Title: Phase II Study Investigating the Efficacy of Neoadjuvant Dual Checkpoint Inhibition and Cryoablation Therapy in Children, Adolescents, and Young Adults With Relapsed/Refractory Solid Tumors
Principal Investigator: Marie Nelson, MD, Children’s National Research Institute, Washington, DC
Description: Researchers are conducting a phase 2, single-arm, multisite trial to evaluate the combination of cryoablation therapy and dual checkpoint inhibition with nivolumab and ipilimumab in pediatric and young adult patients with relapsed or refractory solid tumors.
Patients with osteosarcoma, Ewing sarcoma, rhabdomyosarcoma, melanoma, hepatoblastoma, hepatocellular carcinoma, neuroblastoma, Wilms tumor, and relapsed or refractory pediatric solid tumors are being enrolled.
Patients with at least 2 sites of disease will receive the current pediatric recommended phase 2 dose of nivolumab and ipilimumab for 1 cycle and undergo cryoablation at 1 tumor site. Patients will continue to receive nivolumab and ipilimumab until disease progression, unacceptable toxicity, or the completion of 13 cycles.
The primary outcome measures of the trial are disease response (measured with consistent imaging utilizing RECIST v1.1) and the incidence and severity of treatment-related adverse events (measured by CTCAE v.5).
Patients must be 1 to 40 years of age and have histologically confirmed solid tumors at the time of original diagnosis or relapse. For a complete list of eligibility criteria, please see the reference.
This study is sponsored by Children’s National Research Institute in collaboration with Bristol-Myers Squibb.
Clinicaltrials.gov. Neoadjuvant dual checkpoint inhibition and cryoablation in relapsed/refractory pediatric solid tumors. NCT05302921. Accessed May 23, 2022.