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

April 11, 2023 updated by: Julie Krystal, Northwell Health
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.

Study Overview

Status

Available

Conditions

Intervention / Treatment

Study Type

Expanded Access

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • New York
      • New Hyde Park, New York, United States, 11040
        • Available
        • Cohen Children's Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year to 29 years (Child, Adult)

Accepts Healthy Volunteers

N/A

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Northwell Health

Collaborators

Cohen Children's Medical Center

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

January 5, 2017

First Submitted That Met QC Criteria

January 6, 2017

First Posted (Estimate)

January 10, 2017

Study Record Updates

Last Update Posted (Actual)

April 13, 2023

Last Update Submitted That Met QC Criteria

April 11, 2023

Last Verified

April 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CCMC1611

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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