DrugEvidence

Diseases / Neuroblastoma

Neuroblastoma

Disease

Neuroblastoma is a malignant embryonal tumor of the sympathetic nervous system that usually arises in the adrenal medulla or paraspinal ganglia and occurs predominantly in young children.

Trials
650
Recruiting
90
Completed
330
With results
121

Last revised

Pipeline

Phase by status

PhaseRecruitingNot yetBy invitationActiveCompletedTerminatedWithdrawnSuspendedUnknownApproved for marketingAvailableNo longer availableTotal
Early Phase 1 514113 15
Phase 1 19211611723916 194
Phase 1/2 16315251014 65
Phase 2 1931995151016 177
Phase 2/3 22 4
Phase 3 612436 40
Phase 4 1111 4
Not applicable 613282511 56
Unknown 1721539819166 95

Trials started per year

2021
7
2022
15
2023
18
2024
26
2025
22
2026
17

Conditions studied, by phase

ConditionEarly Phase 1Phase 1Phase 1/2Phase 2Phase 2/3Phase 3Phase 4Not applicableUnknownTotal
Neuroblastoma 151946517744045695 650
Sarcoma 411240131310 120
Osteosarcoma 1511535356 116
Wilms Tumor 41832811012 112
Rhabdomyosarcoma 38143225 91
Medulloblastoma 1291130346 84
Neoplasms 131611271115 84
Sarcoma, Ewing 331324126 79
Recurrence 1271212788 75
Lymphoma, Non-Hodgkin 96277148 71

Most active sponsors

#SponsorTrialsPhase 3
1National Cancer Institute (NCI)682
2Children's Oncology Group579
3Memorial Sloan Kettering Cancer Center501
4New Approaches to Neuroblastoma Therapy Consortium240
5St. Jude Children's Research Hospital230
6Giselle Sholler180
7Baylor College of Medicine140
8Fred Hutchinson Cancer Center143
9Children's Cancer and Leukaemia Group134
10Dana-Farber Cancer Institute100

Most studied interventions

#InterventionTrials
1Cyclophosphamide101
2Etoposide60
3Carboplatin55
4Vincristine51
5Filgrastim49
6Doxorubicin44
7Isotretinoin44
8Melphalan44
9Temozolomide44
10Topotecan44

Evidence

Results reported
36.7%121 of 330 completed
Published
18.2%60 of 330 completed
Primary endpoint met
62.5%10 of 16 analyses
Median months to report
12

Documents disclosed

Protocol available
76
Statistical analysis plan
73
PubMed citations
208

Trial profile

Median enrollment
38
Median duration
70.9months
Countries
48
Study sites
2503

Who is enrolled

Adults
596
Children
612
Older adults
214
Healthy volunteers
14

Median enrollment by phase

Early Phase 1
15
Phase 1
24
Phase 1/2
40
Phase 2
40
Phase 2/3
30
Phase 3
360
Phase 4
132
Not applicable
44
Unknown
125

Where trials run

United States
460
Canada
87
France
52
Australia
49
United Kingdom
48
China
37
Spain
35
Germany
34
Italy
34
Puerto Rico
28

Busiest cities

#CityCountryTrials
1New YorkUnited States150
2PhiladelphiaUnited States121
3BostonUnited States116
4SeattleUnited States113
5San FranciscoUnited States108
6CincinnatiUnited States107
7HoustonUnited States107
8ChicagoUnited States104
9Los AngelesUnited States101
10AtlantaUnited States95

Terminations

Terminated
64
Withdrawn
26
Suspended
1
Stopped for recruitment
5.1%33 of 650 trials

Stated reasons

Other / unclear
34
Recruitment
33
Business decision
9
Regulatory
5
Lack of efficacy
3
Funding
3
Safety
3
COVID-19
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
NCT04539366 Suspended Suspended Other - review of safety data 2040-12-31
NCT02439788 Withdrawn Withdrawn Based on newly available preclinical data we changed the CAR construct to a more effective version and will now study that product on a different protocol. 2030-10-31
NCT06335745 Withdrawn Withdrawn Study halted prematurely, prior to enrollment of first participant 2028-09-30
NCT06182410 Withdrawn Withdrawn Industry sponsor decision 2027-05-31
NCT04909515 Withdrawn Withdrawn Study terminated due to business priorities 2027-04-30
NCT06233903 Withdrawn Withdrawn Logistical considerations 2025-12-31
NCT04239092 Terminated Terminated The decision to conclude study was made to optimize the clinical study design and protocol to further evaluate the safety profile of elraglusib (9-ING-41) in pediatric and adult patients with refractory Ewings Sarcoma. 2025-07-07
NCT04544995 Terminated Terminated Sponsor Decision 2025-04-23
NCT04023331 Terminated Terminated Sponsor decision 2025-03-25
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

Trial records