131-I-MIBG Therapy for Refractory Neuroblastoma, Expanded Access Protocol
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|ClinicalTrials.gov Identifier: NCT01163383|
Expanded Access Status : Available
First Posted : July 15, 2010
Last Update Posted : January 27, 2021
Metaiodobenzylguanidine (MIBG) is a substance that is taken up by neuroblastoma cells. MIBG is combined with radioactive iodine (131 I) in the laboratory to form a radioactive compound 131 I-MIBG. This radioactive compound delivers radiation specifically to the cancer cells and causes them to die.
The purpose of this research protocol provides a mechanism to deliver MIBG therapy when clinically indicated, but also to provide a mechanism to continue to collect efficacy and toxicity data that will be provided.
A recent New Approaches to Neuroblastoma Therapy (NANT) phase 2 randomized trial of 131I-MIBG with or without radiation sensitizers for relapsed refractory or persistent neuroblastoma enrolled 114 patients ages 1-30 years showed that Arm A (MIBG alone) had a response rate of 17%, Arm B (MIBG with Vincristine and Irnotecan) had a response rate of 14% and Arm C (MIBG with vorinostat) had a response rate of 32% after the first cycle. After the second cycle, Arm A had a response rate of 33%, Arm B had 30% response rate and Arm C had a 75% response rate. There was an excess of toxicities in Arm B, and no significant SAEs in Arm C. These data were reported at the American Society of Clinical Oncology meeting in June of 2020.
Vorinostat has been used extensively in adults and has been granted US FDA approval for the treatment of cutaneous T-cell lymphoma. The approved adult dose is 400 mg orally once daily. Vorinostat is not FDA approved for use in neuroblastoma.
|Condition or disease||Intervention/treatment|
|Neuroblastoma Childhood Metastatic Pheochromocytoma||Drug: 131 I-Metaiodobenzylguanidine (131 I-MIBG) Drug: 131 I-MIBG|
|Study Type :||Expanded Access|
|Official Title:||I-Metaiodobenzylguanidine (131 I-MIBG) Therapy for Refractory Neuroblastoma, Expanded Access Protocol|
- Drug: 131 I-Metaiodobenzylguanidine (131 I-MIBG)
131 I-MIBG 1-18mCi/kg given intravenously on day 1. Subjects may receive multiple courses every 4-8 weeks depending on the dose given.Other Names:
- 131 I-Metaiodobenzylguanidine
- radioactive Iodine (131)
- Drug: 131 I-MIBG
131-I-MIBG will be infused intravenously over 60-90 minutes once per course. The dose will be determined by the treating physician.Other Names:
- 131 I- Metaidobenzylguanidine
- Radioactive iodine
- Metaiodobenzylguanidine therapy
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01163383
|Contact: Maria Gemino-Borromeoemail@example.com|
|Contact: Yael Mosse, MDfirstname.lastname@example.org|
|United States, Pennsylvania|
|The Children's Hospital of Philadelphia||Available|
|Philadelphia, Pennsylvania, United States, 19104|
|Principal Investigator:||John M Maris, MD||Children's Hospital of Philadelphia|