Thinking of joining a study?

Register your interest

Become a volunteer?

NCT05734066 | RECRUITING | Refractory Ewing Sarcoma

Study of Lurbinectedin Monotherapy in Pediatric and Young Adult Participants With Relapsed/Refractory Ewing Sarcoma
Sponsor:

Jazz Pharmaceuticals

Brief 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.

Condition or disease

Refractory Ewing Sarcoma

Relapsed Ewing Sarcoma

Ewing Sarcoma

Intervention/treatment

Lurbinectedin

Phase

PHASE1

PHASE2

Study Type : INTERVENTIONAL
Estimated Enrollment : 60 participants
Masking : NONE
Primary Purpose : TREATMENT
Official Title : 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.
Actual Study Start Date : 2023-05-23
Estimated Primary Completion Date : 2026-12-01
Estimated Study Completion Date : 2027-06-01

Information not available for Arms and Intervention/treatment

Ages Eligible for Study: 2 Years to 30 Years
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers:
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 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 count (ANC) ≥ 1.0 × 109/L (independent of growth factor support within 1 week of screening laboratories).
        • * Platelets ≥ 100 × 109/L (without platelet transfusion within previous 7 days of screening laboratories).
        • * Hemoglobin ≥ 8 g/dL (note: may have been transfused).
        • * The participant has an adequate renal function
          • * Calculated creatinine clearance (use Cockcroft-Gault formula for participants ≥ 18 years; Schwartz equation for participants \< 18 years) ≥ 60 mL/min.
          • * The participant has an adequate cardiac function
            • * Left ventricular ejection fraction or shortening fraction per institutional norm ≥ institutional lower level of normal.
            • * The participant has creatine phosphokinase ≤ 2.5 × institutional ULN.
            • Weight
            • * The participant has body weight ≥ 15 kg.
            • Sex and Contraceptive/Barrier Requirements
            • Male participants
              • Male participants are eligible to participate if they agree to the following during the study intervention period and for at least 4 months after the last dose of study intervention
                • * Refrain from donating sperm.
                • PLUS, either
                  • * Be abstinent from heterosexual intercourse as their preferred and usual lifestyle (abstinent on a long-term and persistent basis) and agree to remain abstinent.
                  • OR
                  • * Must agree to use contraception/barrier as detailed below
                    • * Agree to use a male condom with female partner and use of an additional highly effective contraceptive method with a failure rate of \< 1% per year when having sexual intercourse with a Woman of childbearing potential (WOCBP) who is not currently pregnant.
                    • * Note: male participants who are azoospermic (vasectomized or due to a medical cause) are still required to follow the protocol-specified contraception/barrier criteria.
                    • Female participants
                      • A female participant is eligible to participate if she is not pregnant or breastfeeding, and one of the following conditions applies
                        • * Is a Woman of nonchildbearing potential (WONCBP). OR
                        • * Is a WOCBP and using an acceptable contraceptive method during the study intervention period (at least 7 months after the last dose of study intervention). The investigator should evaluate the potential for contraceptive method failure (eg, noncompliance, recently initiated) in relationship to the first dose of study intervention.
                        • * A WOCBP must have a negative highly sensitive pregnancy test (urine or serum as required by local regulations) within 7 days before the first dose of study intervention.
                        • * If a urine test cannot be confirmed as negative (eg, an ambiguous result), a serum pregnancy test is required. In such cases, the participant must be excluded from participation if the serum pregnancy result is positive.
                        • * Additional requirements for pregnancy testing during and after study intervention.
                        • * The investigator is responsible for review of medical history, menstrual history, and recent sexual activity to decrease the risk for inclusion of a woman with an early undetected pregnancy.
                        • Informed Consent
                        • * Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.
                        • Key Exclusion Criteria
                          • Medical Conditions
                          • * corrected QT interval (QTc) prolongation defined as a QTc ≥ 470 ms using the Bazett formula.
                          • * Known symptomatic Central nervous system (CNS) metastases requiring steroids. Participants with previously diagnosed CNS metastases are eligible if they have completed their treatment and have recovered from the acute effects of radiation therapy or surgery prior to enrollment, have discontinued high dose steroid treatment for these metastases for at least 2 weeks, and are neurologically stable (physiologic doses of steroids and short courses of steroids for other indications are acceptable).
                          • * Persisting toxicity related to prior therapy; however, alopecia, sensory neuropathy, hypothyroidism, and rash Grade ≤ 2 are acceptable, and other Grade ≤ 2 adverse events (AEs) not constituting a safety risk based on the investigator's judgement are acceptable.
                          • * An uncontrolled intercurrent illness including but not limited to ongoing or active infection requiring antibiotic, antifungal, or antiviral therapy, symptomatic heart failure, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
                          • * Any other major illness that, in the investigator's judgment, could substantially increase the risk associated with participation in this study.
                          • * Any other diseases, metabolic dysfunction, physical examination finding, or clinical laboratory finding giving reasonable suspicion of a disease or condition that contraindicates the use of an investigational drug or that may affect the interpretation of the results or render the participant at high-risk for treatment complications.
                          • Prior/Concomitant Therapy
                          • * Received prior treatment with lurbinectedin or trabectedin.
                          • * Received prior treatment with any investigational product within 4 weeks of first infusion of study intervention. Observational studies are permitted.
                          • * Received live or live attenuated vaccines within 4 weeks of the first dose of study treatment or plans to receive live vaccines during study participation. Administration of inactive vaccines or messenger ribonucleic acid (mRNA) vaccines (for example, inactivated influenza vaccines or COVID-19 vaccines) are allowed.
                          • * Had major surgery ≤ 4 weeks or radiation therapy ≤ 2 weeks prior to enrollment unless fully recovered. Prior palliative radiotherapy is permitted, provided it was completed at least 2 weeks prior to participant enrollment.
                          • * Received prior allogeneic bone marrow transplantation or solid organ transplant.
                          • * Received chemotherapy ≤ 3 weeks prior to start of study intervention.
                          • Diagnostic Assessments
                          • * Hepatitis B virus (HBV) or Hepatitis C virus (HCV) infection at screening (positive HBV surface antigen or Polymerase chain reaction (PCR) test for HCV RNA if HCV antibody test is positive).
                          • * Human immunodeficiency infection at screening (positive anti-HIV antibody).
                          • Other Exclusions
                          • * Has a known or suspected hypersensitivity to any of the components of the study intervention.
                          • * The participant or parent(s)/guardian(s) is/are unable to comply with the study visit schedule and other protocol requirements, in the opinion of the investigator
Study of Lurbinectedin Monotherapy in Pediatric and Young Adult Participants With Relapsed/Refractory Ewing Sarcoma

Location Details

NCT05734066

Please Choose a site

How to Participate

Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.

Locations

RECRUITING

United States, California

Children's Hospital Los Angeles

Los Angeles, California, United States, 90027

RECRUITING

United States, California

Stanford Children's Health

Palo Alto, California, United States, 94304

RECRUITING

United States, District of Columbia

Childrens National Hospital Michigan

Washington, District of Columbia, United States, 20010

RECRUITING

United States, Florida

Johns Hopkins All Children's Hospital

Saint Petersburg, Florida, United States, 33701

RECRUITING

United States, Georgia

Arthur M. Blank Hospital Children's Healthcare of Atlanta

Atlanta, Georgia, United States, 30329

RECRUITING

United States, Maryland

Johns Hopkins University

Baltimore, Maryland, United States, 21238

RECRUITING

United States, Road cancer

Corewell Health

Grand Rapids, Road cancer, United States, 49503

RECRUITING

United States, New York

Memorial Sloan Kettering

New York, New York, United States, 10065

RECRUITING

United States, Ohio

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States, 45229

RECRUITING

United States, Ohio

Nationwide Childrens

Columbus, Ohio, United States, 43205

RECRUITING

United States, Pennsylvania

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States, 19104

RECRUITING

United States, Tennessee

St. Jude Children's Research Hospital

Memphis, Tennessee, United States, 38105

RECRUITING

United States, Texas

UT Southwestern in Texas

Dallas, Texas, United States, 75390

RECRUITING

United States, Texas

MD Anderson Cancer Center

Houston, Texas, United States, 77030

RECRUITING

Canada, Ontario

The Hospital for Sick Children

Toronto, Ontario, Canada, M5G 1X8