MIBG + Dinutuximab Therapy for Neuroblastoma
Trial Summary
Will I have to stop taking my current medications?
The trial protocol does not specify if you must stop taking your current medications. However, certain medications that interfere with MIBG uptake, prolong QTc, or include valproic acid (for Part B) are not allowed. It's best to discuss your current medications with the study team.
What data supports the effectiveness of the MIBG + Dinutuximab treatment for neuroblastoma?
Research shows that 131-I-MIBG, a component of the treatment, has been effective in reducing tumor size in neuroblastoma patients, with some achieving complete or partial responses. It is considered an active treatment option, especially for advanced or relapsed cases, and is comparable to chemotherapy in efficacy with less toxicity.12345
Is MIBG + Dinutuximab therapy safe for treating neuroblastoma?
131I-MIBG therapy has been used in treating neuroblastoma, and while it can be effective, there are safety concerns such as the risk of developing secondary leukemia. However, some studies have shown that it can be used without significant blood-related side effects, especially when used at lower doses.13567
What makes MIBG + Dinutuximab therapy unique for treating neuroblastoma?
MIBG + Dinutuximab therapy is unique because it combines a targeted radioactive treatment (MIBG) with an immunotherapy (Dinutuximab) that specifically targets neuroblastoma cells, potentially enhancing the effectiveness of treatment while reducing toxicity compared to traditional chemotherapy.178910
What is the purpose of this trial?
This trial tests a combination of a radioactive drug, an antibody, and possibly another drug in children with hard-to-treat neuroblastoma. The treatment works by killing cancer cells directly and helping the immune system attack them.
Research Team
Thomas Cash, MD
Principal Investigator
Children's Healthcare of Atlanta
Araz Marachelian, MD, MS
Principal Investigator
Children's Hospital Los Angeles
Eligibility Criteria
This trial is for pediatric patients with high-risk neuroblastoma, a type of cancer. They must have evidence of MIBG uptake in tumors or increased urinary catecholamines and meet specific criteria regarding their disease state (recurrent/progressive, refractory, persistent). Participants need to be recovered from prior treatments and have adequate organ function. Pregnant individuals or those unable to follow the study plan are excluded.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive 131I-MIBG, dinutuximab, and GM-CSF, with optional vorinostat in Cohort B
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- 131I-MIBG
- Dinutuximab
- Vorinostat
131I-MIBG is already approved in United States, European Union for the following indications:
- Pheochromocytoma
- Neuroblastoma
- Neuroblastoma
- Pheochromocytoma
- Paraganglioma
Find a Clinic Near You
Who Is Running the Clinical Trial?
New Approaches to Neuroblastoma Therapy Consortium
Lead Sponsor
National Cancer Institute (NCI)
Collaborator
United Therapeutics
Industry Sponsor
Dr. Martine Rothblatt
United Therapeutics
Chief Executive Officer since 1996
PhD in Medical Ethics from the Royal London College of Medicine and Dentistry, JD and MBA from UCLA
Dr. Michael Benkowitz
United Therapeutics
Chief Medical Officer since 2023
MD from Harvard Medical School