131I-omburtamab Radioimmunotherapy for Neuroblastoma Central Nervous System/Leptomeningeal Metastases

JRCT ID: jRCT2021200024

Registered date:21/10/2020

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: Neuroblastoma
• Date of first enrollment: 26/05/2021
• Target sample size: 5
• Countries of recruitment: United States,Japan,Denmark,Japan,Spain,Japan
• Study type: Interventional
• Intervention(s): Patients will receive up to two cycles of intracerebroventricular 131I-Omburtamab.

Outcome(s)

• Primary Outcome: 1. Overall survival rate at 3 years after the first treatment dose of 131I-omburtamab.

Secondary Outcome

1. CNS/LM PFS at 12 months
2. ORR assessed as a combination of partial response and complete response as defined by the Response assessment in Neuro-Oncology (RANO) group criteria for brain metastasis (Lin et al, 2015) or leptomeningeal metastases as defined by EANO-ESMO criteria (Le Rhun et al, 2017). ORR will be assessed at 6 months after the first treatment dose of 131I-omburtamab
3. ORR according to CSF cytology. Response is defined as a complete response when CSF converts from positive at baseline to negative after treatment with 131I-omburtamab
4. CNS/LM progression will be assessed at 6 months after the first treatment dose of 131Iomburtamab. Criteria from the Response assessment in Neuro-Oncology (RANO) group will be used to assess the CNS progression (Lin et al, 2015) while EANO-ESMO criteria will be used to assess LM progression (Le Rhun et al, 2017)
5. Whole-body, organ, blood, and CSF radiation dosimetry.
6. Pharmacokinetic analysis of activity in blood and CSF including derivation of best-fit uptake and/or clearance parameters (half-times, maximum value) of time-activity concentration curves and of I-131 residence times (i.e., cumulated activity) concentrations.
7. The frequency, type, and duration of treatment-emergent severe adverse events and serious adverse events, including clinically significant laboratory abnormalities. All adverse events will be graded according to Common Terminology Criteria for Adverse Events (CTCAE), version 4.0.
8. Performance assessment to monitor gross changes in neurological function is performed at week 26 and subsequently every 6 months during trial period.

Key inclusion & exclusion criteria

• Age minimum: >=
• Age maximum: < 18age old
• Gender: Both

Include criteria

1. Patients must have a histologically confirmed diagnosis of neuroblastoma with relapse in the central nervous system or in the meninges (leptomeningeal).
2. Patients must be between the ages of birth and 18 years at the time of screening.
3. Patients must have a life expectancy of at least 3 months.

Exclude criteria

1. Patients with primary neuroblastoma in central nervous system.
2. Patients must not have an uncontrolled life-threatening infection.
3. Patients must not have received cranial or spinal irradiation less than 3 weeks prior to first dose of 131I-omburtamab in this trial.
4. Patients must not have received systemic chemotherapy (corticosteroids not included) less than 3 weeks prior to enrollment in this trial.
5. Patients must not have severe major non-hematologic organ toxicity; specifically, any renal, cardiac, hepatic, pulmonary, and gastrointestinal system toxicity must fall below Grade 3 prior to enrollment in this trial. Patients with stable neurological deficits (due to brain tumor) are not excluded. Patients with Grade 3 or lower hearing loss are not excluded.

Related Information

• Primary Sponsor: Tomita Koji