A Phase 1/2, Open-label Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK), Recommended Phase 2 Dose (RP2D), and Efficacy of Lurbinectedin Monotherapy in Pediatric Participants With Previously Treated Solid Tumors Followed by Expansion to Assess Efficacy and Safety in Pediatric and Young Adult Participants With Relapsed/Refractory Ewing Sarcoma.

Jazz Pharmaceuticals

Summary

This study is conducted in two phases. The phase 1 portion of the study evaluates the safety, tolerability, pharmacokinetics (PK), recommended phase 2 dose (RP2D), and effectiveness of lurbinectedin monotherapy in pediatric participants with previously treated solid tumors. This is followed by the phase 2 portion, to further assess the effectiveness and safety in pediatric and young adult participants with recurrent/refractory Ewing sarcoma.

Eligibility

Age range: 2–30 years
Sex: All
Healthy volunteers: No

Detailed criteria

Key Inclusion Criteria: Age * Participant must meet the following age requirements at the time the informed consent form (ICF) (and assent form, if applicable) is signed: * Phase 1 Part 1: participants must be ≥ 2 to \< 18 years of age. * Phase 1 Part 2: participants must be ≥ 2 to ≤ 30 years of age. * Phase 2: participants must be ≥ 2 to ≤ 30 years of age. Type of Participant and Disease Characteristics * Participant has a confirmed solid tumor * The participant has a Lansky/Karnofsky performance status score of ≥ 50%. * The participant has adequate liver function, evidenced by the…

Interventions

Drug
Lurbinectedin
Administered as intravenous (IV) infusion once every 3 weeks (Q3W)

Locations (15)

Children's Hospital of Los Angeles
Los Angeles, California
Lucile Packard Children's Hospital
Palo Alto, California
Children's National Hospital
Washington D.C., District of Columbia
Johns Hopkins All Children's Hospital
St. Petersburg, Florida
Children's Healthcare of Atlanta at Arthur M. Blank Hospital
Atlanta, Georgia
Johns Hopkins University
Baltimore, Maryland