Clinical Trial Finder

131I-Labeled MIBG for Refractory Neuroblastoma: A Compassionate Use Protocol

Study Purpose

This is a compassionate use protocol to allow patients with advanced neuroblastoma palliative access to 131I-metaiodobenzylguanidine (131I-MIBG).

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:

  • - Diagnosis: Refractory or relapsed neuroblastoma with original diagnosis based on tumor histopathology or elevated urine catecholamines with typical tumor cells in the bone marrow.
  • - Age > 1 year and able to cooperate with radiation safety restrictions during therapy period.
  • - Life Expectancy: greater than 6 weeks.
  • - Lanksy and Karnofsky Performance Status: 60% or higher.
  • - 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 (any new lesion or an increase in size of >25% of a pre-existing lesion).
Disease evaluable by MIBG scan must be present within 6 weeks of study entry and subsequent to any intervening therapy.
  • - Stem cells: Patients must have an autologous hematopoietic stem cell product available for re-infusion after MIBG treatment at doses of >12 mCi/kg if needed.
The minimum quantity for purged or unpurged peripheral blood stem cells is 1.0 x 106 CD34+ cells/kg (optimum > 2 x 106 CD34+ cells/kg). The minimum dose for bone marrow is 1.0 x 108 mononuclear cells/kg (optimum > 2.0 x 108 mononuclear cells/kg). If no stem cells are available, then the dose of 131I-MIBG should be <12 mCi/kg .
  • - 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. At least 2 weeks should have elapsed since any anti-tumor therapy and the patient must meet hematologic criteria below. Three months should have elapsed in the case of completing radiation to any of the following fields: craniospinal, total abdominal, whole lung, total body irradiation). Cytokine therapy (eg G-CSF, GM-CSF, IL-6, erythropoietin) must be discontinued a minimum or 24 hours prior to MIBG therapy. Prior 131I-MIBG therapy is allowed if > 6 months previous and if the patient has adequate hematopoietic stem cells available.
  • - Organ Function - Liver function: bilirubin <2x normal and AST/ALT < 10x normal.
  • - Kidney function: Creatinine less than or equal to 2 - Hematopoietic Criteria Patients must have adequate hematopoietic function (without transfusion): ANC >.
750 x 10E9/L; Platelets >50 x 10E9/L if stem cells are not available; if stem cells are available, the patient should be independent of platelet transfusions with a platelet count of at least 20 x 10E9/L. Hemoglobin >10g/dl at time of treatment (transfusion allowed). Patients with granulocytopenia and/or thrombocytopenia due to tumor metastatic to the bone marrow may be eligible after discussion with Dr. Matthay or designee.
  • - Normal lung function as manifested by no dyspnea at rest or exercise intolerance, no oxygen requirement.
  • - No clinically significant cardiac dysfunction - Signed informed consent: The patient and/or the patient's legally authorized guardian must acknowledge in writing that consent to become a study subject has been obtained, in accordance with institutional policies approved by the U.
S. Department of Health and Human Services.

Exclusion Criteria:

  • - Patients with disease of any major organ system that would compromise their ability to withstand therapy.
Any significant organ impairment should be discussed with the Study Chair or Vice Chair prior to patient entry.
  • - 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 pregnancy.
  • - Patients who are on hemodialysis.
  • - Patients with active infections that meet grade 3-4 toxicity criteria.

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:
NCT01370330

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
University of California, San Francisco

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
Additional Details

Neuroblastoma remains a fatal disease for a large percentage of patients, especially those with high-risk disease features who become resistant to conventional therapy. 131I-metaiodobenzylguanidine (131I-MIBG) is a norepinephrine analog that concentrates in adrenergic tissue and therefore holds promise for cell-specific treatment of neuroblastoma. 131I-MIBG is active against relapsed or refractory neuroblastoma and associated hematopoietic toxicity can be abrogated with autologous stem cell rescue. 131I-MIBG given in doses of 10-18 mCi/kg with stem cell rescue, if necessary, is safe and effective palliative therapy for refractory or relapsed neuroblastoma patients.

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.

University of California, San Francisco, San Francisco, California

Status

Address

University of California, San Francisco

San Francisco, California, 94143

Site Contact

Katherine Matthay, M.D.

matthayk@peds.ucsf.edu

415-476-4764

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