IR-101 for relapsed or refractory neuroblastoma

A Study to Evaluate Safety and Efficacy of Radiopharmaceutical IR-101 in Patients With Relapsed or Refractory Neuroblastoma

This trial will test whether IR-101, a targeted radiopharmaceutical, can safely treat children (age ≥12 months) and adolescents with relapsed or refractory high-risk neuroblastoma that shows MIBG uptake.

Quick facts

• Phase: Early Phase 1
• Study type: Interventional
• Enrollment: 10 (estimated)
• Ages: 12 Months and up
• Sex: All
• Sponsor: Sichuan University (Academic / other)
• Drugs / interventions: immunotherapy
• Locations: 2 sites (Chengdu, Sichuang and 1 other locations)
• Trial ID: NCT07067346 on ClinicalTrials.gov

What this trial studies

This early Phase 1 interventional trial gives participants IR-101, a radiopharmaceutical targeted to MIBG-avid neuroblastoma lesions, to characterize safety and preliminary efficacy. Eligible patients must have relapsed or refractory high-risk neuroblastoma with all soft-tissue lesions showing MIBG uptake, be at least 12 months old, have Lansky performance ≥50%, and meet organ function and blood-count requirements. Key exclusions include recent antibody immunotherapy, recent [131I]MIBG or Lu-177 radionuclide therapy, recent autologous or allogeneic transplant within protocol windows, active GVHD, or very recent radiotherapy. The trial is conducted at West China Hospital in Chengdu and will follow participants through treatment and post-treatment safety and response assessments.

Who should consider this trial

Good fit: Children and adolescents (≥12 months) with relapsed or refractory high-risk neuroblastoma that is clearly MIBG-avid, who have adequate organ function and a Lansky score of at least 50%, are ideal candidates.

Not a fit: Patients without MIBG-avid disease, those recently treated with [131I]MIBG or Lu-177 radionuclide therapy, those too soon after transplant, or those with active GVHD or poor organ function are unlikely to qualify or derive benefit.

Why it matters

Potential benefit: If successful, IR-101 could provide a new targeted option that delivers radiation directly to MIBG-avid tumor sites and potentially shrink tumors in patients who have exhausted other therapies.

How similar studies have performed: Related radiopharmaceutical approaches such as [131I]MIBG and Lu-177 targeted therapies have shown activity in relapsed neuroblastoma, but IR-101 appears to be a new agent that has not been broadly tested yet.

Eligibility criteria

Inclusion Criteria:

1. Relapsed or refractory high-risk neuroblastoma with MIBG avid lesions in accordance with the Revised International Neuroblastoma Response Criteria (INRC);
2. All soft-tissue lesions detected on CT/MRI must be MIBG-avid;
3. Age ≥12 months;
4. Lansky performance status ≥50%;
5. Adequate organ function and hematologic parameters;

Exclusion Criteria:

1. Antibody-based immunotherapy within 5 half-lives or 30 days (whichever is shorter), or not yet recovered from adverse reactions of prior biologic therapies；
2. Treatment with \[¹³¹I\]MIBG or Lu-177 targeted radionuclide therapy \<3 months of last administration;
3. Autologous transplant \<12 weeks, or Allogeneic transplant \<4 months (patients \>4 months post-transplant must be free of active GVHD);
4. Radiotherapy within 2 weeks prior to first dose of study drug (However, if the subjects undergone radiotherapy for the sole lesion have MIBG uptake after 2 weeks of radiotherapy completion, they will be eligible); or Extensive-field radiotherapy (e.g., Cranio-spinal cord, whole lung, whole abdomen, or \>50% bone marrow) within 12 weeks prior to first dose of study drug;
5. Renal Insufficiency;
6. Active Infections;

Where this trial is running

Chengdu, Sichuang and 1 other locations

• West China Hospital — Chengdu, Sichuang, China (Recruiting)
• West China Hospital — Chengdu, China (Recruiting)

Study contacts

• Principal investigator: Rong Tian, MD — West China Hospital
• Study coordinator: Rong Tian, MD

Conditions Neuroblastoma; Radiopharmaceutical