Refractory Ewing SarcomaPhase 1/2NCT05734066
Study of Lurbinectedin Monotherapy in Pediatric and Young Adult Participants With Relapsed/Refractory Ewing Sarcoma
A Phase 1/2 study testing Lurbinectedin in people with Refractory Ewing Sarcoma and Relapsed Ewing Sarcoma, run by Jazz Pharmaceuticals at 16 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 to 30 years
- Conditions the study is for
- Refractory Ewing Sarcoma, Relapsed Ewing Sarcoma, Ewing Sarcoma
- What is being tested?
- Lurbinectedin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 16 sitesin 2 countries
- Study dates
- 23 May 2023to 20 Apr 2028
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Show all 42Show fewer
- 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 × 10^9/L (independent of growth factor support within 1 week of screening laboratories).
- Platelets ≥ 100 × 10^9/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.
Who cannot join
- Medical Conditions
- corrected QT interval (QTc) prolongation defined as a QTc ≥ 460 ms using the Bazett formula in age < 18 years and QTc ≥ 470 ms using the Bazett formula in age ≥ 18 years.
- 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.
Show all 20Show fewer
- 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
Trial locations and contacts
16 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
16 sites in 2 countries
United States15 sites, 15 recruiting
- Recruiting
Children's Healthcare of Atlanta at Arthur M. Blank Hospital
Atlanta, Georgia, 30329
- Recruiting
Johns Hopkins University
Baltimore, Maryland, 21238
- Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
- Recruiting
Nationwide Children's Hospital
Columbus, Ohio, 43205
- Recruiting
Children's Health Dallas
Dallas, Texas, 75235
- Recruiting
Duke University School of Medicine
Durham, North Carolina, 27710
- Recruiting
Corewell Health
Grand Rapids, Michigan, 49503
- Recruiting
The University of Texas MD Anderson Cancer Center
Houston, Texas, 77030
- Recruiting
Children's Hospital of Los Angeles
Los Angeles, California, 90027
- Recruiting
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
- Recruiting
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
- Recruiting
Lucile Packard Children's Hospital
Palo Alto, California, 94304
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
- Recruiting
Johns Hopkins All Children's Hospital
St. Petersburg, Florida, 33701
- Recruiting
Children's National Hospital
Washington D.C., District of Columbia, 20010
Canada1 site, 1 recruiting
- Recruiting
The Hospital for Sick Children
Toronto, Ontario, M5G 1X8
About this study
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.
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.
- Being tested
- Lurbinectedin
- Keywords
- Solid Tumors, lurbinectedin, ewing's sarcoma, soft tissue sarcoma, sarcomas
- Medical subject headings
- Sarcoma, Ewing, Sarcoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LurbinectedinDrug
Also called JZP712
Administered as intravenous (IV) infusion once every 3 weeks (Q3W)
| Study group | Role | What they receive |
|---|---|---|
| Phase 1 Part 1: Dose Selection | Experimental | Pediatric participants ≥ 2 to < 18 years of age with previously treated solid tumors of any histology at 5 dose levels to determine the RP2D, followed by a safety expansion cohort.
Participants aged ≥ 6 to < 18 years will be enrolled at the starting dose of 3.2 mg/m^2 lurbinectedin. If, after review, the starting dose of 3.2 mg/m^2 lurbinectedin Q3W is deemed safe in participants aged ≥ 6 to < 18 years, participants aged ≥ 2 to < 6 years may enroll and start at the dose as determined by the DMC. After this, the study opens to all participants (aged ≥ 2 to < 18 years) for all dose levels.
Upon completion of the cohort at all dose levels, participants may be eligible to enroll in a safety expansion cohort. Receives: Drug: Lurbinectedin |
| Phase 1 Part 2: RP2D | Experimental | Participants aged ≥ 2 to ≤ 30 years with recurrent/refractory Ewing sarcoma at the RP2D to assess safety and efficacy signals. Receives: Drug: Lurbinectedin |
| Phase 2 | Experimental | If a signal of efficacy is observed in Phase 1 Part 2, additional participants aged ≥ 2 to ≤ 30 years with recurrent/refractory Ewing sarcoma will be enrolled. Phase 2 will further assess the safety and efficacy of lurbinectedin monotherapy. Receives: Drug: Lurbinectedin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Phase 1: Number of Participants Experiencing Dose-limiting Toxicities (DLTs)
Measured: 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)
Measured: Post-baseline (Day 1) up to approximately 31 months.
Phase 1: Number of Participants With Dose Modifications
Measured: Post-baseline (Day 1) up to approximately 31 months.
Phase 1: Number of Participants Who Discontinued Study Intervention Due to TEAEs
Measured: Post-baseline (Day 1) up to approximately 31 months.
Phase 2: Objective Response Rate (ORR) Based on Investigator Assessment (IA)
Measured: Day -28 up to a total of 13 months postdose.
Secondary outcomes (38)
Phase 1: Plasma Concentration of Lurbinectedin
Measured: 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)
Measured: Day -28 up to a total of 31 months postdose.
Phase 1: Progression-Free Survival (PFS) Based on Investigator Assessment (IA)
Measured: 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)
Measured: Day -28 up to a total of 31 months postdose.
Phase 1: Disease Control Rate (DCR)
Measured: 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
Measured: Day -28 up to a total of 31 months postdose.
Phase 1: Overall Survival (OS)
Measured: Post-baseline (Day 1) up to 31 months postdose.
Phase 1: Change From Baseline in Respiratory Rate
Measured: Post-baseline (Day 1) up to approximately 31 months.
Phase 1: Change From Baseline in Pulse Rate
Measured: Post-baseline (Day 1) up to approximately 31 months.
Phase 1: Change From Baseline in Blood Pressure
Measured: Post-baseline (Day 1) up to approximately 31 months.
Phase 1: Change From Baseline in Weight
Measured: Post-baseline (Day 1) up to approximately 31 months.
Phase 1: Change from Baseline in Platelet Count
Measured: Post-baseline (Day 1) up to approximately 31 months.
Phase 1: Change from Baseline in Red Blood Cell Count
Measured: Post-baseline (Day 1) up to approximately 31 months.
Phase 1: Change from Baseline in Hemoglobin
Measured: Post-baseline (Day 1) up to approximately 31 months.
Phase 1: Change from Baseline in Differential White Blood Cell Count
Measured: Post-baseline (Day 1) up to approximately 31 months.
Phase 1: Change From Baseline in AST/ALT Levels
Measured: Post-baseline (Day 1) up to approximately 31 months.
Phase 1: Change From Baseline in Creatinine Levels
Measured: Post-baseline (Day 1) up to approximately 31 months.
Phase 1: Change From Baseline in CPK Levels
Measured: Post-baseline (Day 1) up to approximately 31 months.
Phase 2: Plasma Concentration of Lurbinectedin
Measured: 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)
Measured: Post-baseline (Day 1) up to approximately 13 months.
Phase 2: Number of Participants With Dose Modifications
Measured: Post-baseline (Day 1) up to approximately 13 months.
Phase 2: Number of Participants Who Discontinued Study Intervention Due to TEAEs
Measured: Post-baseline (Day 1) up to approximately 13 months.
Phase 2: Progression-Free Survival (PFS) Based on IA
Measured: 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)
Measured: Day -28 up to a total of 13 months postdose.
Phase 2: Disease Control Rate (DCR)
Measured: 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
Measured: Day -28 up to a total of 13 months postdose.
Phase 2: Overall Survival (OS)
Measured: Post-baseline (Day 1) up to 13 months postdose.
Phase 2: Change From Baseline in Respiratory Rate
Measured: Post-baseline (Day 1) up to approximately 13 months.
Phase 2: Change From Baseline in Pulse Rate
Measured: Post-baseline (Day 1) up to approximately 13 months.
Phase 2: Change From Baseline in Blood Pressure
Measured: Post-baseline (Day 1) up to approximately 13 months.
Phase 2: Change From Baseline in Weight
Measured: Post-baseline (Day 1) up to approximately 13 months.
Phase 2: Change From Baseline in Platelet Count
Measured: Post-baseline (Day 1) up to approximately 13 months.
Phase 2: Change From Baseline in Red Blood Cell Count
Measured: Post-baseline (Day 1) up to approximately 13 months.
Phase 2: Change From Baseline in Hemoglobin
Measured: Post-baseline (Day 1) up to approximately 13 months.
Phase 2: Change From Baseline in Differential White Blood Cell Count
Measured: Post-baseline (Day 1) up to approximately 13 months.
Phase 2: Change From Baseline in AST/ALT Levels
Measured: Post-baseline (Day 1) up to approximately 13 months.
Phase 2: Change From Baseline in Creatinine Levels
Measured: Post-baseline (Day 1) up to approximately 13 months.
Phase 2: Change From Baseline in CPK Levels
Measured: Post-baseline (Day 1) up to approximately 13 months.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Feb 2023
- Study start23 May 2023
- Primary completion28 Jan 2028 (estimated)
- Study completion20 Apr 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Refractory Ewing SarcomaStudies of the same condition, open and under way.
- More trials run by Jazz PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Jazz Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Jazz Pharmaceuticals Ireland Limited
- Responsible party
- Sponsor
- Investigators
- Jazz Study Director, Study director, Jazz Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05734066
- Sponsor's protocol ID
- JZP712-101
- Status last verified
- Nov 2026
- First submitted
- 2 Feb 2023
- Last updated
- 5 Nov 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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.
- ClinicalTrials.gov ID
- NCT05734066
- Acronym / sponsor ID
- JZP712-101
- Keywords
- Solid Tumors, lurbinectedin, ewing's sarcoma, soft tissue sarcoma, sarcomas
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Jazz PharmaceuticalsIndustry (pharma, biotech, devices)
- Collaborators
- Jazz Pharmaceuticals Ireland Limited
- Study start
- 23 May 2023 (actual)
- Primary completion
- 28 Jan 2028 (estimated)
- Study completion
- 20 Apr 2028 (estimated)
- First posted
- 17 Feb 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Phase 1 Part 1: Dose Selection | Experimental | Drug: LurbinectedinPediatric participants ≥ 2 to < 18 years of age with previously treated solid tumors of any histology at 5 dose levels to determine the RP2D, followed by a safety expansion cohort. Participants aged ≥ 6 to < 18 years will be enrolled at the starting dose of 3.2 mg/m^2 lurbinectedin. If, after review, the starting dose of 3.2 mg/m^2 lurbinectedin Q3W is deemed safe in participants aged ≥ 6 to < 18 years, participants aged ≥ 2 to < 6 years may enroll and start at the dose as determined by the DMC. After this, the study opens to all participants (aged ≥ 2 to < 18 years) for all dose levels. Upon completion of the cohort at all dose levels, participants may be eligible to enroll in a safety expansion cohort. |
| Phase 1 Part 2: RP2D | Experimental | Drug: LurbinectedinParticipants aged ≥ 2 to ≤ 30 years with recurrent/refractory Ewing sarcoma at the RP2D to assess safety and efficacy signals. |
| Phase 2 | Experimental | Drug: LurbinectedinIf a signal of efficacy is observed in Phase 1 Part 2, additional participants aged ≥ 2 to ≤ 30 years with recurrent/refractory Ewing sarcoma will be enrolled. Phase 2 will further assess the safety and efficacy of lurbinectedin monotherapy. |
Who may qualify
- Ages
- 2 to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (42 listed)
- Age
- Participant must meet the following age requirements at the time the informed consent form (ICF) (and assent form, if applicable) is signed:
- Type of Participant and Disease Characteristics
- Participant has a confirmed solid tumor
- The participant has a Lansky/Karnofsky performance status score of ≥ 50%.
Key exclusion criteria (20 listed)
- Medical Conditions
- corrected QT interval (QTc) prolongation defined as a QTc ≥ 460 ms using the Bazett formula in age < 18 years and QTc ≥ 470 ms using the Bazett formula in age ≥ 18 years.
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LurbinectedinAlso: JZP712Administered as intravenous (IV) infusion once every 3 weeks (Q3W) | Drug | Phase 1 Part 1: Dose Selection, Phase 1 Part 2: RP2D, Phase 2 |
Primary outcome
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.
38 secondary outcomes
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.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Nov 2026.
- Individual participant dataSharing planned: No
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Tipifarnib for the Treatment of Advanced Solid Tumors, Lymphoma, or Histiocytic Disorders With HRAS Gene Alterations, a Pediatric MATCH Treatment Trial
Malignant Solid Neoplasm, Recurrent Adrenal Gland Pheochromocytoma
Active, not recruitingPhase 2
Selpercatinib for the Treatment of Advanced Solid Tumors, Lymphomas, or Histiocytic Disorders With Activating RET Gene Alterations, a Pediatric MATCH Treatment Trial
Hematopoietic and Lymphatic System Neoplasm, Recurrent Ependymoma
RecruitingPhase 1
Phase 1 Dose-escalating Study of MM-398 (Irinotecan Sucrosofate Liposome Injection) Plus Intravenous Cyclophosphamide in Recurrent or Refractory Pediatric Solid Tumors
Recurrent or Refractory Solid Tumors, Ewing Sarcoma
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Jazz Study Director study director
Jazz Pharmaceuticals
16 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Lurbinectedin in people with Refractory Ewing Sarcoma and Relapsed Ewing Sarcoma, run by Jazz Pharmaceuticals at 16 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 42 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 5 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Clinical Trial Disclosure & Transparency (215-832-3750, ClinicalTrialDisclosure@JazzPharma.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Phase 1: Number of Participants Experiencing Dose-limiting Toxicities (DLTs) (time frame: From the first dose through end of Cycle 1 (21 days).). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Nov 2026; AzTrials last refreshed its copy on 8 Nov 2026.