A Compassionate Use/Expanded Access Protocol Using 131I-MIBG Therapy for Patients With Refractory Neuroblastoma and Metastatic Pheochromocytoma

Study Purpose

This is an expanded access protocol/compassionate use single institution study designed to determine the palliative benefit and toxicity of 131I-MIBG in patients with progressive neuroblastoma and metastatic pheochromocytoma who are not eligible for therapies of higher priority. Response rate, toxicity, and time to progression and death will be evaluated.

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 365 Days - 29 Years
Gender All
More Inclusion & Exclusion Criteria

Inclusion Criteria:

  • - Diagnosis: Relapsed/Refractory neuroblastoma, with an original diagnosis made histologically or from elevated urine catecholamines with abnormal tumor cells in bone marrow OR relapsed/refractory pheochromocytoma.
  • - Disease status: Progressive disease at any time (defined as any new lesion or an increase in size by >25% of pre-existing disease), or a failure to respond to standard therapy.
Patients must have evidence of MIBG uptake into tumor at ≥ one site within 6 weeks prior to entry on study and subsequent to any intervening therapy
  • - Prior therapy: A minimum of two weeks since should have elapsed since any chemotherapy causing myelosuppression.
It must be a minimum of three months since receiving radiation to any of the following fields: craniospinal, total abdominal, whole lung, total body irradiation. For any other sites of radiation, at least 2 weeks should have elapsed. For patients who received radiation to the only site of MIBG-avid disease within two months of study entry, biopsy confirmation of residual active disease is required, with positive bone marrow being sufficient. At least 7 days should have elapsed since completion of therapy with a biologic agent and at least 3 half-lives should have elapsed since therapy with a monoclonal antibody. No cytokine therapy may be given within 24 hours of receiving 131I-MIBG. Patients may have received prior MIBG therapy, provided they demonstrated a response or stable disease initially, with progressive disease occurring at least 35 days following treatment.
  • - Organ function: ANC >500/uL, platelets >20,000/uL with transfusion allowed.
Bilirubin ≤2x ULN, AST/ALT ≤10x ULN.Serum Creatinine ≤2x ULN OR 24-hour creatinine clearance OR GFR ≥60ml/min/1.73m2. Normal lung function demonstrated by no dyspnea, exercise intolerance or oxygen requirement. Oxygen saturation ≥94% on room air. No clinically significant cardiac dysfunction and ejection fraction ≥45% on echocardiogram.
  • - Stem cells: Patients must have a minimum of 2.0 x106/kg viable CD34+ peripheral blood stem cells for re-infusion following 131I-MIBG.
An additional back-up of 2.0x106/kg CD34+ cells is recommended but not required.
  • - Life expectancy longer than 8 weeks,Karnofsky or Lansky performance status of ≥ 50%

    Exclusion Criteria:

    - Pregnant or lactating patients - Disease of any organ system that would compromise the patient's ability to participate in the study, including hemodialysis.
Significant organ impairment should be discussed with the Principal Investigator prior to study entry
  • - Patients with active grade 3-4 infection, as defined by the NCI CTCAE V4.0.
  • - Patients with known MBIG-avid brain parenchymal disease (leptomeningeal or skull based metastases are eligible).
  • - In patients with metastatic pheochromocytoma, a urinalysis must be preformed prior to study enrollment.
If proteinuria is present, a 24 hour urine must be collected and total protein determined. If the 24-hour urine protein is above the institutional upper limit of normal, the patient is excluded.

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.

NCT03015844
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.

Northwell Health
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.

OtherOther
Overall Status Available
Countries United States
Conditions

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

Neuroblastoma, Pheochromocytoma

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.

Cohen Children's Medical Center, New Hyde Park, New York

Status

Address

Cohen Children's Medical Center

New Hyde Park, New York, 11040

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