Clinical Trial Finder

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

Study Purpose

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.

Recruitment Criteria

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

Accepts Healthy Volunteers
No

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.

Study Type
Expanded Access
Eligible Ages 1 Year and Over
Gender All
More Inclusion & Exclusion Criteria

Inclusion Criteria:

  • - 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.
Disease evaluable by MIBG scan must be present within 8 weeks of study entry and subsequent to any intervening therapy. The principal or co-investigator can waive the requirement for intervening therapy if in their judgment this would pose undue risk and would not affect ability to judge treatment effectiveness.
  • - 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.
The recommended minimum quantity for peripheral blood stem cells is 1.0 x 10^6 CD34+ cells/kg. The minimum dose for bone marrow is 1.0 x 10^8 mononuclear cells/kg. If no stem cells are available, the dose of 131 I-MIBG should be 12mCi/kg or less.
  • - Prior Therapy: Patients may enter this study with or without re-induction therapy for recurrent tumor.
Patients must have fully recovered from the toxic effects of any prior therapy. Subjects cannot be receiving chemotherapy, cytokine therapy or other investigational agents, and must have fully recovered from the toxic effects of any prior therapy.
  • - 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.
Exclusion criteria
  • - 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 of childbearing potential must practice an effective method of birth control while participating on this study, to avoid possible damage to the fetus.
  • - 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.
Each protocol exception must be reviewed by the Institutional Review Board before therapy is initiated.

Trial Details

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.

Trial ID:
NCT01163383

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.

Phase
N/A

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Lead Sponsor
Children's Hospital of Philadelphia

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Agency Class
Other
Overall Status Available
Countries United States

The disease, disorder, syndrome, illness, or injury that is being studied.

Conditions
Neuroblastoma, Childhood Metastatic Pheochromocytoma, Paraganglioma
Study Website: View Trial Website
Additional Details

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.
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.

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The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania

Status

Address

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104

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