Comparing 3 approaches for refractory ewing sarcoma
Official title: Study of Lurbinectedin Monotherapy in Pediatric and Young Adult Participants With Relapsed/Refractory Ewing Sarcoma
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.
- Phase 1
- 3 groups
- One site, in Toronto
- Recruiting
Interventions
- Medication
Lurbinectedin
Administered as intravenous (IV) infusion once every 3 weeks (Q3W)
Canadian Sites (1)
1 of 1 recruiting
- Recruiting
The Hospital for Sick Children
Toronto, Ontario
Eligibility Criteria
See who this study is looking for59 criteria
The trial’s own eligibility text, word for word from the registry. Only the trial site can say who takes part.
Inclusion
- +Participant must meet the following age requirements at the time the informed consent formInformed consentThe process of being told what taking part involves, then choosing freely.Read more → (ICF) (and assent form, if applicable) is signed:
- +Phase 1Phase 1The earliest stage of human testing, in a small group, focused on safety.Read more → 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 2Phase 2A middle-stage study looking at what a treatment does and watching for side effects.Read more →: 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 × 10\^9/L (independent of growth factor support within 1 week of screeningScreeningThe checks done before joining, to see whether a study fits.Read more → 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.
- +The participant has body weight ≥ 15 kg.
- +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.
- +Female participants:
- +Is a Woman of nonchildbearing potential (WONCBP). OR
- +Informed Consent
- +Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in this protocolProtocolThe detailed plan a study must follow.Read more →.
- +Sex and Contraceptive/Barrier Requirements
- +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.
- +A female participant is eligible to participate if she is not pregnant or breastfeeding, and one of the following conditions applies:
- +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 investigatorPrincipal investigatorThe doctor or researcher responsible for running the study at a site.Read more → 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 inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more → of a woman with an early undetected pregnancy.
Exclusion
- −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.
- −Has a known or suspected hypersensitivity to any of the components of the study intervention.
- −Hepatitis B virus (HBV) or Hepatitis C virus (HCV) infection at screeningScreeningThe checks done before joining, to see whether a study fits.Read more → (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).
- −Medical Conditions
- −Persisting toxicity related to prior therapy; however, alopecia, sensory neuropathy, hypothyroidism, and rash Grade ≤ 2 are acceptable, and other Grade ≤ 2 adverse eventsAdverse eventAny medical problem that happens during a study, whether or not the treatment caused it.Read more → (AEs) not constituting a safety risk based on the investigatorPrincipal investigatorThe doctor or researcher responsible for running the study at a site.Read more →'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. ObservationalObservational studyA study that watches what happens without assigning any treatment.Read more → 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.
- −Received prior allogeneic bone marrow transplantation or solid organ transplant.
- −Diagnostic Assessments
- −Other Exclusions
- −The participant or parent(s)/guardian(s) is/are unable to comply with the study visit schedule and other protocolProtocolThe detailed plan a study must follow.Read more → requirements, in the opinion of the investigator
- −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 enrollmentEnrolmentThe number of participants a study plans to include, or has included.Read more →, 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).
- −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 chemotherapy ≤ 3 weeks prior to start of study intervention.
In plain language
Assembled directly from this trial’s registry record. Every sentence traces to a field below — nothing here is generated or interpreted.
What this study is
This study is testing a treatment for a condition.
Phase 1Phase 1The earliest stage of human testing, in a small group, focused on safety.Read more →/2 — a combined study that starts with early safety and continues into what the treatment does.
From the trial registry
Built from these fields:
- designModule.designInfo.primaryPurpose
- designModule.phases
Who receives what
There are 3 groups in this study.
Groups A, B and C receive Lurbinectedin.
Registry label: A: Phase 1 Part 1: Dose Selection · B: Phase 1 Part 2: RP2D · C: Phase 2
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].label
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
How the study is run
Groups are assigned by the study team using set rules, rather than by chance.
This study is open-labelOpen-labelEveryone knows which treatment is being given — nothing is hidden.Read more →: everyone knows which treatment is being given, including you and the study team.
From the trial registry
Built from these fields:
- designModule.designInfo.allocation
- designModule.designInfo.maskingInfo.masking
Is there a placebo?
This study does not list a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group.
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
Who the study is looking for
The study lists an age range of 2 years to 30 years.
The study is open to people of any sex.
The study does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
These are the criteria the study lists. Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part.
From the trial registry
Built from these fields:
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How big and how long
The study aims to enrol about 60 people.
The study is currently expected to finish around April 2028.
The main measurement is taken over: From the first dose through end of Cycle 1 (21 days).
From the trial registry
Built from these fields:
- designModule.enrollmentInfo.count
- statusModule.completionDateStruct.date
- outcomesModule.primaryOutcomes[].timeFrame
What the study measures
Phase 1Phase 1The earliest stage of human testing, in a small group, focused on safety.Read more →: Number of Participants Experiencing Dose-limiting Toxicities (DLTs) — measured over From the first dose through end of Cycle 1 (21 days).
Phase 1: Number of Participants Experiencing Serious Adverse EventsSerious adverse eventAn adverse event meeting a formal severity threshold, such as requiring hospital admission.Read more → (SAEs) and Treatment Emergent Adverse EventsAdverse eventAny medical problem that happens during a study, whether or not the treatment caused it.Read more → (TEAEs) — measured over Post-baselineBaselineYour starting measurements, taken before treatment begins.Read more → (Day 1) up to approximately 31 months.
Phase 1: Number of Participants With Dose Modifications — measured over Post-baseline (Day 1) up to approximately 31 months.
Phase 1: Number of Participants Who Discontinued Study Intervention Due to TEAEs — measured over Post-baseline (Day 1) up to approximately 31 months.
The study lists 1 further main measurements.
From the trial registry
Built from these fields:
- outcomesModule.primaryOutcomes[].measure
- outcomesModule.primaryOutcomes[].timeFrame
Source: NCT05734066 on ClinicalTrials.gov. The full registry text is further down this page — this summary never replaces it.
Not medical advice. What do these terms mean?
What is being tested — in plain terms
About LurbinectedinDrug
Administered as intravenous (IV) infusion once every 3 weeks (Q3W)
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
Common questions
Answered from this trial’s registry record. Where the record doesn’t say, these answers say so rather than filling the gap.
Am I eligible for this trial?
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part. Concord cannot make that determination, and neither can any tool that has not examined you.
What the study lists: an age range of 2 years to 30 years.
The full inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more → and exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → are published on this page, exactly as the study team wrote them.
The useful next step is to bring this trial to your doctor. Answering a few questions first gives them something concrete to review.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.eligibilityCriteria
Is there a placebo?
This study does not list a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group.
From the trial registry
- armsInterventionsModule.armGroups[].type
Would I know which treatment I am getting?
This study is open-labelOpen-labelEveryone knows which treatment is being given — nothing is hidden.Read more →: everyone knows which treatment is being given, including you and the study team.
Groups are assigned by the study team using set rules, rather than by chance.
From the trial registry
- designModule.designInfo.maskingInfo.masking
- designModule.designInfo.allocation
Who can join?
The study lists an age range of 2 years to 30 years.
It is open to people of any sex.
It does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can confirm whether a particular person can take part.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How long would this take?
The study's main measurement is taken over: From the first dose through end of Cycle 1 (21 days)..
The study as a whole is currently expected to finish around 2028-04-20.
How long any one person takes part can differ from the study length. The study team can tell you what the schedule looks like in practice.
From the trial registry
- outcomesModule.primaryOutcomes[].timeFrame
- statusModule.completionDateStruct.date
How many people are taking part?
The study aims to enrol about 60 people.
From the trial registry
- designModule.enrollmentInfo.count
Where is this happening?
This study lists 3 locations, including: Toronto, Ontario, Canada; Grand Rapids, Michigan, United States; New York, New York, United States.
Sites can open and close during a study, so confirm with the team before travelling.
From the trial registry
- trial_locations
About This Trial
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.
Other Sites (2)
Corewell Health
Grand Rapids, Michigan, United States
Memorial Sloan Kettering Cancer Center
New York, New York, United States
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See if this trial could fit youThis page is for informational purposes only and does not constitute medical advice. Clinical trial eligibility can only be determined by the trial site after proper screening. Trial information is sourced from ClinicalTrials.gov and may not reflect the most current status. Always consult your healthcare provider before making decisions about clinical trial participation.