Actively Recruiting
ONC201 in H3 K27M-mutant Diffuse Glioma Following Radiotherapy (the ACTION Study)
Led by Jazz Pharmaceuticals · Updated on 2026-04-16
510
Participants Needed
162
Research Sites
279 weeks
Total Duration
On this page
Sponsors
J
Jazz Pharmaceuticals
Lead Sponsor
C
Chimerix, Inc.
Collaborating Sponsor
AI-Summary
What this Trial Is About
This is a randomized, double-blind, placebo-controlled, parallel-group, international, Phase 3 study in patients with newly diagnosed H3 K27M-mutant diffuse glioma to assess whether treatment with dordaviprone (ONC201) following frontline radiotherapy will extend overall survival and progression-free survival in this population. Eligible participants will have histologically diagnosed H3 K27M-mutant diffuse glioma and have completed standard frontline radiotherapy.
CONDITIONS
Official Title
ONC201 in H3 K27M-mutant Diffuse Glioma Following Radiotherapy (the ACTION Study)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Able to understand study procedures and provide written informed consent or assent when applicable
- Body weight of at least 10 kg at randomization
- Histologically confirmed diagnosis of newly diagnosed H3 K27M-mutant diffuse glioma by tumor tissue testing
- At least one high-quality, contrast-enhanced brain MRI obtained before starting radiotherapy
- At least one high-quality, contrast-enhanced brain MRI obtained 2 to 6 weeks after completing frontline radiotherapy (non-enhanced MRI allowed if venous access is not possible)
- Receipt of standard frontline radiotherapy initiated within 12 weeks of diagnosis and completed 2 to 6 weeks before randomization
- Karnofsky or Lansky Performance Status score of 70 or higher at randomization
- Stable or decreasing corticosteroid and anti-seizure medication doses for 7 days before randomization, if applicable
You will not qualify if you...
- Primary spinal tumor
- Diffuse intrinsic pontine glioma (DIPG) with pontine epicenter and diffuse pons involvement
- Evidence of leptomeningeal spread or cerebrospinal fluid dissemination of disease
- Any known concurrent malignancy
- New tumor lesions outside the radiation field
- Prior whole-brain radiotherapy
- Prior proton therapy for glioma
- Prior use of dordaviprone (ONC201), ONC206, systemic bevacizumab since diagnosis, temozolomide within 3 weeks, tumor treating fields, DRD2 antagonists within 2 weeks, investigational therapies within 4 weeks, strong CYP3A4 inhibitors within 3 days, or strong CYP3A4 inducers within 2 weeks
- Laboratory abnormalities within 2 weeks before randomization: absolute neutrophil count below 1.0 x 10^9/L, platelets below 75 x 10^9/L, total bilirubin above 1.5 times upper limit of normal (ULN) except Gilbert's syndrome with normal direct bilirubin, AST or ALT above 2.5 times ULN, creatinine clearance 60 mL/min or less
- QTc interval greater than 480 msec during screening
- Known allergy to any ingredients in the study treatment
- Pregnant, breastfeeding, or planning pregnancy during treatment or within 3 months after last dose; must have negative pregnancy test if of childbearing potential
- Uncontrolled illness or infection requiring systemic therapy or psychiatric/social conditions limiting compliance
- Any other condition that may interfere with safety or study completion as judged by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 162 locations
1
Banner MD Anderson Cancer Center
Phoenix, Arizona, United States, 85006
Completed
2
Barrow Neurological Institute
Phoenix, Arizona, United States, 85013
Active, Not Recruiting
3
Phoenix Childrens Hospital
Phoenix, Arizona, United States, 85016
Completed
4
Mayo Clinic Arizona
Phoenix, Arizona, United States, 85054
Completed
5
UC San Diego Moores Cancer Center
La Jolla, California, United States, 92093
Active, Not Recruiting
6
Kaiser Permanente Los Angeles Medical Center
Los Angeles, California, United States, 90027
Completed
7
UCLA University of California Los Angeles
Los Angeles, California, United States, 90095
Completed
8
Children's Hospital of Orange County
Orange, California, United States, 92868
Completed
9
University of California Irvine
Orange, California, United States, 92868
Active, Not Recruiting
10
UCSF Benioff Children's Hospital
San Francisco, California, United States, 94143
Active, Not Recruiting
11
University of California San Francisco
San Francisco, California, United States, 94143
Active, Not Recruiting
12
Providence Saint John's Cancer Institute
Santa Monica, California, United States, 90404
Active, Not Recruiting
13
Stanford Cancer Center
Stanford, California, United States, 94350
Completed
14
Yale University
New Haven, Connecticut, United States, 06511
Active, Not Recruiting
15
MedStar Georgetown University Hospital
Washington D.C., District of Columbia, United States, 20007
Completed
16
Mayo Clinic Jacksonville
Jacksonville, Florida, United States, 32224
Active, Not Recruiting
17
Miami Cancer Institute
Miami, Florida, United States, 33176
Active, Not Recruiting
18
St Joseph's Children's Hospital of Tampa
Tampa, Florida, United States, 33607
Completed
19
Moffitt Cancer Center
Tampa, Florida, United States, 33612
Active, Not Recruiting
20
Cleveland Clinic Florida
Weston, Florida, United States, 33331
Completed
21
Children's Healthcare of Atlanta
Atlanta, Georgia, United States, 30342
Completed
22
Kapi'olani Medical Center for Women and Children
Honolulu, Hawaii, United States, 96826
Completed
23
Feinberg School of Medicine Northwestern University
Chicago, Illinois, United States, 60611
Active, Not Recruiting
24
Indiana University School of Medicine - Indianapolis
Indianapolis, Indiana, United States, 46202
Active, Not Recruiting
25
University of Iowa Hospitals & Clinics
Iowa City, Iowa, United States, 52242
Active, Not Recruiting
26
Norton Healthcare
Louisville, Kentucky, United States, 40241
Active, Not Recruiting
27
Ochsner Medical Center - New Orleans
New Orleans, Louisiana, United States, 70121
Completed
28
University of Maryland School of Medicine
Baltimore, Maryland, United States, 21201
Completed
29
Massachusetts General Hospital Cancer Center
Boston, Massachusetts, United States, 02114
Active, Not Recruiting
30
Dana Farber Cancer Institute
Boston, Massachusetts, United States, 02215
Active, Not Recruiting
31
University of Michigan Hospital
Ann Arbor, Michigan, United States, 48109
Active, Not Recruiting
32
University of Minnesota
Minneapolis, Minnesota, United States, 55455
Active, Not Recruiting
33
Mayo Clinic - Cancer Center - Rochester
Rochester, Minnesota, United States, 55905
Active, Not Recruiting
34
Washington University School of Medicine/St. Louis Children's Hospital
St Louis, Missouri, United States, 63110
Active, Not Recruiting
35
Benefis Hospital Sletten Cancer Institute
Great Falls, Montana, United States, 59405
Withdrawn
36
University of Nebraska Medical Center
Omaha, Nebraska, United States, 68114
Completed
37
Jersey Shore University Medical Center
Neptune City, New Jersey, United States, 07753
Active, Not Recruiting
38
Overlook Medical Center
Summit, New Jersey, United States, 07901
Active, Not Recruiting
39
Albany Medical Center
Albany, New York, United States, 12208
Completed
40
Children's Hospital at Montefiore Medical Center
New York, New York, United States, 10029
Completed
41
Montefiore Medical Park
New York, New York, United States, 10029
Active, Not Recruiting
42
Laura & Isaac Perlmutter Cancer Center - NYU ACC
New York, New York, United States, 10032-3726
Active, Not Recruiting
43
Columbia University Medical Center
New York, New York, United States, 10032
Active, Not Recruiting
44
Lenox Hill Hospital
New York, New York, United States, 10075
Completed
45
University of Rochester Medical Center
Rochester, New York, United States, 14642
Completed
46
Levine Cancer Institute/ Atrium Health
Charlotte, North Carolina, United States, 28204
Active, Not Recruiting
47
Duke Cancer Institute
Durham, North Carolina, United States, 27710
Active, Not Recruiting
48
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Active, Not Recruiting
49
Cleveland Clinic
Cleveland, Ohio, United States, 44195
Active, Not Recruiting
50
Ohio State University
Columbus, Ohio, United States, 43210
Active, Not Recruiting
51
University of Oklahoma Peggy and Charles Stephenson Cancer Center
Oklahoma City, Oklahoma, United States, 73104
Active, Not Recruiting
52
Providence Health and Services St. Vincent Medical Center
Portland, Oregon, United States, 97239
Active, Not Recruiting
53
University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
Active, Not Recruiting
54
Thomas Jefferson University
Philadelphia, Pennsylvania, United States, 19107
Active, Not Recruiting
55
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15232
Completed
56
UPMC Hillman Cancer Center
Pittsburgh, Pennsylvania, United States, 15232
Completed
57
Dell Children's Medical Center of Central Texas
Austin, Texas, United States, 78723
Completed
58
Neuro-Oncology Associates
Dallas, Texas, United States, 75246
Active, Not Recruiting
59
University of Texas Southwestern Medical Center
Dallas, Texas, United States, 75390
Active, Not Recruiting
60
University of Texas MD Anderson Cancer Center
Houston, Texas, United States, 77030
Active, Not Recruiting
61
UT Health Houston Neurosciences - Texas Medical Center
Houston, Texas, United States, 77030
Completed
62
University of Texas - San Antonio - Health Science Center
San Antonio, Texas, United States, 78229
Active, Not Recruiting
63
University of Utah - Huntsman Cancer Institute
Salt Lake City, Utah, United States, 84112
Active, Not Recruiting
64
Inova Schar Cancer Institute
Fairfax, Virginia, United States, 22031
Active, Not Recruiting
65
Children's Hospital of The King's Daughter
Norfolk, Virginia, United States, 23507
Completed
66
University of Washington
Seattle, Washington, United States, 98195
Active, Not Recruiting
67
University Of Wisconsin - Madison
Madison, Wisconsin, United States, 53792
Active, Not Recruiting
68
FLENI Neurologia
Buenos Aires, Argentina, C1426ABP
Actively Recruiting
69
Sydney Children's Hospital
Randwick, New South Wales, Australia, 2031
Actively Recruiting
70
Royal North Shore Hospital
Sydney, New South Wales, Australia, 2065
Actively Recruiting
71
Royal Brisbane and Women's Hospital
Herston, Queensland, Australia
Actively Recruiting
72
Royal Hobart Hospital
Hobart, Tasmania, Australia, 7000
Actively Recruiting
73
Olivia Newton-John Cancer Research Institute (ONJCRI)
Heidelberg, Victoria, Australia, 3084
Actively Recruiting
74
Perth Children's Hospital
Nedlands, Western Australia, Australia, 6009
Actively Recruiting
75
Medical University of Vienna - Adults
Vienna, State of Vienna, Austria, 01090
Actively Recruiting
76
Medical University of Vienna - Pediatrics
Vienna, State of Vienna, Austria
Actively Recruiting
77
Hospital do GRAACC
São Paulo, Brazil, 04023-062
Actively Recruiting
78
Hcor Research Institute
São Paulo, Brazil
Actively Recruiting
79
Instituto Do Cancer Do Estado De São Paulo
São Paulo, Brazil
Actively Recruiting
80
Tom Baker Cancer Cetre
Calgary, Alberta, Canada, T2N 2T9
Actively Recruiting
81
BC Cancer - The Vancouver Center
Vancouver, British Columbia, Canada, V5Z 4E6
Actively Recruiting
82
Children's & Women's Health Care of BC
Vancouver, British Columbia, Canada, V6H 0B3
Actively Recruiting
83
London Health Sciences Centre
London, Ontario, Canada, N6A 5W9
Actively Recruiting
84
Childrens Hospital of Eastern Ontario
Ottawa, Ontario, Canada, K1H 8L1
Actively Recruiting
85
Sunnybrook Health Sciences Centre
Toronto, Ontario, Canada, M4N 3M5
Actively Recruiting
86
Princess Margaret Hospital
Toronto, Ontario, Canada, M5G 1X8
Actively Recruiting
87
Hopital Notre Dame, Lachapelle
Montreal, Quebec, Canada, H2X 3E4
Actively Recruiting
88
Copenhagen University Hospital
Copenhagen, Capital, Denmark, 2100
Actively Recruiting
89
Aalborg Universitetshospital
Aalborg, North Denmark, Denmark, 9000
Actively Recruiting
90
F345 H.C. Andersen's Children's Hospital
Odense, Region Syddanmark, Denmark, 5000
Actively Recruiting
91
Odense Universitetshospital
Odense, Region Syddanmark, Denmark, 5000
Actively Recruiting
92
University Clinic Heidelberg
Heidelberg, Baden-Wurttemberg, Germany, 69120
Actively Recruiting
93
Universitätsklinikum Heidelberg
Heidelberg, Baden-Wurttemberg, Germany, 69120
Actively Recruiting
94
Universitätsklinikum Augsburg
Augsburg, Bavaria, Germany, 86156
Actively Recruiting
95
University Clinic Regensburg
Regensburg, Bavaria, Germany, 93053
Actively Recruiting
96
Klinikum der Johann-Wolfgang Goethe-Universitat
Frankfurt am Main, Hesse, Germany, 60528
Actively Recruiting
97
Universitätsklinikum Bonn
Bonn, North Rhine-Westphalia, Germany, 53127
Actively Recruiting
98
Uniklinik Köln
Cologne, North Rhine-Westphalia, Germany, 50937
Actively Recruiting
99
Universitätsklinikum Essen
Essen, North Rhine-Westphalia, Germany, 45147
Actively Recruiting
100
Vivantes Klinikum Neukölln
Berlin, Germany, 12351
Actively Recruiting
101
Klinikum Mannheim Universitätsklinikum gGmbH
Manheim, Germany, 68167
Actively Recruiting
102
Universitätsklinikum Tübingen
Tübingen, Germany, 72076
Actively Recruiting
103
Queen Mary Hospital
Hong Kong, Hong Kong, 999077
Actively Recruiting
104
Sheba Medical Center
Ramat Gan, Tel Aviv, Israel, 5262100
Actively Recruiting
105
Soroka University Medical Centre
Beersheba, Israel, 50937
Actively Recruiting
106
Rambam Medical Center
Haifa, Israel, 31096
Actively Recruiting
107
Hadassah Ein Kerem Medical Center
Jerusalem, Israel, 91120
Actively Recruiting
108
Rabin Medical Center
Petah Tikva, Israel, 49372
Actively Recruiting
109
Schneider Children's Medical Center of Israel
Petah Tikvah, Israel, 4920235
Actively Recruiting
110
Tel Aviv Sourasky Medical Center
Tel Aviv, Israel, 64239
Actively Recruiting
111
Ospedale Bellaria
Bologna, Emilia-Romagna, Italy, 40139
Actively Recruiting
112
Istituto Nazionale Tumori Regina Elena
Rome, Lazio, Italy, 00144
Withdrawn
113
Ospedale San Raffaele S.r.l.
Milan, Lombardy, Italy, 20132
Actively Recruiting
114
Fondazione IRCCS Di Rilievo Nazionale Istituto Nazionale Neurologico Carlo Besta
Milan, Lombardy, Italy, 20133
Actively Recruiting
115
Istituto Clinico Humanitas
Rozzano, Lombardy, Italy, 20089
Actively Recruiting
116
Azienda Ospedaliera Città della Salute e della Scienza di Torino
Turin, Piedmont, Italy, 10126
Actively Recruiting
117
Istituto Oncologico Veneto
Padova, Veneto, Italy, 35128
Actively Recruiting
118
National Cancer Center Hospital
Chūō, Japan, 104-0045
Actively Recruiting
119
National Hospital Organization Kyushu Cancer Center
Fukuoka, Japan, 812-8582
Actively Recruiting
120
Kyoto University Hospital
Kyoto, Japan, 606-8507
Actively Recruiting
121
Osaka City General Hospital
Osaka, Japan, 534-0021
Actively Recruiting
122
Hokkaido University Hospital
Sapporo, Japan, 060-8648
Actively Recruiting
123
The University of Tokyo Hospital
Tokyo, Japan
Actively Recruiting
124
Erasmus MC
Rotterdam, South Holland, Netherlands, 3015 GD
Actively Recruiting
125
Universitair Medisch Centrum Utrecht Cancer Center
Utrecht, Netherlands, 3584 CX
Actively Recruiting
126
KK Women's and Children's Hospital
Singapore, Singapore, 229899
Actively Recruiting
127
National Cancer Centre
Singapore, Singapore, 308433
Actively Recruiting
128
National Neuroscience Institute
Singapore, Singapore, 308433
Actively Recruiting
129
CHA Bundang Medical Center
Bundang-Gu, Seongnam-Si, Gyeonggido, South Korea, 13496
Actively Recruiting
130
Seoul National University Bundang Hospital
Bundang-gu, Seongnam-si, Gyeonggido, South Korea, 13620
Actively Recruiting
131
Seoul National University Hospital
Bundang-gu, Seongnam-si, Gyeonggido, South Korea, 13620
Actively Recruiting
132
National Cancer Center
Ilsandong-Gu, Goyang-Si, Gyeonggido, South Korea, 10408
Actively Recruiting
133
Gangnam Severance Hospital, Yonsei University Health System
Gangnam, Seoul Teugbyeolsi, South Korea, 06273
Actively Recruiting
134
Samsung Medical Center
Gangnam-Gu, Seoul Teugbyeolsi, South Korea, 06351
Actively Recruiting
135
Seoul National University Hospital
Jongno-Gu, Seoul Teugbyeolsi, South Korea, 03080
Actively Recruiting
136
Asan Medical Center
Seoul, Seoul Teugbyeolsi, South Korea, 5505
Actively Recruiting
137
Severance Hospital Yonsei University Health System
Seoul, South Korea, 03722
Actively Recruiting
138
The Catholic University of Korea, Seoul St. Mary's Hospital
Seoul, South Korea, 6591
Actively Recruiting
139
Hospital Sant Joan de Deu
Esplugues de Llobregat, Barcelona, Spain, 8950
Actively Recruiting
140
Clinica Universidad Navarra
Pamplona, Navarre, Spain, 31008
Actively Recruiting
141
Hospital Clinic de Barcelona
Barcelona, Spain, 08036
Actively Recruiting
142
Hospital Universitario Vall d'Hebron
Barcelona, Spain, 8035
Actively Recruiting
143
Hospital Infantil Universitario Niño Jesus
Madrid, Spain, 28009
Actively Recruiting
144
Hospital Universitario 12 de Octubre
Madrid, Spain, 28041
Actively Recruiting
145
Hospital Universitario HM Sanchinarro
Madrid, Spain, 28050
Actively Recruiting
146
Complejo Asistencial Universitario de Salamanca - H. Clinico
Salamanca, Spain, 37007
Actively Recruiting
147
Hospital Universitari i Politecnic La Fe de Valencia - Adults
Valencia, Spain, 46026
Actively Recruiting
148
Hospital Universitari i Politecnic La Fe de Valencia - Pediatrics
Valencia, Spain, 46026
Actively Recruiting
149
Centre Hospitalier Universitaire Vaudois
Lausanne, Canton of Vaud, Switzerland, 1011
Actively Recruiting
150
Universitätsspital Zürich
Zurich, Switzerland, 8091
Actively Recruiting
151
Addenbrooke's Hospital
Cambridge, Cambridgeshire, United Kingdom, CB2 0QQ
Actively Recruiting
152
Beatson West of Scotland Cancer Centre
Glasgow, Lanarkshire, United Kingdom, G12 0YN
Actively Recruiting
153
Royal Hospital for Children (Glasgow)
Glasgow, Lanarkshire, United Kingdom, G51 4TF
Actively Recruiting
154
Clatterbridge Cancer Centre - Liverpool
Liverpool, Lancashire, United Kingdom, L7 8YA
Actively Recruiting
155
The Christie NHS Foundation Trust
Manchester, Lancashire, United Kingdom, M20 4BX
Actively Recruiting
156
The Royal Marsden in Sutton, Surrey
Sutton, Surrey, United Kingdom, SM2 5PT
Actively Recruiting
157
Royal Victoria Infirmary
Newcastle upon Tyne, Tyne and Wear, United Kingdom, NE1 4LP
Actively Recruiting
158
Freeman Hospital
Newcastle upon Tyne, Tyne and Wear, United Kingdom, NE7 7DN
Actively Recruiting
159
St James University Hospital
Leeds, West Yorkshire, United Kingdom, LS9 7TF
Actively Recruiting
160
The Leeds Teaching Hospitals NHS Trust, Leeds General Infimary
Leeds, United Kingdom, LS1 3EX
Actively Recruiting
161
Guy's Hospital
London, United Kingdom, SE19RT
Actively Recruiting
162
Churchill Hospital
Oxford, United Kingdom, Ox3 7LE
Actively Recruiting
Research Team
C
Clinical Trial Disclosure & Transparency
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
TRIPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
3
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