131I-omburtamab for the Treatment of Central Nervous System/Leptomeningeal Neoplasms in Children and Young Adults

NCT05064306 | Unknown

• 1 other identifier: 21-345
• Study Type: expanded_access
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

The researchers are doing this study to provide access to treatment with 131I-omburtamab for children and young adults who have CNS/leptomeningeal neoplasms. 131I-omburtamab is an investigational drug; the FDA has not approved it to treat this cancer or any other disease. However, the agency has granted the drug Breakthrough Therapy Designation for the treatment of neuroblastoma with CNS metastases.

Conditions

Central Nervous System/Leptomeningeal Neoplasms

Keywords

131I-omburtamab; neuroblastoma; medulloblastoma; rhabdoid tumors; pineoblastoma; retinoblastoma; PNET; rhabdomyosarcoma; Ewing's sarcoma; Wilm's tumor; hepatoblastoma; non-embryonal tumors; histologic confirmation of B7-H3 reactivity; 21-345

Interventions

131I-omburtamab DRUG

50 mCi 131I-omburtamab

Eligibility Criteria

• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients must have a histologically confirmed diagnosis of an embryonal malignancy (including but not limited to neuroblastoma, medulloblastoma, rhabdoid tumors, pineoblastoma, retinoblastoma, PNET, rhabdomyosarcoma, Ewing's sarcoma, Wilm's tumor, hepatoblastoma) other non-embryonal tumors must have histologic confirmation of B7-H3 reactivity.
• Patients must have a diagnosis of CNS/ leptomeningeal disease which has been treated with conventional therapies or for which no conventional therapy exists or a recurrent brain tumor with a predilection for leptomeningeal dissemination (i.e. PNET, rhabdoid tumor).
• Chemotherapy:
• °Patients must have received their last dose of myelosuppressive anticancer therapy at least 21 days (3 weeks) prior to receiving treatment dose(s).
• Biologic or investigational agent (anti-neoplastic):
• Patients who are currently on biological (small molecule inhibitors such as ALK inhibitors, ROS inhibitors or MEK inhibitors etc.) or investigational agents that are considered non-myelosuppressive can continue treatment but should have recovered from any acute toxicity potentially related to the agent.
• Patients may be treated if they received one prior treatment dose with 131I-omburtamab
• Monoclonal antibody treatment and agents with known prolonged half-lives:
• Patients must have recovered from any acute toxicity potentially related to the agent and received their last dose of the agent ≥ 21 days prior to enrollment.
• Monoclonal antibody treatment and agents with known prolonged half-lives: Patient must have recovered from any acute toxicity potentially related to the agent and received their last dose of the agent ≥ 28 days prior to receiving treatment dose(s).
• Neurologic Status:
• Patients with neurological deficits should have deficits that are stable for a minimum of 1 week prior to enrollment.
• Patients with seizure disorders may be enrolled if seizures are controlled.
• Performance Status:
• °Karnofsky Performance Scale (KPS for \> 16 years of age) or Lansky Performance Score (LPS for ≤ 16 years of age) assessed within 2 weeks prior to study enrollment must be ≥ 50%. Patients who are unable to walk because of neurologic deficits, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.

You may not qualify if:

• Patients with obstructive or symptomatic communicating hydrocephalus
• Patients with an uncontrolled life-threatening infection
• Patients who are pregnant: negative pregnancy test is required for all women of childbearing age, and appropriate contraception is required during the study period.
• Patients who have received cranial or spinal irradiation less than 3 weeks prior to first dose

Sponsors & Collaborators

• Memorial Sloan Kettering Cancer Center: lead
• Y-mAbs Therapeutics: collaborator

Study Sites (1)

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

Location

Related Links

• Memorial Sloan Kettering Cancer Center

MeSH Terms

Conditions

Meningeal Neoplasms; Neuroblastoma; Medulloblastoma; Rhabdoid Tumor; Pinealoma; Retinoblastoma; Neuroectodermal Tumors, Primitive; Rhabdomyosarcoma; Sarcoma, Ewing; Wilms Tumor; Hepatoblastoma

Interventions

omburtamab I-131

Condition Hierarchy (Ancestors)

Central Nervous System Neoplasms; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Nervous System Diseases; Neuroectodermal Tumors, Primitive, Peripheral; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue; Glioma; Neoplasms, Complex and Mixed; Brain Neoplasms; Brain Diseases; Central Nervous System Diseases; Retinal Neoplasms; Eye Neoplasms; Eye Diseases, Hereditary; Eye Diseases; Retinal Diseases; Myosarcoma; Neoplasms, Muscle Tissue; Neoplasms, Connective and Soft Tissue; Sarcoma; Osteosarcoma; Neoplasms, Bone Tissue; Neoplasms, Connective Tissue; Kidney Neoplasms; Urologic Neoplasms; Urogenital Neoplasms; Neoplastic Syndromes, Hereditary; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Kidney Diseases; Urologic Diseases; Male Urogenital Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Sameer Farouk Sait, MD

  Memorial Sloan Kettering Cancer Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: expanded access
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 29, 2021
• First Posted: October 1, 2021
• Last Updated: August 1, 2025

Record last verified: 2025-07

Locations

US (1)