131I-Labeled MIBG for Refractory Neuroblastoma: A Compassionate Use Protocol
Expanded access is currently available for this treatment.
Verified June 2012 by University of California, San Francisco
Information provided by (Responsible Party):
Katherine Matthay, University of California, San Francisco
First received: May 27, 2011
Last updated: June 14, 2012
Last verified: June 2012
This is a compassionate use protocol to allow patients with advanced neuroblastoma palliative access to 131I-metaiodobenzylguanidine (131I-MIBG).
Drug: Metaiodobenzylguanidine (MIBG)
What is Expanded Access?
||131I-Labeled MIBG for Refractory Neuroblastoma: A Compassionate Use Protocol
| Study Start Date:
| Estimated Study Completion Date:
| Estimated Primary Completion Date:
||May 2016 (Final data collection date for primary outcome measure)
Drug: Metaiodobenzylguanidine (MIBG)
131I-MIBG Therapeutic Administration. Therapeutic 131I-MIBG will be synthesized at Nuclear Diagnostic Products (NDP; Rockaway, New Jersey) with specific activities of 9-18 Ci/mmole. The therapeutic dose (8-18 mCi/kg at investigator's discretion; any dose greater than 12 requires stored stem cells) will be diluted in 25 ml of normal saline, and will be infused intravenously through a patient's central line, if already present, or a peripheral IV if a central line is not present over 120 minutes.
Other Name: 131I-MIBG
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.
|Ages Eligible for Study:
||1 Year and older
|Genders Eligible for Study:
|Accepts Healthy Volunteers:
- 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.
- 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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01370330
|University of California, San Francisco
|San Francisco, California, United States, 94143 |
|Contact: Katherine Matthay, M.D. 415-476-4764 firstname.lastname@example.org |
|Principal Investigator: Katherine Matthay, M.D. |
University of California, San Francisco
||Katherine Matthay, M.D.
||University of California, San Francisco
No publications provided
||Katherine Matthay, Principal Investigator, University of California, San Francisco
History of Changes
|Other Study ID Numbers:
|Study First Received:
||May 27, 2011
||June 14, 2012
||United States: Food and Drug Administration
Keywords provided by University of California, San Francisco:
Additional relevant MeSH terms:
ClinicalTrials.gov processed this record on March 06, 2014
Neuroectodermal Tumors, Primitive, Peripheral
Neuroectodermal Tumors, Primitive
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Molecular Mechanisms of Pharmacological Action