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Systematic review of clinical drug development activities for neuroblastoma from 2011 to 2020
Jaclynne H Nader1, Florence Bourgeois2,3, Rochelle Bagatell4
1UMass Chan Medical School, Worcester, Massachusetts, USA.
Background:
Understanding the landscape of clinical trials for patients with neuroblastoma may inform efforts to improve drug development.
Procedure:
We evaluated therapeutic trials for patients with neuroblastoma from 2011 to 2020 in our search using clinical trial information from ClinicalTrials.gov, Clinicaltrialregister.eu, PubMed, and American Society of Clinical Oncology (ASCO) annual meeting collection. Trends in trials and treatments over time were evaluated qualitatively.
Results:
A total of 192 trials met inclusion criteria. A median of 20.5 trials were started per year, which was stable over time. There were 87 (45%) phase 1, 100 (51%) phase 2, and only five (2.6%) phase 3 trials. The median time to completion was 4.9 years for phase 1 and 2 trials (no phase 3 trials reported as completed during the study period). In all, 34% of trials were international, while 20% of trials were intercontinental. Eighty-nine percent of nonmyeloablative trials included at least one novel agent. 48% of these trials studied combination therapies, and 86% of these combinations included conventional chemotherapy. Among 157 trials that included a targeted agent, 78 targets were identified, with GD2 being the primary target under investigation in 16.7% of these trials. Only eight trials were included in regulatory decisions, which led to European Medicines Agency (EMA) or Food and Drug Administration (FDA) approval for neuroblastoma.
Conclusions:
The large number of trials initiated per year, the range of targets, and the rate of intercontinental collaboration are encouraging. The paucity of late-stage trials, the prolonged trial duration, and relative lack of combination studies are major causes of concern. This work will inform future drug development for neuroblastoma.
Insights
Neuroblastoma clinical trials show stable initiation but a concerning lack of late-stage studies and combination therapies. This analysis aims to guide future neuroblastoma drug development strategies.
Area of Science:
- Pediatric Oncology
- Clinical Pharmacology
- Translational Medicine
Background:
- Understanding neuroblastoma clinical trials is crucial for advancing drug development.
- A comprehensive analysis of therapeutic trials for neuroblastoma patients was conducted.
Purpose of the Study:
- To evaluate trends in neuroblastoma clinical trials from 2011 to 2020.
- To identify key challenges and opportunities in neuroblastoma drug development.
Main Methods:
- Therapeutic trials for neuroblastoma were identified using multiple databases (ClinicalTrials.gov, Clinicaltrialregister.eu, PubMed, ASCO).
- Trends in trial initiation, phase distribution, duration, and collaboration were qualitatively assessed.
- Investigated novel agents, combination therapies, targeted agents, and regulatory approvals.
Main Results:
- 192 trials met inclusion criteria, with a median of 20.5 trials initiated annually.
- A majority of trials were Phase 1 (45%) and Phase 2 (51%), with very few Phase 3 (2.6%).
- GD2 was a primary target; only eight trials led to EMA or FDA approval.
Conclusions:
- Encouraging aspects include numerous trials, diverse targets, and international collaboration.
- Significant concerns involve the scarcity of late-stage trials, long trial durations, and limited combination studies.
- Findings will inform and guide future neuroblastoma drug development efforts.
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