DrugEvidence

Diseases / Medulloblastoma

Medulloblastoma

Disease

Medulloblastoma is a malignant embryonal tumor of the cerebellum and the most common malignant brain tumor in children. It can spread through cerebrospinal pathways and requires multimodal treatment.

Trials
311
Recruiting
36
Completed
167
With results
64

Last revised

Pipeline

Phase by status

PhaseRecruitingNot yetBy invitationActiveCompletedTerminatedWithdrawnSuspendedUnknownAvailableNo longer availableTotal
Early Phase 1 21141 9
Phase 1 139731275 119
Phase 1/2 41211711 27
Phase 2 611644526 80
Phase 2/3 11 2
Phase 3 2182 13
Phase 4 11 2
Not applicable 3210232 22
Unknown 55111621411 37

Trials started per year

2021
3
2022
11
2023
6
2024
10
2025
10
2026
14

Conditions studied, by phase

ConditionEarly Phase 1Phase 1Phase 1/2Phase 2Phase 2/3Phase 3Phase 4Not applicableUnknownTotal
Medulloblastoma 9119278021322237 311
Astrocytoma 2697173710 115
Central Nervous System Neoplasms 15083111713 112
Ependymoma 24093312916 112
Glioma 24312262714 106
Brain Neoplasms 34062123711 93
Oligodendroglioma 51612379 88
Neuroblastoma 1291130346 84
Familial ependymoma 144416135 74
Glioblastoma 135811366 70

Most active sponsors

#SponsorTrialsPhase 3
1National Cancer Institute (NCI)530
2Children's Oncology Group185
3Pediatric Brain Tumor Consortium160
4St. Jude Children's Research Hospital110
5Giselle Sholler70
6Novartis Pharmaceuticals70
7Memorial Sloan Kettering Cancer Center60
8National Institutes of Health Clinical Center (CC)60
9City of Hope Medical Center50
10Duke University50

Most studied interventions

#InterventionTrials
1Cyclophosphamide35
2Temozolomide33
3Etoposide26
4Filgrastim26
5Carboplatin25
6Cisplatin22
7Vincristine22
8Radiotherapy20
9Magnetic Resonance Spectroscopy18
10Thiotepa14

Evidence

Results reported
38.3%64 of 167 completed
Published
22.2%37 of 167 completed
Primary endpoint met
57.1%4 of 7 analyses
Median months to report
12

Documents disclosed

Protocol available
44
Statistical analysis plan
43
PubMed citations
47

Trial profile

Median enrollment
33
Median duration
68.9months
Countries
34
Study sites
1504

Who is enrolled

Adults
292
Children
257
Older adults
92
Healthy volunteers
10

Median enrollment by phase

Early Phase 1
14
Phase 1
26
Phase 1/2
30
Phase 2
42
Phase 2/3
267
Phase 3
130
Phase 4
225
Not applicable
33
Unknown
145

Where trials run

United States
243
Canada
42
Australia
28
Puerto Rico
24
France
19
Germany
15
Italy
15
Switzerland
15
United Kingdom
15
China
14

Busiest cities

#CityCountryTrials
1HoustonUnited States82
2New YorkUnited States70
3Washington D.C.United States69
4ChicagoUnited States66
5MemphisUnited States61
6PhiladelphiaUnited States60
7PittsburghUnited States57
8CincinnatiUnited States54
9Los AngelesUnited States54
10BostonUnited States53

Terminations

Terminated
31
Withdrawn
13
Suspended
1
Stopped for recruitment
4.8%15 of 311 trials

Stated reasons

Other / unclear
19
Recruitment
15
Funding
3
Regulatory
3
Business decision
2
Safety
2
Lack of efficacy
1

Reasons are grouped from the sponsor's own wording with a rule-based classifier; the original text is shown below.

Recently stopped

TrialStatusStated reasonStopped
NCT06639607 Suspended Suspended Drugs/Equipment Unavailable 2051-06-30
NCT07660458 Withdrawn Withdrawn Study withdrawn prior to enrollment due to administrative review 2027-07-30
NCT06466798 Withdrawn Withdrawn Inability to find participants meeting eligibility critieria 2027-07-01
NCT05934630 Terminated Terminated The NCI will not extend the PBTC grant beyond March 31, 2026. 2026-04-17
NCT00897286 Terminated Terminated New Protocol that replaces the current study 2025-09-25
NCT04402073 Terminated Terminated Poor accrual 2025-09-10
NCT03904862 Terminated Terminated The decision to permanently close PBTC-053 was made following communication from the NCI that the PBTC grant will not be extended beyond March 31, 2026. 2025-08-11
NCT03173950 Terminated Terminated Support was withdrawn from study resulting in early termination of study. Investigator had to close study due to no drug supply. 2025-06-23
NCT05429502 Terminated Terminated Study halted early due to high toxicity risk (from BHLRM model) in combo treatment and inability to identify recommended Phase 2 dose in initial phase (Phase 1-Part A), making continuation to subsequent phases (Phase 1-Part B and Phase 2) unfeasible. 2025-02-26
NCT04521946 Withdrawn Withdrawn No participants enrolled. 2022-12-20

Trial records