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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
60
Administered as intravenous (IV) infusion once every 3 weeks (Q3W)
Children's Hospital of Los Angeles
Los Angeles, California, United States
RECRUITINGLucile Packard Children's Hospital
Palo Alto, California, United States
RECRUITINGChildren's National Hospital
Washington D.C., District of Columbia, United States
RECRUITINGJohns Hopkins All Children's Hospital
St. Petersburg, Florida, United States
RECRUITINGChildren's Healthcare of Atlanta at Arthur M. Blank Hospital
Atlanta, Georgia, United States
RECRUITINGJohns Hopkins University
Baltimore, Maryland, United States
RECRUITINGCorewell Health
Grand Rapids, Michigan, United States
RECRUITINGMemorial Sloan Kettering Cancer Center
New York, New York, United States
RECRUITINGCincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
RECRUITINGNationwide Children's Hospital
Columbus, Ohio, United States
RECRUITING...and 5 more locations
Phase 1: Number of Participants Experiencing Dose-limiting Toxicities (DLTs)
Time frame: From the first dose through end of Cycle 1 (21 days).
Phase 1: Number of Participants Experiencing Serious Adverse Events (SAEs) and Treatment Emergent Adverse Events (TEAEs)
Time frame: Post-baseline (Day 1) up to approximately 31 months.
Phase 1: Number of Participants With Dose Modifications
Time frame: Post-baseline (Day 1) up to approximately 31 months.
Phase 1: Number of Participants Who Discontinued Study Intervention Due to TEAEs
Time frame: Post-baseline (Day 1) up to approximately 31 months.
Phase 2: Objective Response Rate (ORR) Based on Investigator Assessment (IA)
Time frame: Day -28 up to a total of 13 months postdose.
Phase 1: Plasma Concentration of Lurbinectedin
Time frame: Day 1: Predose up to approximately 168 hours postdose; Days 16, 31, 46: Predose up to approximately 5 minutes postdose.
Phase 1: Objective Response Rate (ORR) Based on Investigator Assessment (IA)
Time frame: Day -28 up to a total of 31 months postdose.
Phase 1: Progression-Free Survival (PFS) Based on Investigator Assessment (IA)
Time frame: Day -28 up to a total of 31 months postdose.
Phase 1: Duration of Response (DOR) in Participants with Confirmed Complete Response (CR) or Partial Response (PR)
Time frame: Day -28 up to a total of 31 months postdose.
Phase 1: Disease Control Rate (DCR)
Time frame: Day -28 up to a total of 31 months postdose.
Phase 1: Clinical Benefit Rate (CBR) With Stable Disease (SD) for At Least 12 Weeks
Time frame: Day -28 up to a total of 31 months postdose.
Phase 1: Overall Survival (OS)
Time frame: Post-baseline (Day 1) up to 31 months postdose.
Phase 1: Change From Baseline in Respiratory Rate
Time frame: Post-baseline (Day 1) up to approximately 31 months.
Phase 1: Change From Baseline in Pulse Rate
Time frame: Post-baseline (Day 1) up to approximately 31 months.
Phase 1: Change From Baseline in Blood Pressure
Time frame: Post-baseline (Day 1) up to approximately 31 months.
Phase 1: Change From Baseline in Weight
Time frame: Post-baseline (Day 1) up to approximately 31 months.
Phase 1: Change from Baseline in Platelet Count
Time frame: Post-baseline (Day 1) up to approximately 31 months.
Phase 1: Change from Baseline in Red Blood Cell Count
Time frame: Post-baseline (Day 1) up to approximately 31 months.
Phase 1: Change from Baseline in Hemoglobin
Time frame: Post-baseline (Day 1) up to approximately 31 months.
Phase 1: Change from Baseline in Differential White Blood Cell Count
Time frame: Post-baseline (Day 1) up to approximately 31 months.
Phase 1: Change From Baseline in AST/ALT Levels
Time frame: Post-baseline (Day 1) up to approximately 31 months.
Phase 1: Change From Baseline in Creatinine Levels
Time frame: Post-baseline (Day 1) up to approximately 31 months.
Phase 1: Change From Baseline in CPK Levels
Time frame: Post-baseline (Day 1) up to approximately 31 months.
Phase 2: Plasma Concentration of Lurbinectedin
Time frame: Day 1: Predose up to approximately 168 hours postdose; Days 16, 31, 46: Predose up to approximately 5 minutes postdose.
Phase 2: Number of Participants Experiencing Serious Adverse Events (SAEs) and Treatment-emergent Adverse Events (TEAEs)
Time frame: Post-baseline (Day 1) up to approximately 13 months.
Phase 2: Number of Participants With Dose Modifications
Time frame: Post-baseline (Day 1) up to approximately 13 months.
Phase 2: Number of Participants Who Discontinued Study Intervention Due to TEAEs
Time frame: Post-baseline (Day 1) up to approximately 13 months.
Phase 2: Progression-Free Survival (PFS) Based on IA
Time frame: Day -28 up to a total of 13 months postdose.
Phase 2: Duration of Response (DOR) in Participants with Confirmed Complete Response (CR) or Partial Response (PR)
Time frame: Day -28 up to a total of 13 months postdose.
Phase 2: Disease Control Rate (DCR)
Time frame: Day -28 up to a total of 13 months postdose.
Phase 2: Clinical Benefit Rate (CBR) With Stable Disease (SD) for At Least 12 Weeks
Time frame: Day -28 up to a total of 13 months postdose.
Phase 2: Overall Survival (OS)
Time frame: Post-baseline (Day 1) up to 13 months postdose.
Phase 2: Change From Baseline in Respiratory Rate
Time frame: Post-baseline (Day 1) up to approximately 13 months.
Phase 2: Change From Baseline in Pulse Rate
Time frame: Post-baseline (Day 1) up to approximately 13 months.
Phase 2: Change From Baseline in Blood Pressure
Time frame: Post-baseline (Day 1) up to approximately 13 months.
Phase 2: Change From Baseline in Weight
Time frame: Post-baseline (Day 1) up to approximately 13 months.
Phase 2: Change From Baseline in Platelet Count
Time frame: Post-baseline (Day 1) up to approximately 13 months.
Phase 2: Change From Baseline in Red Blood Cell Count
Time frame: Post-baseline (Day 1) up to approximately 13 months.
Phase 2: Change From Baseline in Hemoglobin
Time frame: Post-baseline (Day 1) up to approximately 13 months.
Phase 2: Change From Baseline in Differential White Blood Cell Count
Time frame: Post-baseline (Day 1) up to approximately 13 months.
Phase 2: Change From Baseline in AST/ALT Levels
Time frame: Post-baseline (Day 1) up to approximately 13 months.
Phase 2: Change From Baseline in Creatinine Levels
Time frame: Post-baseline (Day 1) up to approximately 13 months.
Phase 2: Change From Baseline in CPK Levels
Time frame: Post-baseline (Day 1) up to approximately 13 months.
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