131-I-MIBG Therapy for Refractory Neuroblastoma and Metastatic Paraganglioma/Pheochromocytoma
|First Received Date ICMJE||July 13, 2010|
|Last Updated Date||July 25, 2017|
|Start Date ICMJE||Not Provided|
|Primary Completion Date||Not Provided|
|Current Primary Outcome Measures ICMJE||Not Provided|
|Original Primary Outcome Measures ICMJE
||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.
|Change History||Complete list of historical versions of study NCT01163383 on ClinicalTrials.gov Archive Site|
|Current Secondary Outcome Measures ICMJE||Not Provided|
|Original Secondary Outcome Measures ICMJE
||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
|Current Other Outcome Measures ICMJE||Not Provided|
|Original Other Outcome Measures ICMJE||Not Provided|
|Brief Title ICMJE||131-I-MIBG Therapy for Refractory Neuroblastoma and Metastatic Paraganglioma/Pheochromocytoma|
|Official Title ICMJE||I-Metaiodobenzylguanidine (131 I-MIBG) Therapy for Refractory Neuroblastoma and Paraganglioma/Pheochromocytoma Expanded Access Protocol|
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.
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 radiation and the high level of radiation surrounding the subject during therapy, the nurses' contact will be limited to complex medical care, so that they are available for subjects in the event of an emergency. Adult family members will be expected to be present at all times during the hospitalization to:
Isolation: For 2-5 days subjects will be placed in a single room with a bed surrounded by lead shielding to prevent exposure of visitors and hospital personnel to radioactivity. Family members may visit in the room, but must wear a radiation badge to measure exposure. A single family member can sleep in the room, but no one is allowed to go behind the shields or sleep in the bed with the child.
Subjects will receive fluids through the central venous catheter. The fluids will begin at least four hours before and continue at least 72 hours after the 131I-MIBG treatment begins.
Subjects will take a medicine by mouth, potassium iodide, to prevent thyroid damage from the radioactive iodine contained in the 131I-MIBG. This medication will be taken on the day of the treatment and will continue for a total of 6 weeks.
The 131I-MIBG will be given through an intravenous catheter over 1-2 hours.
During the administration of the drug, subject's blood pressure and heart rate will be checked frequently.
Before and at regular intervals after treatment, subjects will have routine blood tests to check his/her blood counts, hormone, liver and kidney functions. Blood will be checked frequently for the first 1-6 weeks, and 6 weeks after the treatment. Approximately 1 teaspoon of blood will be drawn each time during the first 6 weeks to perform the tests listed above.
An MIBG scan will be performed to see where the drug is concentrating in the body following treatment. No injection of a radioactive marker will be required and this is usually done on the day for discharge. It is identical to the pretherapy MIBG scan except for not needing MIBG injection and it is usually shorter (15-30 minutes).
Six to eight weeks after treatment, an MIBG and other scans will be done to evaluate the response of the tumor to the treatment.
If the subject's tumor is responding or stable 6-7 weeks after each treatment, they may be eligible for a second and third course of treatment, as long as their white blood counts have recovered from the treatment and the patient has stem cells available if needed. If another MIBG treatment is given, it will be at the same dose as the first treatment, unless it is thought to be necessary for safety reasons (based on toxicity information from the first infusion) to decrease the dose.
If the subject's blood counts decrease following the therapy, treatment with Filgrastim (G-CSF) or Neulasta may be required. G-CSF/neulasta is a medicine that helps increase the white blood cells. This is given by a subcutaneous (under the skin) injection (like an insulin shot). GCSF is given daily and Neulasta is given every 14 days instead of GCSF. Neulasta works like GCSF to help increase the white blood counts but lasts longer in your child's body. This medicine will start if the absolute neutrophil count (ANC), a measure of the infection fighting cells, goes below 750 and will continue until it rises above 5000.
|Study Type ICMJE||Expanded Access|
|Study Phase||Not Provided|
|Study Design ICMJE||Not Provided|
|Study Arms||Not Provided|
|Publications *||Not Provided|
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
|Recruitment Status ICMJE||Available|
|Enrollment ICMJE||Not Provided|
|Completion Date||Not Provided|
|Primary Completion Date||Not Provided|
|Eligibility Criteria ICMJE||
|Ages||1 Year and older (Child, Adult, Senior)|
|Accepts Healthy Volunteers||No|
|Listed Location Countries ICMJE||United States|
|Removed Location Countries|
|NCT Number ICMJE||NCT01163383|
|Other Study ID Numbers ICMJE||2005-02-4159
CHP-830 ( Other Identifier: The Children's Hospital of Philadelphia )
|Has Data Monitoring Committee||Not Provided|
|U.S. FDA-regulated Product||Not Provided|
|IPD Sharing Statement||Not Provided|
|Responsible Party||Children's Hospital of Philadelphia|
|Study Sponsor ICMJE||Children's Hospital of Philadelphia|
|Collaborators ICMJE||Not Provided|
|PRS Account||Children's Hospital of Philadelphia|
|Verification Date||July 2017|
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