Expanded Access Protocol Using 131I-MIBG

Study Purpose

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.

Recruitment Criteria

Accepts Healthy Volunteers

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

Unknown
Study Type

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.


Searching Both is inclusive of interventional and observational studies.

Expanded Access
Eligible Ages 12 Months and Over
Gender All
More Inclusion & Exclusion Criteria

INCLUSION CRITERIA:

1. 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. 2. Age ≥12 months and able to cooperate with radiation safety restrictions during therapy period with/without pharmacologic anxiolysis. 3. 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). 4. 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. 5. 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: 1. At least 2 weeks should have elapsed since any anti-tumor therapy and the patient must meet certain hematologic criteria. 2. 3 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. 3. Cytokine therapy (e.g. G-CSF, GM-CSF, IL-6, erythropoietin) must be discontinued a minimum of 24 hours prior to 131I-MIBG therapy. 4. Minimum of six weeks from previous 131I-MIBG therapy. 5. 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). 6. 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. 6. Organ Function: 1. Liver function: Bilirubin ≤ 2x upper limit of normal; AST/ALT ≤ 10x upper limit of normal. 2. Kidney function: i. Serum Creatinine ≤ 2x upper limit of normal OR ii. 24-hr creatinine clearance OR GFR ≥ 60 ml/min/1.73m2. c. Hematologic Criteria: ANC ≥750/uL; Platelets ≥ 50,000/uL without transfusion if stem cells are not available (ANC ≥ 500 and any platelet count allowed if stem cells available). Patient must be off myeloid growth factors for at least 24 hours. If the patient has received prior treatment with MIBG, they may be thrombocytopenic, but requiring no more than 2 platelet transfusions per week to maintain counts above 20,000/uL. Hemoglobin must be ≥ 10gm/dL (transfusion allowed) regardless of stored stem cell availability. d. Normal lung function, as manifested by no dyspnea at rest or exercise intolerance, no oxygen requirement. e. No clinically significant cardiac dysfunction. 7. Signed informed consent/assent has been obtained.

EXCLUSION CRITERIA:

1. Patients 12 years and older with iobenguane scan positive, unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma and marketed product is available. 2. Patients eligible for the Phase II (OPTIMUM) trial. 3. 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. 4. 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]. 5. Patients who are on hemodialysis. 6. 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. 7. Patients with active infections that meet grade 3-4 according to the current version of the NCI CTCAE. 8. Patients with known MIBG-avid parenchymal brain metastases are not eligible. (Patients with leptomeningeal or skull-based metastases are eligible.)

Trial Details

Trial ID:

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.

NCT01590680
Phase

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.

Lead Sponsor

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

Jubilant DraxImage Inc.
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

N/A
Principal Investigator Affiliation N/A
Agency Class

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

Industry
Overall Status Available
Countries United States
Conditions

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

Neuroblastoma, Pheochromocytoma, Paraganglioma
Additional Details

Primary Objectives:

  • - To provide 131I-MIBG for compassionate use in patients with neuroblastoma, who otherwise do not qualify for inclusion or cannot participate in the sponsor's pivotal Phase II, FDA-approved, clinical trial.
  • - To provide 131I-MIBG for compassionate use in patients with neuroblastoma in the absence of a commercially available FDA approved product for the indication.
  • - Provide palliative therapy with 131I-MIBG for patients with advanced neuroblastoma.
  • - To provide alternative therapeutic 131I-MIBG options for patients with pheochromocytoma / paraganglioma, not qualifying for FDA-approved MIBG treatment.
  • - To provide alternative therapeutic 131I-MIBG options for patients with pheochromocytoma / paraganglioma, in the absence of a commercially available FDA-approved product for that indication.
  • - Gain more information about acute and late toxicity of 131I-MIBG therapy for patients with refractory neuroblastoma, pheochromocytoma, or paraganglioma.
Patients will receive a therapeutic dose at the investigator's discretion (5-18 mCi/kg). However, a dose of 12 mCi/kg or higher requires stored stem cells. Patients may be eligible for additional 131I-MIBG treatments (up to a cumulative total of 3 treatments) if they meet certain criteria. Treatments with 131I-MIBG must be separated by a minimum of six weeks from previous 131I-MIBG therapy. Post-treatment evaluation will be performed 5-9 weeks (35-63 days) post treatment, and patients will be followed every 6 months until 2 years from therapy. All patients will have toxicity monitoring for 2 years following 131I-MIBG therapy, or until going off study.

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.

Children's Hospital Los Angeles, Los Angeles, California

Status

Available

Address

Children's Hospital Los Angeles

Los Angeles, California, 90027

Site Contact

Araz Marachelian, MD, MS

[email protected]

323-361-4624

Children's Hospital Colorado, Aurora, Colorado

Status

Available

Address

Children's Hospital Colorado

Aurora, Colorado, 80045

Site Contact

Suzanne B Smolik, RN,MSN

[email protected]

720-777-6823

Children's Healthcare of Atlanta, Atlanta, Georgia

Status

Available

Address

Children's Healthcare of Atlanta

Atlanta, Georgia, 30322

Site Contact

Sarah G Carlin, RN,BSN,APHON

[email protected]

404-785-0083

University of Chicago Medical Center, Chicago, Illinois

Status

Available

Address

University of Chicago Medical Center

Chicago, Illinois, 60637

Site Contact

Melissa Z Marx, MPH

[email protected]

773-702-2927

Dana-Farber Cancer Institute, Boston, Massachusetts

Status

Available

Address

Dana-Farber Cancer Institute

Boston, Massachusetts, 02215

Site Contact

Nora Hussey

[email protected]

617-632-5419

Ann Arbor, Michigan

Status

Available

Address

Michigan Medicine, University of Michigan

Ann Arbor, Michigan, 48105

Site Contact

Anne M Ellis, BS

[email protected]

734-936-5388

Washington University School of Medicine, Saint Louis, Missouri

Status

Available

Address

Washington University School of Medicine

Saint Louis, Missouri, 63110

Site Contact

Sally Jones

[email protected]

+1-215-930-4550

North Carolina Children's Hospital, Chapel Hill, North Carolina

Status

Available

Address

North Carolina Children's Hospital

Chapel Hill, North Carolina, 27599

Site Contact

Juanita Ramirez, BA

[email protected]

919-966-5785

Charlotte, North Carolina

Status

Available

Address

Carolinas Medical Center/ Levine Children's Hospital

Charlotte, North Carolina, 28203

Site Contact

Jontyce Green, RN

[email protected]

980-442-2356

Duke University Medical Center, Durham, North Carolina

Status

Available

Address

Duke University Medical Center

Durham, North Carolina, 27710

Site Contact

Tyler M Ray, BA

[email protected]

919-681-9186

Cincinnati Children's Hospital, Cincinnati, Ohio

Status

Available

Address

Cincinnati Children's Hospital

Cincinnati, Ohio, 45229-3039

Site Contact

Cancer Referral Line

[email protected]

513-626-2799

The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania

Status

Available

Address

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104

Site Contact

Maria Gemino-Borromeo, CCRP

[email protected]

267-425-1987

UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania

Status

Available

Address

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224

Site Contact

WeiPing DeBlasio, MBA,BSN,RN

[email protected]

412-692-5485

Nashville, Tennessee

Status

Available

Address

Monroe Carell Jr. Children's Hospital at Vanderbilt

Nashville, Tennessee, 37232

Site Contact

Eugenia DC Owens, BS

[email protected]

615-343-6169

Children's Medical Center Dallas, Dallas, Texas

Status

Available

Address

Children's Medical Center Dallas

Dallas, Texas, 75235

Site Contact

Tanya C Watt, MD

[email protected]

214-456-2382

Cook Children's Medical Center, Fort Worth, Texas

Status

Available

Address

Cook Children's Medical Center

Fort Worth, Texas, 76104

Site Contact

Tracey Easley, RN,BSN,CPHON

[email protected]

682-885-4017

Houston, Texas

Status

Available

Address

Baylor College of Medicine, Texas Children's Hospital

Houston, Texas, 77035

Site Contact

Akudo A Anyanwu, BSN,RN

[email protected]

832-824-3480

Seattle Children's Hospital, Seattle, Washington

Status

Available

Address

Seattle Children's Hospital

Seattle, Washington, 98105

Site Contact

Christine Goetz, BA,CCRC

[email protected]

206-884-1149

Madison, Wisconsin

Status

Available

Address

University of Wisconsin Hospital and Clinics, American Family Children's Hospital

Madison, Wisconsin, 53792

Site Contact

Jenny L Weiland

[email protected]

608-890-8070

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