Promising new drug targets rare childhood brain cancer in global trial

NCT ID NCT05580562

Recruiting now Disease control Sponsor: Jazz Pharmaceuticals Source: ClinicalTrials.gov ↗

First seen Apr 28, 2026 · Last updated May 13, 2026 · Updated 4 times

Summary

This study tests whether the drug ONC201 can help people with a rare and aggressive brain tumor called H3 K27M-mutant glioma live longer. Participants must have completed standard radiation therapy. The trial is double-blind, meaning neither patients nor doctors know who gets the drug or a placebo. About 510 people worldwide will take part.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

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Study contacts

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Aalborg Universitetshospital
RECRUITING

Aalborg, North Denmark, 9000, Denmark

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Addenbrooke's Hospital
RECRUITING

Cambridge, Cambridgeshire, CB2 0QQ, United Kingdom

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Albany Medical Center
COMPLETED

Albany, New York, 12208, United States
Asan Medical Center
RECRUITING

Seoul, Seoul Teugbyeolsi, 5505, South Korea

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Azienda Ospedaliera Città della Salute e della Scienza di Torino
RECRUITING

Turin, Piedmont, 10126, Italy

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BC Cancer - The Vancouver Center
RECRUITING

Vancouver, British Columbia, V5Z 4E6, Canada

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Banner MD Anderson Cancer Center
COMPLETED

Phoenix, Arizona, 85006, United States
Barrow Neurological Institute
ACTIVE_NOT_RECRUITING

Phoenix, Arizona, 85013, United States
Beatson West of Scotland Cancer Centre
RECRUITING

Glasgow, Lanarkshire, G12 0YN, United Kingdom

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Benefis Hospital Sletten Cancer Institute
WITHDRAWN

Great Falls, Montana, 59405, United States
CHA Bundang Medical Center
RECRUITING

Bundang-Gu, Seongnam-Si, Gyeonggido, 13496, South Korea

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Centre Hospitalier Universitaire Vaudois
RECRUITING

Lausanne, Canton of Vaud, 1011, Switzerland

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Children's & Women's Health Care of BC
RECRUITING

Vancouver, British Columbia, V6H 0B3, Canada

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Children's Healthcare of Atlanta
COMPLETED

Atlanta, Georgia, 30342, United States
Children's Hospital at Montefiore Medical Center
COMPLETED

New York, New York, 10029, United States
Children's Hospital of Orange County
COMPLETED

Orange, California, 92868, United States
Children's Hospital of The King's Daughter
COMPLETED

Norfolk, Virginia, 23507, United States
Childrens Hospital of Eastern Ontario
RECRUITING

Ottawa, Ontario, K1H 8L1, Canada

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Churchill Hospital
RECRUITING

Oxford, Ox3 7LE, United Kingdom

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Cincinnati Children's Hospital Medical Center
ACTIVE_NOT_RECRUITING

Cincinnati, Ohio, 45229, United States
Clatterbridge Cancer Centre - Liverpool
RECRUITING

Liverpool, Lancashire, L7 8YA, United Kingdom

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Cleveland Clinic
ACTIVE_NOT_RECRUITING

Cleveland, Ohio, 44195, United States
Cleveland Clinic Florida
COMPLETED

Weston, Florida, 33331, United States
Clinica Universidad Navarra
RECRUITING

Pamplona, Navarre, 31008, Spain

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Columbia University Medical Center
ACTIVE_NOT_RECRUITING

New York, New York, 10032, United States
Complejo Asistencial Universitario de Salamanca - H. Clinico
RECRUITING

Salamanca, 37007, Spain

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Copenhagen University Hospital
RECRUITING

Copenhagen, Capital, 2100, Denmark

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Dana Farber Cancer Institute
ACTIVE_NOT_RECRUITING

Boston, Massachusetts, 02215, United States
Dell Children's Medical Center of Central Texas
COMPLETED

Austin, Texas, 78723, United States
Duke Cancer Institute
ACTIVE_NOT_RECRUITING

Durham, North Carolina, 27710, United States
Erasmus MC
RECRUITING

Rotterdam, South Holland, 3015 GD, Netherlands

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F345 H.C. Andersen's Children's Hospital
RECRUITING

Odense, Region Syddanmark, 5000, Denmark

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FLENI Neurologia
RECRUITING

Buenos Aires, C1426ABP, Argentina

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Feinberg School of Medicine Northwestern University
ACTIVE_NOT_RECRUITING

Chicago, Illinois, 60611, United States
Fondazione IRCCS Di Rilievo Nazionale Istituto Nazionale Neurologico Carlo Besta
RECRUITING

Milan, Lombardy, 20133, Italy

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Freeman Hospital
RECRUITING

Newcastle upon Tyne, Tyne and Wear, NE7 7DN, United Kingdom

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Gangnam Severance Hospital, Yonsei University Health System
RECRUITING

Gangnam, Seoul Teugbyeolsi, 06273, South Korea

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Guy's Hospital
RECRUITING

London, SE19RT, United Kingdom

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Hadassah Ein Kerem Medical Center
RECRUITING

Jerusalem, 91120, Israel

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Hcor Research Institute
RECRUITING

São Paulo, Brazil

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Hokkaido University Hospital
RECRUITING

Sapporo, 060-8648, Japan

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Hopital Notre Dame, Lachapelle
RECRUITING

Montreal, Quebec, H2X 3E4, Canada

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Hospital Clinic de Barcelona
RECRUITING

Barcelona, 08036, Spain

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Hospital Infantil Universitario Niño Jesus
RECRUITING

Madrid, 28009, Spain

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Hospital Sant Joan de Deu
RECRUITING

Esplugues de Llobregat, Barcelona, 8950, Spain

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Hospital Universitari i Politecnic La Fe de Valencia - Adults
RECRUITING

Valencia, 46026, Spain

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Hospital Universitari i Politecnic La Fe de Valencia - Pediatrics
RECRUITING

Valencia, 46026, Spain

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Hospital Universitario 12 de Octubre
RECRUITING

Madrid, 28041, Spain

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Hospital Universitario HM Sanchinarro
RECRUITING

Madrid, 28050, Spain

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Hospital Universitario Vall d'Hebron
RECRUITING

Barcelona, 8035, Spain

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Hospital do GRAACC
RECRUITING

São Paulo, 04023-062, Brazil

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Indiana University School of Medicine - Indianapolis
ACTIVE_NOT_RECRUITING

Indianapolis, Indiana, 46202, United States
Inova Schar Cancer Institute
ACTIVE_NOT_RECRUITING

Fairfax, Virginia, 22031, United States
Instituto Do Cancer Do Estado De São Paulo
RECRUITING

São Paulo, Brazil

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Istituto Clinico Humanitas
RECRUITING

Rozzano, Lombardy, 20089, Italy

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Istituto Nazionale Tumori Regina Elena
WITHDRAWN

Rome, Lazio, 00144, Italy
Istituto Oncologico Veneto
RECRUITING

Padova, Veneto, 35128, Italy

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Jersey Shore University Medical Center
ACTIVE_NOT_RECRUITING

Neptune City, New Jersey, 07753, United States
KK Women's and Children's Hospital
RECRUITING

Singapore, 229899, Singapore

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Kaiser Permanente Los Angeles Medical Center
COMPLETED

Los Angeles, California, 90027, United States
Kapi'olani Medical Center for Women and Children
COMPLETED

Honolulu, Hawaii, 96826, United States
Klinikum Mannheim Universitätsklinikum gGmbH
RECRUITING

Manheim, 68167, Germany

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Klinikum der Johann-Wolfgang Goethe-Universitat
RECRUITING

Frankfurt am Main, Hesse, 60528, Germany

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Kyoto University Hospital
RECRUITING

Kyoto, 606-8507, Japan

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Laura & Isaac Perlmutter Cancer Center - NYU ACC
ACTIVE_NOT_RECRUITING

New York, New York, 10032-3726, United States
Lenox Hill Hospital
COMPLETED

New York, New York, 10075, United States
Levine Cancer Institute/ Atrium Health
ACTIVE_NOT_RECRUITING

Charlotte, North Carolina, 28204, United States
London Health Sciences Centre
RECRUITING

London, Ontario, N6A 5W9, Canada

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Massachusetts General Hospital Cancer Center
ACTIVE_NOT_RECRUITING

Boston, Massachusetts, 02114, United States
Mayo Clinic - Cancer Center - Rochester
ACTIVE_NOT_RECRUITING

Rochester, Minnesota, 55905, United States
Mayo Clinic Arizona
COMPLETED

Phoenix, Arizona, 85054, United States
Mayo Clinic Jacksonville
ACTIVE_NOT_RECRUITING

Jacksonville, Florida, 32224, United States
MedStar Georgetown University Hospital
COMPLETED

Washington D.C., District of Columbia, 20007, United States
Medical University of Vienna - Adults
RECRUITING

Vienna, State of Vienna, 01090, Austria

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Medical University of Vienna - Pediatrics
RECRUITING

Vienna, State of Vienna, Austria

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Miami Cancer Institute
ACTIVE_NOT_RECRUITING

Miami, Florida, 33176, United States
Moffitt Cancer Center
ACTIVE_NOT_RECRUITING

Tampa, Florida, 33612, United States
Montefiore Medical Park
ACTIVE_NOT_RECRUITING

New York, New York, 10029, United States
National Cancer Center
RECRUITING

Ilsandong-Gu, Goyang-Si, Gyeonggido, 10408, South Korea

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National Cancer Center Hospital
RECRUITING

Chūō, 104-0045, Japan

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National Cancer Centre
RECRUITING

Singapore, 308433, Singapore

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National Hospital Organization Kyushu Cancer Center
RECRUITING

Fukuoka, 812-8582, Japan

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National Neuroscience Institute
RECRUITING

Singapore, 308433, Singapore

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Neuro-Oncology Associates
ACTIVE_NOT_RECRUITING

Dallas, Texas, 75246, United States
Norton Healthcare
ACTIVE_NOT_RECRUITING

Louisville, Kentucky, 40241, United States
Ochsner Medical Center - New Orleans
COMPLETED

New Orleans, Louisiana, 70121, United States
Odense Universitetshospital
RECRUITING

Odense, Region Syddanmark, 5000, Denmark

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Ohio State University
ACTIVE_NOT_RECRUITING

Columbus, Ohio, 43210, United States
Olivia Newton-John Cancer Research Institute (ONJCRI)
RECRUITING

Heidelberg, Victoria, 3084, Australia

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Osaka City General Hospital
RECRUITING

Osaka, 534-0021, Japan

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Ospedale Bellaria
RECRUITING

Bologna, Emilia-Romagna, 40139, Italy

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Ospedale San Raffaele S.r.l.
RECRUITING

Milan, Lombardy, 20132, Italy

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Overlook Medical Center
ACTIVE_NOT_RECRUITING

Summit, New Jersey, 07901, United States
Perth Children's Hospital
RECRUITING

Nedlands, Western Australia, 6009, Australia

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Phoenix Childrens Hospital
COMPLETED

Phoenix, Arizona, 85016, United States
Princess Margaret Hospital
RECRUITING

Toronto, Ontario, M5G 1X8, Canada

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Providence Health and Services St. Vincent Medical Center
ACTIVE_NOT_RECRUITING

Portland, Oregon, 97239, United States
Providence Saint John's Cancer Institute
ACTIVE_NOT_RECRUITING

Santa Monica, California, 90404, United States
Queen Mary Hospital
RECRUITING

Hong Kong, 999077, Hong Kong

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Rabin Medical Center
RECRUITING

Petah Tikva, 49372, Israel

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Rambam Medical Center
RECRUITING

Haifa, 31096, Israel

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Royal Brisbane and Women's Hospital
RECRUITING

Herston, Queensland, Australia

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Royal Hobart Hospital
RECRUITING

Hobart, Tasmania, 7000, Australia

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Royal Hospital for Children (Glasgow)
RECRUITING

Glasgow, Lanarkshire, G51 4TF, United Kingdom

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Royal North Shore Hospital
RECRUITING

Sydney, New South Wales, 2065, Australia

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Royal Victoria Infirmary
RECRUITING

Newcastle upon Tyne, Tyne and Wear, NE1 4LP, United Kingdom

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Samsung Medical Center
RECRUITING

Gangnam-Gu, Seoul Teugbyeolsi, 06351, South Korea

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Schneider Children's Medical Center of Israel
RECRUITING

Petah Tikvah, 4920235, Israel

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Seoul National University Bundang Hospital
RECRUITING

Bundang-gu, Seongnam-si, Gyeonggido, 13620, South Korea

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Seoul National University Hospital
RECRUITING

Bundang-gu, Seongnam-si, Gyeonggido, 13620, South Korea

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Seoul National University Hospital
RECRUITING

Jongno-Gu, Seoul Teugbyeolsi, 03080, South Korea

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Severance Hospital Yonsei University Health System
RECRUITING

Seoul, 03722, South Korea

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Sheba Medical Center
RECRUITING

Ramat Gan, Tel Aviv, 5262100, Israel

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Soroka University Medical Centre
RECRUITING

Beersheba, 50937, Israel

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St James University Hospital
RECRUITING

Leeds, West Yorkshire, LS9 7TF, United Kingdom

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St Joseph's Children's Hospital of Tampa
COMPLETED

Tampa, Florida, 33607, United States
Stanford Cancer Center
COMPLETED

Stanford, California, 94350, United States
Sunnybrook Health Sciences Centre
RECRUITING

Toronto, Ontario, M4N 3M5, Canada

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Sydney Children's Hospital
RECRUITING

Randwick, New South Wales, 2031, Australia

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Tel Aviv Sourasky Medical Center
RECRUITING

Tel Aviv, 64239, Israel

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The Catholic University of Korea, Seoul St. Mary's Hospital
RECRUITING

Seoul, 6591, South Korea

Contact

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The Christie NHS Foundation Trust
RECRUITING

Manchester, Lancashire, M20 4BX, United Kingdom

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The Leeds Teaching Hospitals NHS Trust, Leeds General Infimary
RECRUITING

Leeds, LS1 3EX, United Kingdom

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The Royal Marsden in Sutton, Surrey
RECRUITING

Sutton, Surrey, SM2 5PT, United Kingdom

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The University of Tokyo Hospital
RECRUITING

Tokyo, Japan

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Thomas Jefferson University
ACTIVE_NOT_RECRUITING

Philadelphia, Pennsylvania, 19107, United States
Tom Baker Cancer Cetre
RECRUITING

Calgary, Alberta, T2N 2T9, Canada

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UC San Diego Moores Cancer Center
ACTIVE_NOT_RECRUITING

La Jolla, California, 92093, United States
UCLA University of California Los Angeles
COMPLETED

Los Angeles, California, 90095, United States
UCSF Benioff Children's Hospital
ACTIVE_NOT_RECRUITING

San Francisco, California, 94143, United States
UPMC Children's Hospital of Pittsburgh
COMPLETED

Pittsburgh, Pennsylvania, 15232, United States
UPMC Hillman Cancer Center
COMPLETED

Pittsburgh, Pennsylvania, 15232, United States
UT Health Houston Neurosciences - Texas Medical Center
COMPLETED

Houston, Texas, 77030, United States
Uniklinik Köln
RECRUITING

Cologne, North Rhine-Westphalia, 50937, Germany

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Universitair Medisch Centrum Utrecht Cancer Center
RECRUITING

Utrecht, 3584 CX, Netherlands

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University Clinic Heidelberg
RECRUITING

Heidelberg, Baden-Wurttemberg, 69120, Germany

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University Clinic Regensburg
RECRUITING

Regensburg, Bavaria, 93053, Germany

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University Of Wisconsin - Madison
ACTIVE_NOT_RECRUITING

Madison, Wisconsin, 53792, United States
University of California Irvine
ACTIVE_NOT_RECRUITING

Orange, California, 92868, United States
University of California San Francisco
ACTIVE_NOT_RECRUITING

San Francisco, California, 94143, United States
University of Iowa Hospitals & Clinics
ACTIVE_NOT_RECRUITING

Iowa City, Iowa, 52242, United States
University of Maryland School of Medicine
COMPLETED

Baltimore, Maryland, 21201, United States
University of Michigan Hospital
ACTIVE_NOT_RECRUITING

Ann Arbor, Michigan, 48109, United States
University of Minnesota
ACTIVE_NOT_RECRUITING

Minneapolis, Minnesota, 55455, United States
University of Nebraska Medical Center
COMPLETED

Omaha, Nebraska, 68114, United States
University of Oklahoma Peggy and Charles Stephenson Cancer Center
ACTIVE_NOT_RECRUITING

Oklahoma City, Oklahoma, 73104, United States
University of Pennsylvania
ACTIVE_NOT_RECRUITING

Philadelphia, Pennsylvania, 19104, United States
University of Rochester Medical Center
COMPLETED

Rochester, New York, 14642, United States
University of Texas - San Antonio - Health Science Center
ACTIVE_NOT_RECRUITING

San Antonio, Texas, 78229, United States
University of Texas MD Anderson Cancer Center
ACTIVE_NOT_RECRUITING

Houston, Texas, 77030, United States
University of Texas Southwestern Medical Center
ACTIVE_NOT_RECRUITING

Dallas, Texas, 75390, United States
University of Utah - Huntsman Cancer Institute
ACTIVE_NOT_RECRUITING

Salt Lake City, Utah, 84112, United States
University of Washington
ACTIVE_NOT_RECRUITING

Seattle, Washington, 98195, United States
Universitätsklinikum Augsburg
RECRUITING

Augsburg, Bavaria, 86156, Germany

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Universitätsklinikum Bonn
RECRUITING

Bonn, North Rhine-Westphalia, 53127, Germany

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Universitätsklinikum Essen
RECRUITING

Essen, North Rhine-Westphalia, 45147, Germany

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Universitätsklinikum Heidelberg
RECRUITING

Heidelberg, Baden-Wurttemberg, 69120, Germany

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Universitätsklinikum Tübingen
RECRUITING

Tübingen, 72076, Germany

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Universitätsspital Zürich
RECRUITING

Zurich, 8091, Switzerland

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Vivantes Klinikum Neukölln
RECRUITING

Berlin, 12351, Germany

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Washington University School of Medicine/St. Louis Children's Hospital
ACTIVE_NOT_RECRUITING

St Louis, Missouri, 63110, United States
Yale University
ACTIVE_NOT_RECRUITING

New Haven, Connecticut, 06511, United States

Conditions

Explore the condition pages connected to this study.

GLIOMA H3 K27M H3 K27M