A clinical trial in several sites where pediatric patients with Neuroblastoma are treated with injections of 131I-burtomab (the study drug). The tolerability and efficacy of the drug will be tested.

Phase 1

• Conditions: Treatment of pediatric neuroblastoma patients with CNS relapse as evidenced by CNS/LM metastases; MedDRA version: 20.0Level: PTClassification code 10073130Term: Central nervous system neuroblastomaSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps); Therapeutic area: Diseases [C] - Cancer [C04]

• Registration Number: EUCTR2017-001828-22-ES
• Lead Sponsor: Y-mAbs Therapeutics A/S

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2018-02-01
• Last Update Posted: 11 September 2023

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 32

Inclusion Criteria

1. Patients must have a histologically confirmed diagnosis of neuroblastmona with relapse in the CNS or LM.
2. Patients need to have progressed in CNS/LM through induction therapy or have relapsed in CNS/LM follow induction. CNS/LM progression/ relapse is defined as LM disease or metastatic deposits in the CNS parenchyma, (excluding skull bone-based metastases)
3. Stable systemic disease not requiring chemo/ immunotherapy as judged by the investigator
4. Ventriculoperitoneal (VP) shunt is allowed however should be closed during IMP infusion and for approximately 5 hours after treatment and then readjusted. Closure of VP shunt is done at the discretion of the treating physician and the VP shunt should at any time based on patient safety evaluation be re-opened- at the assessment of the treating physician.
5. Patients must be between the ages of birth and 18 years at the time of screening
6. Patients must have a life expectancy of at least 3 months.
7. Acceptable hematological status, (hematological support is allowed if administered at least 2 weeks before administration of 131I-burtomab), defined as:
• Hemoglobin =8 g/dL
• While blood cell count =1000/µL
• Absolute neutrophil count =500/µL
• Platelet count =50,000/µL
8. Acceptable liver function defined as:
• Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) = 5 times upper limit of normal (UNL)
• Bilirubin =1.5 x UNL
9. Acceptable kidney function defined as:
• eGFR >60 mL/min/1.73 m2 calculated by the 2009 revised Bedside Schwartz Equation (Appendix 3).
10. Written informed consent from legal guardian(s) and/or child must be obtained in accordance with local regulations. Pediatric patients must provide assent as required by local regulations
Are the trial subjects under 18? yes
Number of subjects for this age range: 32
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Patients with primary neuroblastoma (NB) in CNS
2. Patients must not have obstructive or symptomatic communicating hydrocephalus.
3. Patients must not have worsening of neurologic function, according to assessment by investigator, within 3 weeks prior to first dose of 131I-burtomab.
4. Patients must not have an uncontrolled life-threatening infection.
5. Patients must not have received cranial or spinal irradiation less than 3 weeks prior to first dose of 131I-burtomab in this trial
6. Patients must not have received systemic chemo/ immunotherapy (corticosteroids not included) less than 3 weeks prior to enrolment in this trial.
7. Patients must not have received any B7-H3 treatment prior to enrolment in this trial.
8. 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 enrolment 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.
9. Patients must not be pregnant or breast-feeding
10. A woman of child-bearing potential who does not agree to use adequate contraception during treatment and for a period of 40 days after the last 131I-burtomab dose: intrauterine devices or hormonal contraceptives (contraceptive pills, implants, transdermal patches, hormonal vaginal devices, or injections with prolonged release). A sterilized or infertile woman is exempt from the requirement to use contraception after 131I-burtomab treatment: she must have undergone surgical sterilization (hysterectomy, or bilateral ovariectomy).

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified