NCT05734066 · Jazz Pharmaceuticals
Study of Lurbinectedin Monotherapy in Pediatric and Young Adult Participants With Relapsed/Refractory Ewing Sarcoma
What this study is about
This study is conducted in two phases. The phase 1 portion of the study evaluates the safety, tolerability, how the drug moves through the body (PK), recommended phase 2 dose (RP2D), and effectiveness of lurbinectedin treatment given alone in pediatric participants with previously treated solid tumors.
View original scientific description
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.
Interventions
DRUG
Lurbinectedin
Administered as intravenous (IV) infusion once every 3 weeks (Q3W)
Primary outcome measures
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.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 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 following laboratory values:
- Aspartate aminotransferase (AST), alanine aminotransferase (ALT) ≤ 2.5 × upper limit of normal (ULN).
- Total bilirubin ≤ 1.5 × institutional ULN (with the exception of participants with Gilbert's syndrome who must have bilirubin \< 3 × institutional ULN).
- The participant has adequate bone marrow function, evidenced by the following:
- Absolute neutrophil
Where
- Los Angeles, California
- Palo Alto, California
- Washington D.C., District of Columbia
- St. Petersburg, Florida
- Atlanta, Georgia
- Baltimore, Maryland
- Grand Rapids, Michigan
- New York, New York
- Cincinnati, Ohio
- Columbus, Ohio
- Philadelphia, Pennsylvania
- Memphis, Tennessee
And 2 more locations — see the full list below.
Collaborators
Jazz Pharmaceuticals Ireland Limited
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Feb 3, 2026 · Source of record for eligibility and locations