131I-Metaiodobenzylguanidine (131I-MIBG) Therapy for Relapsed/Refractory Neuroblastoma

NCT03649438 | Unknown

• 1 other identifier: STU-2019-0665
• Study Type: expanded_access
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

This expanded access is the best available therapy/compassionate use designed to determine the palliative benefit and toxicity of 131I-MIBG in patients with relapsed/refractory neuroblastoma or metastatic pheochromocytoma who are not eligible for therapies of higher priority. Patients may receive a range of doses depending on stem cell availability and tumor involvement of bone marrow. Response rate, toxicity, and time to progression and death will be evaluated.

Conditions

Relapsed Neuroblastoma; Metastatic Pheochromocytoma

Interventions

131 I-Metaiodobenzylguanidine DRUG

* Minimum dose of 10 mCi/kg for patients without a stem cell source whose renal function is above the upper limit of normal but still meets eligibility criteria. * Dose of 12 mCi/kg for patients without a stem cell source with normal renal function and meets other eligibility criteria. * Dose of \> 12 mCi/kg to 18 mCi/kg maximum at investigator's discretion for patients meeting eligibility criteria with stem cells available.

Also known as: 131I-MIBG

Potassium Iodide DRUG

For the therapeutic MIBG administration, potassium iodide solution will be administered in a loading dose of 6mg/kg orally at least 8 hours prior to the MIBG injection, and then will be given at 1mg/kg/dose every 4 hours on days 0-6, then 1 mg/kg/day through day 45 post injection.

Also known as: KI solution

G-csf DRUG

It is recommended that patients with ANC less than 750 after MIBG infusion begin G-CSF 5 mcg/kg/day subcutaneously (or receive equivalent single dose of Neulasta every 14 days while neutropenic) until neutrophil recovery (generally \>5000). This will start 24 hours after stem cell infusion (if stem cells are to be infused).

Also known as: Neulasta

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis:
• Relapsed/refractory neuroblastoma with original diagnosis based on tumor histopathology or elevated urine catecholamines with typical neuroblastoma cells in the bone marrow
• Metastatic pheochromocytoma
• Age \>1 year and able to cooperate with radiation safety restrictions during therapy period
• Karnofsky or Lansky performance status of ≥ 50%
• Life expectancy: patients must have a life expectancy of at least 8 weeks
• Disease status: Failure to respond to standard therapy (usually combination chemotherapy with or without radiation and surgery) or development of progressive disease at any phase of standard therapy (any new lesion or an increase in size \>25% of a pre-existing lesion). Patients may enter this study with or without re-induction therapy for recurrent tumor.
• Disease must be evaluable by MIBG scan. A positive MIBG scan must be present within 8 weeks prior to study entry and subsequent to any intervening therapy. If the patient has only one MIBG positive lesion and that lesion was radiated, a biopsy must be done at least 4 weeks after radiation was completed and must show viable neuroblastoma.
• Stem Cells: If a patient does not have a hematopoietic stem cell product available for re-infusion after MIBG treatment, they may not receive a 131IMIBG dose \>12 mCi/kg. Patients must have a hematopoietic stem cell product available for re-infusion after MIBG treatment at doses of \> 12 mCi/kg. The minimum quantity for peripheral blood stem cells is 1.5 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).
• Stem cell source: The patients on this protocol will have autologous PBSCs available.
• Have acceptable organ function as defined below within 7 days of enrollment:
• Bone Marrow: ANC ≥750 X 109 /L, platelets ≥50,000 X 109 /L, and hemoglobin ≥ 8.0 g/dL without transfusion if stem cells are not available. ANC ≥500 X 109 /L, platelets ≥20,000 X 109 /L, and hemoglobin ≥ 8.0 g/dL with transfusions allowed if stem cells are available. Patients with stem cells available are excluded if they require two platelet transfusions per week to maintain the minimum required platelet count. A bone marrow examination is not medically indicated for patients diagnosed with metastatic pheochromocytoma.
• Renal: Creatinine ≤ 2 times upper limit of normal
• Hepatic: Bilirubin ≤2x upper limit of normal; AST/ALT ≤10x upper limit of normal
• Cardiac: Ejection fraction ≥ 45% on echocardiogram or shortening fraction

You may not qualify if:

• Patients with disease of any major organ system that would compromise their ability to withstand therapy.
• Because of the teratogenic potential of the study medication, 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 damage to the fetus.
• Known allergy to any of the agents or their ingredients used in this study.
• Patients who are on hemodialysis
• Patients with untreated positive blood cultures or progressive infections as assessed by radiographic studies
• Patients who have had prior treatment with 131I-MIBG who do not meet the re-treatment criteria.
• In patients with metastatic pheochromocytoma, screening urinalysis required prior to study enrollment. If random collection urine specimen is positive for proteinuria, patients must have 24-hour urine protein determination. Patients with metastatic pheochromocytoma are excluded if 24-hour urine protein is above the institutional upper limit of normal.
• Patients with known MIBG-avid parenchymal brain metastases are excluded.

Sponsors & Collaborators

• University of Texas Southwestern Medical Center: lead

Study Sites (1)

The University of Texas Southwestern Medical Center

Dallas, Texas, 75235, United States

Location

MeSH Terms

Conditions

Neuroblastoma; Pheochromocytoma

Interventions

3-Iodobenzylguanidine; Potassium Iodide; Granulocyte Colony-Stimulating Factor; pegfilgrastim

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors, Primitive, Peripheral; Neuroectodermal Tumors, Primitive; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue; Paraganglioma; Neuroendocrine Tumors

Intervention Hierarchy (Ancestors)

Guanidines; Amidines; Organic Chemicals; Iodobenzenes; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Hydrocarbons, Iodinated; Hydrocarbons, Halogenated; Iodides; Iodine Compounds; Inorganic Chemicals; Potassium Compounds; Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Study Officials

• Tanya Watt, MD

  University of Texas Southwestern Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: expanded access
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor

Study Record Dates

• First Submitted: July 18, 2018
• First Posted: August 28, 2018
• Last Updated: December 11, 2024

Record last verified: 2024-12

Locations

US (1)