Expanded Access Protocol Using 131I-MIBG
An Open Label, Expanded Access Protocol Using 131I-metaiodobenzylguanidine (131I-MIBG) Therapy in Patients With Refractory Neuroblastoma, Pheochromocytoma, or Paraganglioma (Not Eligible for Approved Treatment)
1 other identifier
expanded_access
N/A
1 country
19
Brief Summary
Protocol JDI2007-01 is an Expanded Access Protocol with therapeutic 131I-MIBG for patients with neuroblastoma or pheochromocytoma / paraganglioma, who otherwise do not qualify for available treatments, or where approved treatment is not commercially available.
Trial Health
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19 active sites
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Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
May 1, 2012
CompletedFirst Posted
Study publicly available on registry
May 3, 2012
CompletedFebruary 28, 2023
February 1, 2023
May 1, 2012
February 26, 2023
Conditions
Interventions
The therapeutic dose (5-18 mCi/kg at investigator's discretion; any dose ≥12 mCi/kg requires stored stem cells) will be diluted in normal saline, and will be infused intravenously over 90-120 minutes.
Eligibility Criteria
You may qualify if:
- Diagnosis: Refractory or relapsed neuroblastoma with original diagnosis based on tumor histopathology or elevated urine catecholamines with typical tumor cells in the bone marrow, OR pheochromocytoma or paraganglioma (less than 12 years of age) not amenable to curative surgery.
- Age ≥12 months and able to cooperate with radiation safety restrictions during therapy period with/without pharmacologic anxiolysis.
- 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 evaluation must be completed within 8 weeks of study entry. If possible, the disease evaluation should take place subsequent to any intervening therapy; if intervening therapy does occur, evaluations should be done as clinically indicated. If patient has received prior treatment with MIBG, they must have a response or stable disease after the most recent MIBG infusion. Patient may have PD after showing an initial response to MIBG therapy (at \[or around\] the day 35-63 post-MIBG therapy evaluation).
- Stem cells: Patients must have a hematopoietic stem cell product available for re-infusion after 131I-MIBG treatment at doses of 12 mCi/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, meeting the following criteria:
- At least 2 weeks should have elapsed since any anti-tumor therapy and the patient must meet certain hematologic criteria.
- months should have elapsed in the case of completing external beam radiation for total abdominal, whole lung, total body irradiation (spot irradiation to skull-based metastases is NOT a contraindication). Patients who receive localized emergency radiation to sites of life-threatening or function-threatening disease prior to or immediately after establishment of the definitive diagnosis are not contraindicated for treatment on this protocol.
- Cytokine therapy (e.g. G-CSF, GM-CSF, IL-6, erythropoietin) must be discontinued a minimum of 24 hours prior to 131I-MIBG therapy.
- Minimum of six weeks from previous 131I-MIBG therapy.
- The lifetime cumulative injected activity should be evaluated by the Investigator on a case-by-case basis with special attention to any recovery from past 131I-MIBG dose(s).
- For patients who received a stem cell infusion for a previous 131I-MIBG therapy but do NOT have remaining stored stem cells:
- i. If the stem cell reinfusion was protocol driven but not based upon the development of profound cytopenias (e.g. automatic stem cell reinfusion on Day 14), the patient is eligible for retreatment with MIBG at a dose \<12 mCi/kg at the investigators discretion; ii. If the stem cell reinfusion was given based upon the development of profound cytopenias, decisions for re-treatment with 131I-MIBG will require a case-by-case evaluation by the Investigator.
- Organ Function:
- Liver function: Bilirubin ≤ 2x upper limit of normal; AST/ALT ≤ 10x upper limit of normal.
- Kidney function:
- +5 more criteria
You may not qualify if:
- Patients 12 years and older with iobenguane scan positive, unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma and marketed product is available.
- Patients eligible for the Phase II (OPTIMUM) trial.
- 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 Principal Investigator 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, who are sexually active, must practice an effective method of birth control while participating on this study, to avoid possible damage to the fetus . \[e.g. intrauterine device, double-barrier method (i.e., diaphragm, or a cervical cap) with intravaginal spermicidal foam, cream or gel\], or male partner sterilization throughout the study\].
- Patients who are on hemodialysis
- Proteinuria, in the absence of urinary infection, within 4 weeks prior to the planned treatment date is a relative contraindication to receiving therapy for patients with pheochromocytoma/paraganglioma. Patients with pheochromocytoma/paraganglioma with any clinically significant proteinuria must have a 24-hr urine protein determination. If proteinuria is confirmed as being above the institutional upper limit of normal, the patient is ineligible for MIBG therapy.
- Patients with active infections that meet grade 3-4 according to the current version of the NCI CTCAE.
- Patients with known MIBG-avid parenchymal brain metastases are not eligible. (Patients with leptomeningeal or skull-based metastases are eligible.)
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (19)
Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
Children's Hospital Colorado
Aurora, Colorado, 80045, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322, United States
University of Chicago Medical Center
Chicago, Illinois, 60637, United States
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215, United States
Michigan Medicine, University of Michigan
Ann Arbor, Michigan, 48105, United States
Washington University School of Medicine
St Louis, Missouri, 63110, United States
North Carolina Children's Hospital
Chapel Hill, North Carolina, 27599, United States
Carolinas Medical Center/ Levine Children's Hospital
Charlotte, North Carolina, 28203, United States
Duke University Medical Center
Durham, North Carolina, 27710, United States
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229-3039, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224, United States
Monroe Carell Jr. Children's Hospital at Vanderbilt
Nashville, Tennessee, 37232, United States
Children's Medical Center Dallas
Dallas, Texas, 75235, United States
Cook Children's Medical Center
Fort Worth, Texas, 76104, United States
Baylor College of Medicine, Texas Children's Hospital
Houston, Texas, 77035, United States
Seattle Children's Hospital
Seattle, Washington, 98105, United States
University of Wisconsin Hospital and Clinics, American Family Children's Hospital
Madison, Wisconsin, 53792, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- expanded access
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
May 1, 2012
First Posted
May 3, 2012
Last Updated
February 28, 2023
Record last verified: 2023-02