Clinical Trial: 131-I-MIBG Therapy for Refractory Neuroblastoma and Metastatic Paraganglioma/Pheochromocytoma

Study Status: Available
Recruit Status: Available
Study Type: Expanded Access

Official Title: I-Metaiodobenzylguanidine (131 I-MIBG) Therapy for Refractory Neuroblastoma and Paraganglioma/Pheochromocytoma Expanded Access Protocol

Brief Summary:

Metaiodobenzylguanidine (MIBG) is a substance that is taken up by neuroblastoma or pheochromocytoma/paraganglioma tumor 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 is to 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.


Detailed Summary:

131I-MIBG is experimental, but has been used in more than 100 children in the United States by itself to treat relapsed neuroblastoma and metastatic pheochromocytoma/ganglioma. A recent study using increasing doses of 131I-MIBG in both children and adults with relapsed neuroblastoma or metastatic pheochromocytoma/ganglioma showed anti-cancer effects in some of these patients. The main side effect of this treatment was a decrease in the number of normal blood-forming cells (called stem cells) in the bone marrow, but a dose of 12 mCi/kg did not cause permanent damage to the bone marrow in a small number of patients.

There are certain tests or procedures that will need to be done to confirm that the subject is eligible for this therapy. These include lab work, physical exam and MIBG scan. A CT scan, an MRI, a bone scan, Bone marrow aspirate and biopsy and urine tests will be done to evaluate your disease status when clinically indicated. Your doctor will determine which tests are required.

Subjects will need to have an intravenous catheter (tube) placed in a vein before beginning study treatment. An existing central venous catheter can be used to administer the medicine.

Because subjects' urine will be radioactive, a urinary catheter may be inserted to ensure drainage of the urine, which will be radioactive. The catheter will be removed 3-5 days following the treatment. General anesthesia or sedation is typically given for the procedure of inserting the catheter.

Subjects will be treated in a specially prepared room in the CHOP Pediatric Oncology Unit. Upon admission, the nursing staff will instruct caregivers on the care of the subject following the MIBG infusion. Because of the frequent exposure of the nursing staff to radiat
Sponsor: Children's Hospital of Philadelphia

Current Primary Outcome:

Original Primary Outcome: Number of Participants with Disease Response to 131-I-MIBG [ Time Frame: 8 weeks ]

Will measure disease response after treatment with 131 I- MIBG with post therapy scan. Will record response to therapy data for each subject after therapy.


Current Secondary Outcome:

Original Secondary Outcome: Number of Participants with Adverse events related to 131 I- MIBG therapy [ Time Frame: 8 weeks ]

Will measure the number of participants with adverse events as a measure of safety and tolerability


Information By: Children's Hospital of Philadelphia

Dates:
Date Received: July 13, 2010
Date Started: March 2005
Date Completion: July 2015
Last Updated: November 17, 2016
Last Verified: November 2016