater, 61637
- Rekruttering
- OSF Children's Hospital of Illinois
-
Ledende efterforsker:
- Prerna Kumar
-
Kontakt:
- Site Public Contact
- Telefonnummer: 309-624-4945
- E-mail: ChildrensHospitalofIllinois@osfhealthcare.org
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Indiana
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Indianapolis, Indiana, Forenede Stater, 46202
- Rekruttering
- Riley Hospital for Children
-
Kontakt:
- Site Public Contact
- Telefonnummer: 800-248-1199
-
Ledende efterforsker:
- Jennifer A. Belsky
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Iowa
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Des Moines, Iowa, Forenede Stater, 50309
- Rekruttering
- Blank Children's Hospital
-
Kontakt:
- Site Public Contact
- Telefonnummer: 515-241-8912
- E-mail: samantha.mallory@unitypoint.org
-
Ledende efterforsker:
- Samantha L. Mallory
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Iowa City, Iowa, Forenede Stater, 52242
- Rekruttering
- University of Iowa/Holden Comprehensive Cancer Center
-
Kontakt:
- Site Public Contact
- Telefonnummer: 800-237-1225
-
Ledende efterforsker:
- Andrew P. Groves
-
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Kentucky
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Lexington, Kentucky, Forenede Stater, 40536
- Rekruttering
- University of Kentucky/Markey Cancer Center
-
Kontakt:
- Site Public Contact
- Telefonnummer: 859-257-3379
-
Ledende efterforsker:
- James T. Badgett
-
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Louisiana
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New Orleans, Louisiana, Forenede Stater, 70118
- Rekruttering
- Children's Hospital New Orleans
-
Ledende efterforsker:
- Maria C. Velez-Yanguas
-
Kontakt:
- Site Public Contact
- Telefonnummer: 504-894-5377
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New Orleans, Louisiana, Forenede Stater, 70121
- Rekruttering
- Ochsner Medical Center Jefferson
-
Kontakt:
- Site Public Contact
- Telefonnummer: 504-842-8084
- E-mail: Elisemarie.curry@ochsner.org
-
Ledende efterforsker:
- Craig Lotterman
-
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Maryland
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Baltimore, Maryland, Forenede Stater, 21287
- Rekruttering
- Johns Hopkins University/Sidney Kimmel Cancer Center
-
Kontakt:
- Site Public Contact
- Telefonnummer: 410-955-8804
- E-mail: jhcccro@jhmi.edu
-
Ledende efterforsker:
- Elias Zambidis
-
Baltimore, Maryland, Forenede Stater, 21215
- Rekruttering
- Sinai Hospital of Baltimore
-
Ledende efterforsker:
- Jason M. Fixler
-
Kontakt:
- Site Public Contact
- Telefonnummer: 410-601-9083
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Michigan
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East Lansing, Michigan, Forenede Stater, 48823
- Suspenderet
- Michigan State University
-
Grand Rapids, Michigan, Forenede Stater, 49503
- Rekruttering
- Corewell Health Grand Rapids Hospitals - Helen DeVos Children's Hospital
-
Kontakt:
- Site Public Contact
- Telefonnummer: 616-391-1230
- E-mail: crcwm-regulatory@crcwm.org
-
Ledende efterforsker:
- Kathleen Y. Butler
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Kalamazoo, Michigan, Forenede Stater, 49007
- Rekruttering
- Bronson Methodist Hospital
-
Kontakt:
- Site Public Contact
- Telefonnummer: 616-391-1230
- E-mail: crcwm-regulatory@crcwm.org
-
Ledende efterforsker:
- Kathleen Y. Butler
-
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Minnesota
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Minneapolis, Minnesota, Forenede Stater, 55404
- Rekruttering
- Children's Hospitals and Clinics of Minnesota - Minneapolis
-
Ledende efterforsker:
- Michael K. Richards
-
Kontakt:
- Site Public Contact
- Telefonnummer: 612-813-5913
- E-mail: pauline.mitby@childrensmn.org
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Minneapolis, Minnesota, Forenede Stater, 55455
- Aktiv, ikke rekrutterende
- University of Minnesota/Masonic Cancer Center
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-
Mississippi
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Jackson, Mississippi, Forenede Stater, 39216
- Rekruttering
- University of Mississippi Medical Center
-
Kontakt:
- Site Public Contact
- Telefonnummer: 601-815-6700
-
Ledende efterforsker:
- Amanda Strobel
-
-
Missouri
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St Louis, Missouri, Forenede Stater, 63110
- Rekruttering
- Washington University School of Medicine
-
Kontakt:
- Site Public Contact
- Telefonnummer: 800-600-3606
- E-mail: info@siteman.wustl.edu
-
Ledende efterforsker:
- Bryan A. Sisk
-
St Louis, Missouri, Forenede Stater, 63141
- Rekruttering
- Mercy Hospital Saint Louis
-
Kontakt:
- Site Public Contact
- Telefonnummer: 314-251-7066
-
Ledende efterforsker:
- Robin D. Hanson
-
St Louis, Missouri, Forenede Stater, 63104
- Rekruttering
- Cardinal Glennon Children's Medical Center
-
Kontakt:
- Site Public Contact
- Telefonnummer: 314-268-4000
-
Ledende efterforsker:
- William S. Ferguson
-
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Nebraska
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Omaha, Nebraska, Forenede Stater, 68114
- Rekruttering
- Children's Hospital and Medical Center of Omaha
-
Kontakt:
- Site Public Contact
- Telefonnummer: 402-955-3949
-
Ledende efterforsker:
- Jill C. Beck
-
Omaha, Nebraska, Forenede Stater, 68198
- Rekruttering
- University of Nebraska Medical Center
-
Kontakt:
- Site Public Contact
- Telefonnummer: 402-559-6941
- E-mail: unmcrsa@unmc.edu
-
Ledende efterforsker:
- Jill C. Beck
-
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Nevada
-
Las Vegas, Nevada, Forenede Stater, 89135
- Rekruttering
- Alliance for Childhood Diseases/Cure 4 the Kids Foundation
-
Kontakt:
- Site Public Contact
- Telefonnummer: 702-384-0013
- E-mail: research@sncrf.org
-
Ledende efterforsker:
- Joseph L. Lasky
-
Las Vegas, Nevada, Forenede Stater, 89144
- Rekruttering
- Summerlin Hospital Medical Center
-
Kontakt:
- Site Public Contact
- Telefonnummer: 702-384-0013
- E-mail: research@sncrf.org
-
Ledende efterforsker:
- Joseph L. Lasky
-
-
New Jersey
-
Hackensack, New Jersey, Forenede Stater, 07601
- Rekruttering
- Hackensack University Medical Center
-
Ledende efterforsker:
- Katharine Offer
-
Kontakt:
- Site Public Contact
- Telefonnummer: 551-996-2897
-
Morristown, New Jersey, Forenede Stater, 07960
- Rekruttering
- Morristown Medical Center
-
Kontakt:
- Site Public Contact
- Telefonnummer: 973-971-5900
-
Ledende efterforsker:
- Kathryn L. Laurie
-
New Brunswick, New Jersey, Forenede Stater, 08903
- Rekruttering
- Rutgers Cancer Institute of New Jersey-Robert Wood Johnson University Hospital
-
Ledende efterforsker:
- Marissa Botwinick
-
Kontakt:
- Site Public Contact
- Telefonnummer: 732-235-8675
-
Paterson, New Jersey, Forenede Stater, 07503
- Rekruttering
- Saint Joseph's Regional Medical Center
-
Kontakt:
- Site Public Contact
- Telefonnummer: 973-754-2207
- E-mail: HallL@sjhmc.org
-
Ledende efterforsker:
- Alissa Kahn
-
-
New York
-
Albany, New York, Forenede Stater, 12208
- Rekruttering
- Albany Medical Center
-
Kontakt:
- Site Public Contact
- Telefonnummer: 518-262-5513
-
Ledende efterforsker:
- Lauren R. Weintraub
-
Brooklyn, New York, Forenede Stater, 11219
- Rekruttering
- Maimonides Medical Center
-
Kontakt:
- Site Public Contact
- Telefonnummer: 718-765-2500
-
Ledende efterforsker:
- Mahmut Y. Celiker
-
New Hyde Park, New York, Forenede Stater, 11040
- Rekruttering
- The Steven and Alexandra Cohen Children's Medical Center of New York
-
Kontakt:
- Site Public Contact
- Telefonnummer: 718-470-3460
-
Ledende efterforsker:
- Julie I. Krystal
-
New York, New York, Forenede Stater, 10032
- Rekruttering
- NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center
-
Ledende efterforsker:
- Nobuko Hijiya
-
Kontakt:
- Site Public Contact
- Telefonnummer: 212-342-5162
- E-mail: cancerclinicaltrials@cumc.columbia.edu
-
New York, New York, Forenede Stater, 10065
- Rekruttering
- Memorial Sloan Kettering Cancer Center
-
Kontakt:
- Site Public Contact
- Telefonnummer: 212-639-7592
-
Ledende efterforsker:
- Christopher J. Forlenza
-
New York, New York, Forenede Stater, 10016
- Rekruttering
- Laura and Isaac Perlmutter Cancer Center at NYU Langone
-
Ledende efterforsker:
- Elizabeth A. Raetz
-
Kontakt:
- Site Public Contact
- E-mail: CancerTrials@nyulangone.org
-
New York, New York, Forenede Stater, 10029
- Rekruttering
- Mount Sinai Hospital
-
Kontakt:
- Site Public Contact
- Telefonnummer: 212-824-7309
- E-mail: CCTO@mssm.edu
-
Ledende efterforsker:
- Pamela R. Merola
-
New York, New York, Forenede Stater, 10065
- Rekruttering
- NYP/Weill Cornell Medical Center
-
Kontakt:
- Site Public Contact
- Telefonnummer: 212-746-1848
-
Ledende efterforsker:
- Nitya Gulati
-
Syracuse, New York, Forenede Stater, 13210
- Rekruttering
- State University of New York Upstate Medical University
-
Kontakt:
- Site Public Contact
- Telefonnummer: 315-464-5476
-
Ledende efterforsker:
- Melanie A. Comito
-
The Bronx, New York, Forenede Stater, 10467
- Rekruttering
- Montefiore Medical Center - Moses Campus
-
Kontakt:
- Site Public Contact
- Telefonnummer: 718-379-6866
- E-mail: eskwak@montefiore.org
-
Ledende efterforsker:
- Alice Lee
-
Valhalla, New York, Forenede Stater, 10595
- Aktiv, ikke rekrutterende
- New York Medical College
-
-
North Carolina
-
Asheville, North Carolina, Forenede Stater, 28801
- Rekruttering
- Mission Hospital
-
Kontakt:
- Site Public Contact
- Telefonnummer: 828-213-7055
- E-mail: NCDV.ResearchRegulatory@HCAHealthcare.com
-
Ledende efterforsker:
- Douglas J. Scothorn
-
Durham, North Carolina, Forenede Stater, 27710
- Rekruttering
- Duke University Medical Center
-
Ledende efterforsker:
- Jessica M. Sun
-
Kontakt:
- Site Public Contact
- Telefonnummer: 888-275-3853
-
Greenville, North Carolina, Forenede Stater, 27834
- Rekruttering
- East Carolina University
-
Kontakt:
- Site Public Contact
- Telefonnummer: 252-744-1015
- E-mail: eubankss@ecu.edu
-
Ledende efterforsker:
- Andrea R. Whitfield
-
Winston-Salem, North Carolina, Forenede Stater, 27157
- Rekruttering
- Wake Forest University Health Sciences
-
Kontakt:
- Site Public Contact
- Telefonnummer: 336-713-6771
-
Ledende efterforsker:
- Sarah Supples
-
-
Ohio
-
Cleveland, Ohio, Forenede Stater, 44106
- Rekruttering
- Rainbow Babies and Childrens Hospital
-
Kontakt:
- Site Public Contact
- Telefonnummer: 216-844-5437
-
Ledende efterforsker:
- Duncan S. Stearns
-
Columbus, Ohio, Forenede Stater, 43205
- Rekruttering
- Nationwide Children's Hospital
-
Kontakt:
- Site Public Contact
- Telefonnummer: 614-722-6039
- E-mail: Melinda.Triplet@nationwidechildrens.org
-
Ledende efterforsker:
- Mark A. Ranalli
-
Dayton, Ohio, Forenede Stater, 45404
- Rekruttering
- Dayton Children's Hospital
-
Kontakt:
- Site Public Contact
- Telefonnummer: 800-228-4055
-
Ledende efterforsker:
- Jordan M. Wright
-
-
Oklahoma
-
Oklahoma City, Oklahoma, Forenede Stater, 73104
- Rekruttering
- University of Oklahoma Health Sciences Center
-
Kontakt:
- Site Public Contact
- Telefonnummer: 405-271-8777
- E-mail: ou-clinical-trials@ouhsc.edu
-
Ledende efterforsker:
- Rene Y. McNall-Knapp
-
-
Oregon
-
Portland, Oregon, Forenede Stater, 97239
- Rekruttering
- Oregon Health and Science University
-
Kontakt:
- Site Public Contact
- Telefonnummer: 503-494-1080
- E-mail: trials@ohsu.edu
-
Ledende efterforsker:
- Katrina Winsnes
-
-
Pennsylvania
-
Danville, Pennsylvania, Forenede Stater, 17822
- Rekruttering
- Geisinger Medical Center
-
Ledende efterforsker:
- Jagadeesh Ramdas
-
Kontakt:
- Site Public Contact
- Telefonnummer: 570-271-5251
- E-mail: cancerresearch@geisinger.edu
-
Philadelphia, Pennsylvania, Forenede Stater, 19104
- Rekruttering
- Children's Hospital of Philadelphia
-
Ledende efterforsker:
- Michael D. Hogarty
-
Kontakt:
- Site Public Contact
- Telefonnummer: 267-425-5544
- E-mail: CancerTrials@email.chop.edu
-
Philadelphia, Pennsylvania, Forenede Stater, 19134
- Rekruttering
- Saint Christopher's Hospital for Children
-
Kontakt:
- Site Public Contact
- Telefonnummer: 215-427-8991
-
Ledende efterforsker:
- Gregory E. Halligan
-
Pittsburgh, Pennsylvania, Forenede Stater, 15224
- Rekruttering
- Children's Hospital of Pittsburgh of UPMC
-
Kontakt:
- Site Public Contact
- Telefonnummer: 412-692-8570
- E-mail: jean.tersak@chp.edu
-
Ledende efterforsker:
- Julia E. Segal
-
-
South Carolina
-
Columbia, South Carolina, Forenede Stater, 29203
- Rekruttering
- Prisma Health Richland Hospital
-
Ledende efterforsker:
- Stuart L. Cramer
-
Kontakt:
- Site Public Contact
- Telefonnummer: 864-522-4317
- E-mail: Kim.Williams3@prismahealth.org
-
Greenville, South Carolina, Forenede Stater, 29605
- Rekruttering
- BI-LO Charities Children's Cancer Center
-
Ledende efterforsker:
- Aniket Saha
-
Kontakt:
- Site Public Contact
- Telefonnummer: 864-522-4317
- E-mail: Kim.Williams3@prismahealth.org
-
-
South Dakota
-
Sioux Falls, South Dakota, Forenede Stater, 57117-5134
- Rekruttering
- Sanford USD Medical Center - Sioux Falls
-
Kontakt:
- Site Public Contact
- Telefonnummer: 605-312-3320
- E-mail: OncologyClinicalTrialsSF@SanfordHealth.org
-
Ledende efterforsker:
- Jordan Fritch-Hanson
-
-
Tennessee
-
Knoxville, Tennessee, Forenede Stater, 37916
- Rekruttering
- East Tennessee Childrens Hospital
-
Kontakt:
- Site Public Contact
- Telefonnummer: 865-541-8266
-
Ledende efterforsker:
- Susan E. Spiller
-
Nashville, Tennessee, Forenede Stater, 37232
- Rekruttering
- Vanderbilt University/Ingram Cancer Center
-
Kontakt:
- Site Public Contact
- Telefonnummer: 800-811-8480
-
Ledende efterforsker:
- Devang J. Pastakia
-
Nashville, Tennessee, Forenede Stater, 37203
- Rekruttering
- The Children's Hospital at TriStar Centennial
-
Kontakt:
- Site Public Contact
- Telefonnummer: 615-342-1919
-
Ledende efterforsker:
- Clinton M. Carroll
-
-
Texas
-
Austin, Texas, Forenede Stater, 78723
- Rekruttering
- Dell Children's Medical Center of Central Texas
-
Kontakt:
- Site Public Contact
- Telefonnummer: 512-628-1902
- E-mail: TXAUS-DL-SFCHemonc.research@ascension.org
-
Ledende efterforsker:
- Shannon M. Cohn
-
Dallas, Texas, Forenede Stater, 75390
- Rekruttering
- UT Southwestern/Simmons Cancer Center-Dallas
-
Kontakt:
- Site Public Contact
- Telefonnummer: 214-648-7097
- E-mail: canceranswerline@UTSouthwestern.edu
-
Ledende efterforsker:
- Erin B. Butler
-
El Paso, Texas, Forenede Stater, 79905
- Rekruttering
- El Paso Children's Hospital
-
Kontakt:
- Site Public Contact
- Telefonnummer: 915-298-5444
- E-mail: ranjan.bista@ttuhsc.edu
-
Ledende efterforsker:
- Benjamin Carcamo
-
Houston, Texas, Forenede Stater, 77030
- Rekruttering
- Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center
-
Kontakt:
- Site Public Contact
- Telefonnummer: 713-798-1354
- E-mail: burton@bcm.edu
-
Ledende efterforsker:
- Olive S. Eckstein
-
Houston, Texas, Forenede Stater, 77030
- Rekruttering
- UT MD Anderson Cancer Center
-
Kontakt:
- Site Public Contact
- Telefonnummer: 866-632-6789
- E-mail: askmdanderson@mdanderson.org
-
Ledende efterforsker:
- Najat C. Daw
-
San Antonio, Texas, Forenede Stater, 78207
- Rekruttering
- Children's Hospital of San Antonio
-
Kontakt:
- Site Public Contact
- Telefonnummer: 210-704-2894
- E-mail: bridget.medina@christushealth.org
-
Ledende efterforsker:
- Julie Voeller
-
San Antonio, Texas, Forenede Stater, 78229
- Rekruttering
- University of Texas Health Science Center at San Antonio
-
Kontakt:
- Site Public Contact
- Telefonnummer: 210-450-3800
- E-mail: phoresearchoffice@uthscsa.edu
-
Ledende efterforsker:
- Allison C. Grimes
-
San Antonio, Texas, Forenede Stater, 78229
- Rekruttering
- Methodist Children's Hospital of South Texas
-
Kontakt:
- Site Public Contact
- Telefonnummer: 210-575-6240
- E-mail: Vinod.GidvaniDiaz@hcahealthcare.com
-
Ledende efterforsker:
- Jose M. Esquilin
-
Temple, Texas, Forenede Stater, 76508
- Rekruttering
- Scott and White Memorial Hospital
-
Kontakt:
- Site Public Contact
- Telefonnummer: 254-724-5407
-
Ledende efterforsker:
- Nicholas W. McGregor
-
-
Utah
-
Salt Lake City, Utah, Forenede Stater, 84113
- Rekruttering
- Primary Children's Hospital
-
Kontakt:
- Site Public Contact
- Telefonnummer: 801-585-5270
-
Ledende efterforsker:
- Taumoha Ghosh
-
-
Virginia
-
Charlottesville, Virginia, Forenede Stater, 22908
- Rekruttering
- University of Virginia Cancer Center
-
Kontakt:
- Site Public Contact
- Telefonnummer: 434-243-6303
- E-mail: uvacancertrials@hscmail.mcc.virginia.edu
-
Ledende efterforsker:
- Brian C. Belyea
-
Norfolk, Virginia, Forenede Stater, 23507
- Rekruttering
- Children's Hospital of The King's Daughters
-
Kontakt:
- Site Public Contact
- Telefonnummer: 757-668-7243
- E-mail: CCBDCresearch@chkd.org
-
Ledende efterforsker:
- Melissa S. Mark
-
Richmond, Virginia, Forenede Stater, 23298
- Rekruttering
- VCU Massey Comprehensive Cancer Center
-
Ledende efterforsker:
- Nadirah El-Amin
-
Kontakt:
- Site Public Contact
- Telefonnummer: 804-628-6430
- E-mail: CTOclinops@vcu.edu
-
-
Washington
-
Seattle, Washington, Forenede Stater, 98105
- Rekruttering
- Seattle Children's Hospital
-
Kontakt:
- Site Public Contact
- Telefonnummer: 866-987-2000
-
Ledende efterforsker:
- Sarah E. Leary
-
Spokane, Washington, Forenede Stater, 99204
- Rekruttering
- Providence Sacred Heart Medical Center and Children's Hospital
-
Kontakt:
- Site Public Contact
- Telefonnummer: 800-228-6618
- E-mail: HopeBeginsHere@providence.org
-
Ledende efterforsker:
- Judy L. Felgenhauer
-
-
West Virginia
-
Charleston, West Virginia, Forenede Stater, 25304
- Rekruttering
- Vandalia Health/CAMC Institute for Academic Medicine
-
Ledende efterforsker:
- Mohamad H. Badawi
-
Kontakt:
- Site Public Contact
- Telefonnummer: 304-388-9944
- E-mail: CAMCClinicalTrials@vandaliahealth.org
-
-
Wisconsin
-
Green Bay, Wisconsin, Forenede Stater, 54301
- Rekruttering
- Saint Vincent Hospital Cancer Center Green Bay
-
Ledende efterforsker:
- Catherine A. Long
-
Kontakt:
- Site Public Contact
- Telefonnummer: 920-433-8889
- E-mail: WI_research_admin@hshs.org
-
Milwaukee, Wisconsin, Forenede Stater, 53226
- Rekruttering
- Children's Hospital of Wisconsin
-
Ledende efterforsker:
- Peter H. Shaw
-
Kontakt:
- Site Public Contact
- Telefonnummer: 414-955-4727
- E-mail: MACCCTO@mcw.edu
-
-
Deltagelseskriterier
Berettigelseskriterier
Aldre berettiget til at studere
Tager imod sunde frivillige
Køn, der er berettiget til at studere
Beskrivelse
Inclusion Criteria:
-
180 days- < 22 years (at time of study enrollment)
-
Patient must have a body surface area of ≥ 0.3 m^2
-
Patients with progressive, relapsed, or recurrent LCH with measurable disease at study entry
-
Patients must have had histologic verification of LCH (from either original diagnosis or relapse/progression) at the time of study entry
- Tissue confirmation of relapse is recommended but not required.
- Pathology report must be submitted for central confirmation of diagnosis within 7 days of enrollment.
- Formalin-fixed paraffin-embedded (FFPE) blocks or unstained slides (initial diagnosis and/or subsequent biopsies) will be required for retrospective central confirmation of diagnosis and molecular studies
- Patients with mixed histiocytic disorders (e.g. LCH with juvenile xanthogranuloma) may be included
-
Patients must have measurable disease
-
Patients must have progressive or refractory disease or experience relapse after at least one previous systemic treatment strategy
-
Pathogenic somatic mutation detected in genes encoding tyrosine kinase receptors (CSFR1, ERBB3 or ALK), RAS or RAF (may be from original or subsequent biopsy or peripheral blood/bone marrow aspirate). Clinical mutation reports may include quantitative polymerase chain reaction (PCR) (e.g. BRAFV600E) and/or Sanger or next generation sequencing. Immunohistochemistry (e.g. VE1 antibody for BRAFV600E) alone is not sufficient
-
-
Participant must be able to take an enteral dose and formulation of medication. Study medication is only available as an oral suspension or tablet, which may be taken by mouth or other enteral route such as nasogastric, jejunostomy, or gastric tube
-
Karnofsky >= 50% for patients > 16 years of age and Lansky >= 50% for patients =< 16 years of age
-
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
-
Myelosuppressive chemotherapy: Patients must not have received within 14 days of entry onto this study
-
Investigational agent or any other anticancer therapy not defined above: Patients must not have received any investigational agent or any other anticancer therapy (including MAPK pathway inhibitor) for at least 14 days prior to planned start of tovorafenib (DAY101)
-
Radiation therapy (RT): Patient must not have received RT within 2 weeks after the last dose fraction of RT
-
Patients must have fully recovered from any prior surgery
-
Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, targeted inhibitor, and/or radiotherapy with toxicities reduced to grade 1 or less (Common Terminology Criteria for Adverse Events [CTCAE] version 5.0)
-
Steroids: =< 0.5 mg/kg/day of prednisone equivalent (maximum 20 mg/day) averaged during the month prior to study enrollment is permissible
-
Strong inducers or inhibitors of CYP2C8 are prohibited for 14 days before the first dose of tovorafenib (DAY101) and from planned administration for the duration of study participation
-
Medications that are breast cancer resistant protein (BCRP) substrates that have a narrow therapeutic index are prohibited for 14 days before the first dose of tovorafenib (DAY101) and for the duration of study participation
-
Peripheral absolute neutrophil count (ANC) >= 750/uL unless secondary to bone marrow involvement, in such cases bone marrow involvement must be documented (must be performed within 7 days prior to enrollment, must be repeated prior to the start of protocol therapy if > 7 days have elapsed from their most recent prior assessment)
-
Platelet count >= 75,000/uL (unsupported/without transfusion within the past 7 days) (must be performed within 7 days prior to enrollment, must be repeated prior to the start of protocol therapy if > 7 days have elapsed from their most recent prior assessment)
-
Patients with marrow disease must have platelet count of >= 75,000/uL (transfusion support allowed) and must not be refractory to platelet transfusions. Bone marrow involvement must be documented
-
Hemoglobin >= 8 g/dL (unsupported/without transfusion within the past 7 days). Patients with marrow disease must have hemoglobin >= 8 g/dL (transfusion support allowed). Bone marrow involvement must be documented
-
Hematopoietic growth factors: At least 14 days after the last dose of a long-acting growth factor (e.g., Neulasta [registered trademark]) or 7 days for short-acting growth factor
-
A serum creatinine based on age/sex as follows (must be performed within 7 days prior to enrollment, must be repeated prior to the start of protocol therapy if > 7 days have elapsed from their most recent prior assessment)
- Age: 6 months to < 1 year; Maximum Serum Creatinine (mg/dL):= 0.5 mg/dl (male and female)
- Age: 1 to < 2 years; Maximum Serum Creatinine (mg/dL): = 0.6 mg/dl (male and female)
- Age: 2 to < 6 years; Maximum Serum Creatinine (mg/dL): = 0.8 mg/dl (male and female)
- Age: 6 to < 10 years; Maximum Serum Creatinine (mg/dL): = 1.0 mg/dl (male and female)
- Age: 10 to < 13 years; Maximum Serum Creatinine (mg/dL): = 1.2 mg/dl (male and female)
- Age: 13 to < 16 years; Maximum Serum Creatinine (mg/dL): = 1.5 mg/dl (male) and 1.4 mg/dl (female)
- Age: >= 16 years; Maximum Serum Creatinine (mg/dL): = 1.7 mg/dl (male) and 1.4 mg/dl (female)
- OR- a 24 hour urine creatinine clearance >= 50 mL/min/1.73 m^2
- OR- a glomerular filtration rate (GFR) >= 50 mL/min/1.73 m^2. GFR must be performed using direct measurement with a nuclear blood sampling method OR direct small molecule clearance method (iothalamate or other molecule per institutional standard)
- Note: Estimated GFR (eGFR) from serum creatinine, cystatin C or other estimates are not acceptable for determining eligibility
-
Bilirubin (sum of conjugated + unconjugated) =< 1.5 x upper limit of normal (ULN) for age (must be performed within 7 days prior to enrollment, must be repeated prior to the start of protocol therapy if > 7 days have elapsed from their most recent prior assessment)
-
Alanine aminotransferase (ALT) =< 3 x ULN for age (must be performed within 7 days prior to enrollment, must be repeated prior to the start of protocol therapy if > 7 days have elapsed from their most recent prior assessment)
-
Serum albumin >= 2 g/dl must be performed within 7 days prior to enrollment, must be repeated prior to the start of protocol therapy if > 7 days have elapsed from their most recent prior assessment)
-
For patients with liver disease caused by their histiocytic disorder (as evaluated on radiographic imaging or biopsy): patients may be enrolled with abnormal bilirubin, aspartate aminotransferase (AST), ALT and albumin with documentation of histiocytic liver disease
-
Fractional shortening (FS) of >= 25% or ejection fraction of >= 50%, as determined by echocardiography or multigated acquisition scan (MUGA) within 28 days prior to study enrollment. Depending on institutional standard, either FS or left ventricular ejection fraction (LVEF) is adequate for enrollment if only one value is measured; if both values are measured, then both values must meet criteria above (must be obtained within 28 days prior to enrollment and start of protocol therapy) (repeat if necessary)
-
No evidence of dyspnea at rest, no exercise intolerance, and a pulse oximetry > 94% if there is clinical indication for determination; unless it is due to underlying pulmonary LCH
-
Central Nervous System Function Defined As:
- Patients with seizure disorder may be enrolled if well controlled
- Central nervous system (CNS) toxicity =< Grade 2
-
Human immunodeficiency virus (HIV) infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial unless antiretroviral therapy interacts with the metabolism of tovorafenib (DAY101) and cannot safely be changed to antivirals that do not interact with study medication
-
All patients and/or their parent(s) 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:
-
LCH arising along with other hematologic malignancy (e.g. mixed LCH with acute lymphoblastic leukemia) or any history of non-histiocytic malignancy
-
Disease scenarios as below will be excluded
- Skin-limited disease
- Gastrointestinal (GI) tract involvement only (those that have disease that can be determined by endoscopic biopsies only)
- LCH-associated neurodegeneration (LCH-ND) without parenchymal lesions or other systemic lesions
-
Patients with activating mutations in MAP2K1 are not eligible for this study due to drug target specificity. Mutation status will be submitted to study team within 7 days of enrollment
-
Refractory nausea and vomiting, malabsorption, or external biliary shunt that would preclude adequate absorption of tovorafenib (DAY101)
-
Uncontrolled systemic bacterial, viral, or fungal infection
-
Major surgical procedure or significant traumatic injury within 14 days prior to study enrollment, or anticipation of need for major surgical procedure during the course of the study. Placement of a vascular access device or minor surgery is permitted within fourteen (14) days of study enrollment (provided that the wound has healed)
-
History of significant bowel resection that would preclude adequate absorption or other significant malabsorptive disease
-
Ophthalmologic considerations: Patients with known significant ophthalmologic conditions or known risk factors for retinal vein occlusion (RVO) or central serous retinopathy (CSR) are not eligible
-
History of solid organ or hematopoietic bone marrow transplantation
-
Clinically significant active cardiovascular disease, or history of myocardial infarction, or deep vein thrombosis/pulmonary embolism within 6 months prior to enrollment, ongoing cardiomyopathy, or current prolonged QT interval > 440 ms based on triplicate electrocardiogram (ECG) average
-
History of Grade >= 2 CNS hemorrhage or history of any CNS hemorrhage within 28 days of study entry
-
History of any drug reaction with eosinophilia and systemic symptoms (DRESS) syndrome or Stevens Johnsons syndrome (SJS) or who are allergic to tovorafenib (DAY101) or any of its components
-
CTCAE version (V.) 5.0 Grade 3 symptomatic creatinine kinase (CPK) elevation (> 5 x ULN)
-
Female patients who are pregnant are ineligible. A pregnancy test is required for female patients of childbearing potential
-
Lactating females who plan to breastfeed their infants are ineligible
-
Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation are ineligible. Women of childbearing potential must use non-hormonal contraception during tovorafenib treatment and for at least 28 days after the last dose. Men should use effective contraception and must not father a child while taking tovorafenib and for 14 days after the last dose
Studieplan
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Behandling
- Tildeling: N/A
- Interventionel model: Sekventiel tildeling
- Maskning: Ingen (Åben etiket)
Våben og indgreb
Deltagergruppe / Arm |
Intervention / Behandling |
|---|---|
|
Eksperimentel: Treatment (tovorafenib)
Patients receive tovorafenib PO QW on days 1, 8, 15, and 22 of each cycle. Cycles repeat every 28 days for 12 cycles in the absence of disease progression or unacceptable toxicity. Patients also undergo MUGA or ECHO scans, and FDG-PET or CT throughout the trial, and collection of blood and urine samples on study. Patients with suspicion of bone marrow and/or central nervous system involvement will also undergo bone marrow biopsy and aspiration and lumbar puncture on study and during follow up. |
Undergo bone marrow biopsy
Andre navne:
Undergo CT
Andre navne:
Undergo ECHO
Andre navne:
Undergo MUGA
Andre navne:
Undergo bone marrow aspiration Undergo lumbar puncture
Andre navne:
Undergo collection of blood and urine samples
Andre navne:
Undergo FDG-PET imaging
Andre navne:
Given PO
Andre navne:
|
Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Frequency of dose limiting toxicity (dose finding phase)
Tidsramme: Up to 28 days
|
Will be analyzed descriptively. |
Up to 28 days
|
|
Overall response rate (phase II)
Tidsramme: After 2 cycles of therapy (each cycle is 28 days)
|
Will be assessed using minimax Simon's two-stage designs in each of the BRAFV600E and non-BRAFV600E cohorts separately. The two arms will not be directly compared. The 95% confidence interval for the overall response rate will be adjusted for the two-stage design. |
After 2 cycles of therapy (each cycle is 28 days)
|
Sekundære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Event free survival rate
Tidsramme: At 1 and 2 years
|
Will be estimated by the Kaplan-Meier method beginning at study enrollment. Will be evaluated two years after enrollment of the last patient on the trial and estimates at specific timepoints will be presented along with log-log transformed 95% confidence intervals. Events are defined as relapse/progression, second malignant neoplasm, or death. |
At 1 and 2 years
|
|
Progression free survival rate
Tidsramme: Up to 2 years
|
Will be estimated by the Kaplan-Meier method beginning at study enrollment. Will be evaluated by two years after enrollment of the last patient on the trial and estimates at specific timepoints will be presented along with log-log transformed 95% confidence intervals. |
Up to 2 years
|
|
Duration of response rate
Tidsramme: After 12 months of therapy. From the scan confirming the complete response or partial response (whichever is recorded first), until the first occurrence of recurrent or progressive disease or death (event) or last known status on trial
|
Response is based on modified Response Evaluation Criteria for Solid Tumors (RECIST)/Positron Emission Tomography Response Criteria in Solid Tumors (PERCIST) consistent with other recent pediatric Langerhans Cell Histiocytosis trials (NCT02670707 and NCT04079179) and adult histiocytosis trials with MAPK inhibitors. Comparison of response assessed via RECIST versus (vs.) PERCIST will be analyzed by displaying two-way tables of the responses with no formal statistical testing. |
After 12 months of therapy. From the scan confirming the complete response or partial response (whichever is recorded first), until the first occurrence of recurrent or progressive disease or death (event) or last known status on trial
|
|
Overall survival rate
Tidsramme: Up to 2 years
|
Will be estimated by the Kaplan-Meier method beginning at study enrollment. Will be evaluated two years after enrollment of the last patient on the trial and estimates at specific timepoints will be presented along with log-log transformed 95% confidence intervals. |
Up to 2 years
|
Andre resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Percent peripheral blood mononuclear cells with mutated allele
Tidsramme: Up to 2 years
|
Will be analyzed descriptively using logistic regression with complete response/progressive response vs. stable disease/progressive disease as the response variable. |
Up to 2 years
|
Samarbejdspartnere og efterforskere
Sponsor
Efterforskere
- Ledende efterforsker: Carl E Allen, Children's Oncology Group
Datoer for undersøgelser
Studer store datoer
Studiestart (Faktiske)
Primær færdiggørelse (Anslået)
Studieafslutning (Anslået)
Datoer for studieregistrering
Først indsendt
Først indsendt, der opfyldte QC-kriterier
Først opslået (Faktiske)
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
Sidst verificeret
Mere information
Begreber relateret til denne undersøgelse
Yderligere relevante MeSH-vilkår
- Luftvejssygdomme
- Lungesygdomme
- Lymfesygdomme
- Lungesygdomme, interstitielle
- Histiocytose
- Hemiske og lymfatiske sygdomme
- Histiocytose, Langerhans-celle
- Undersøgelsesteknikker
- Terapeutik
- Kliniske laboratorieteknikker
- Diagnostiske teknikker og procedurer
- Diagnose
- Punkteringer
- Kirurgiske procedurer, operative
- Cytologiske teknikker
- Cytodiagnose
- Diagnostiske teknikker, kirurgisk
- Diagnostiske teknikker, neurologisk
- Biopsi
- Håndtering af eksemplar
- Spinal punktering
- Tovorafenib
Andre undersøgelses-id-numre
- NCI-2022-06282
- U10CA180886 (U.S. NIH-bevilling/kontrakt)
- ANHL2121 (Anden identifikator: CTEP)
- NCI-2022-06282 (Registry Identifier: CTRP (Clinical Trial Reporting Program))
- ANHL2121 (Anden identifikator: Children's Oncology Group)
Plan for individuelle deltagerdata (IPD)
Planlægger du at dele individuelle deltagerdata (IPD)?
IPD-planbeskrivelse
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.
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