131-I-MIBG Therapy for Refractory Neuroblastoma and Metastatic Paraganglioma/Pheochromocytoma
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.
Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms
An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.
An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.
|Eligible Ages||1 Year and Over|
- - Refractory or relapsed neuroblastoma OR malignant Paraganglioma or Pheochromocytoma - Age greater than 1 year and able to cooperate with radiation safety restrictions during therapy period.
- - Performance Level: Patients must have a Karnofsky or Lansky performance status of equal to or greater than 50 percent - Disease status: Failure to respond to standard therapy (usually combination chemotherapy with or without radiation and surgery) or development of progressive disease at any time.
- - Stem cells: Patients must have a hematopoietic stem cell product available for re-infusion after MIBG treatment at doses of at least 12 mCi/kg.
- - Prior Therapy: Patients may enter this study with or without re-induction therapy for recurrent tumor.
- - Liver function: Bilirubin ≤2x upper limit of normal; AST/ALT ≤10x upper limit of normal - Kidney function: Creatinine ≤3x upper limit of normal - Signed informed consent: The patient and/or the patient's legally authorized guardian must provide written informed consent to participate in this expanded access protocol.
- - Patients with disease of any major organ system that would compromise their ability to withstand therapy, as deemed by the principal investigator or treating sub-investigator.
- - Because of the teratogenic potential of the study medications, no patients who are pregnant or lactating will be allowed.
- - Patients who are on hemodialysis - Patients with uncontrolled infections - Exceptions to the above eligibility criteria may be allowed if approved by the principal investigator as long as exception does not compromise the safety of the subject and the exception is clearly documented.
This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.
Phase 0: Exploratory study involving very limited human exposure to the drug to determine whether a drug is modulating its target.
Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.
Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.
Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.
Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.
The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.
|Children's Hospital of Philadelphia|
Category of organization(s) involved as sponsor (and collaborator) supporting the trial.
The disease, disorder, syndrome, illness, or injury that is being studied.
|Neuroblastoma, Childhood Metastatic Pheochromocytoma, Paraganglioma|
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:
- - Assist with hygiene - Give oral medications - Offer and empty bedpans - Assist with meals - Change diapers (if used) - Change clothing and bed linens if soiled - Entertain or distract individuals who become upset or restless due to the isolation or procedures.
- - Record Dosimeter readings.
Contact a Trial Team
If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104