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131-I-MIBG Therapy for Refractory Neuroblastoma, Expanded Access Protocol

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT01163383
Expanded Access Status : Available
First Posted : July 15, 2010
Last Update Posted : July 13, 2022
Information provided by (Responsible Party):
John Maris, Children's Hospital of Philadelphia

Brief Summary:

Metaiodobenzylguanidine (MIBG) is a substance that is taken up by neuroblastoma cells. MIBG is combined with radioactive iodine (131 I) in the laboratory to form a radioactive compound 131 I-MIBG. This radioactive compound delivers radiation specifically to the cancer cells and causes them to die.

The purpose of this research protocol provides a mechanism to deliver MIBG therapy when clinically indicated, but also to provide a mechanism to continue to collect efficacy and toxicity data that will be provided.

A recent New Approaches to Neuroblastoma Therapy (NANT) phase 2 randomized trial of 131I-MIBG with or without radiation sensitizers for relapsed refractory or persistent neuroblastoma enrolled 114 patients ages 1-30 years showed that Arm A (MIBG alone) had a response rate of 17%, Arm B (MIBG with Vincristine and Irnotecan) had a response rate of 14% and Arm C (MIBG with vorinostat) had a response rate of 32% after the first cycle. After the second cycle, Arm A had a response rate of 33%, Arm B had 30% response rate and Arm C had a 75% response rate. There was an excess of toxicities in Arm B, and no significant SAEs in Arm C. These data were reported at the American Society of Clinical Oncology meeting in June of 2020.

Vorinostat has been used extensively in adults and has been granted US FDA approval for the treatment of cutaneous T-cell lymphoma. The approved adult dose is 400 mg orally once daily. Vorinostat is not FDA approved for use in neuroblastoma.

Condition or disease Intervention/treatment
Neuroblastoma Childhood Metastatic Pheochromocytoma Drug: 131 I-Metaiodobenzylguanidine (131 I-MIBG) Drug: 131 I-MIBG

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Study Type : Expanded Access
Official Title: I-Metaiodobenzylguanidine (131 I-MIBG) Therapy for Refractory Neuroblastoma, Expanded Access Protocol

Intervention Details:
  • Drug: 131 I-Metaiodobenzylguanidine (131 I-MIBG)
    131 I-MIBG 1-18mCi/kg given intravenously on day 1. Subjects may receive multiple courses every 4-8 weeks depending on the dose given.
    Other Names:
    • MIBG
    • 131 I-Metaiodobenzylguanidine
    • radioactive Iodine (131)
    • Metaiodobenzylguanidine
  • Drug: 131 I-MIBG
    131-I-MIBG will be infused intravenously over 60-90 minutes once per course. The dose will be determined by the treating physician.
    Other Names:
    • 131 I- Metaidobenzylguanidine
    • MIBG
    • Radioactive iodine
    • Metaiodobenzylguanidine therapy

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   1 Year and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Refractory or relapsed neuroblastoma
  • Age greater than 1 year and able to cooperate with radiation safety restrictions during therapy period.
  • Performance Level: Patients must have a Karnofsky or Lansky performance status of equal to or greater than 50 percent
  • 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. Disease evaluable by MIBG scan must be present within 8 weeks of study entry and subsequent to any intervening therapy. The principal or co-investigator can waive the requirement for intervening therapy if in their judgment this would pose undue risk and would not affect ability to judge treatment effectiveness.
  • Stem cells: Patients must have a hematopoietic stem cell product available for re-infusion after MIBG treatment at doses of at least 12 mCi/kg. The recommended minimum quantity for peripheral blood stem cells is 1.0 x 10^6 CD34+ cells/kg. The minimum dose for bone marrow is 1.0 x 10^8 mononuclear cells/kg. If no stem cells are available, the dose of 131 I-MIBG should be 12mCi/kg or less.
  • 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. Subjects cannot be receiving chemotherapy, cytokine therapy or other investigational agents, and must have fully recovered from the toxic effects of any prior therapy. No investigational agents are allowed in this time frame, but FDA-approved drugs for other indications that are not cytotoxic are allowed to be used off label if this is considered in the best interest of the patient by the investigator. No concomitant cytotoxic therapy is permitted with the exception of vorinostat.
  • Liver function: Bilirubin ≤2x upper limit of normal; AST/ALT ≤10x upper limit of normal
  • Kidney function: Creatinine ≤3x upper limit of normal
  • Signed informed consent: The patient and/or the patient's legally authorized guardian must provide written informed consent to participate in this expanded access protocol.

Exclusion criteria

  • Patients with disease of any major organ system that would compromise their ability to withstand therapy, as deemed by the principal investigator or treating sub-investigator.
  • Because of the teratogenic potential of the study medications, 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.
  • Patients who are on hemodialysis
  • Patients with uncontrolled infections
  • Exceptions to the above eligibility criteria may be allowed if approved by the principal investigator as long as exception does not compromise the safety of the subject and the exception is clearly documented. Each protocol exception must be reviewed by the Institutional Review Board before therapy is initiated.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01163383

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Contact: Maria Gemino-Borromeo
Contact: Yael Mosse, MD

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United States, Pennsylvania
The Children's Hospital of Philadelphia Available
Philadelphia, Pennsylvania, United States, 19104
Sponsors and Collaborators
John Maris
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Principal Investigator: John M Maris, MD Children's Hospital of Philadelphia
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Responsible Party: John Maris, Director of Children's Hospital Cancer Center, Children's Hospital of Philadelphia Identifier: NCT01163383    
Other Study ID Numbers: 05-004159
CHP-830 ( Other Identifier: The Children's Hospital of Philadelphia )
First Posted: July 15, 2010    Key Record Dates
Last Update Posted: July 13, 2022
Last Verified: July 2022
Keywords provided by John Maris, Children's Hospital of Philadelphia:
Refractory Neuroblastoma
Recurrent Neuroblastoma
stage IV neuroblastoma
Additional relevant MeSH terms:
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Neuroectodermal Tumors, Primitive, Peripheral
Neuroectodermal Tumors, Primitive
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Neuroendocrine Tumors
Cadexomer iodine
Anti-Infective Agents, Local
Anti-Infective Agents
Trace Elements
Physiological Effects of Drugs
Antineoplastic Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action