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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT05269355
Registration number
NCT05269355
Ethics application status
Date submitted
25/02/2022
Date registered
8/03/2022
Date last updated
13/06/2025
Titles & IDs
Public title
A Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS)
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Scientific title
A Phase 2/3 Study to Evaluate the Efficacy and Safety of Unesbulin in Unresectable or Metastatic, Relapsed or Refractory Leiomyosarcoma
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Secondary ID [1]
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2022-000073-12
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Secondary ID [2]
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PTC596-ONC-008-LMS
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Universal Trial Number (UTN)
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Trial acronym
SUNRISELMS
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Leiomyosarcoma
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Condition category
Condition code
Cancer
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Sarcoma (also see 'Bone') - soft tissue
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Unesbulin
Treatment: Drugs - Dacarbazine
Other interventions - Placebo
Experimental: Unesbulin and Dacarbazine - Participants will receive unesbulin 300 milligrams (mg) tablets administered orally twice weekly in each 3-week treatment cycle in combination with dacarbazine 1000 mg/meter squared (m\^2) intravenously (IV) once every 21 days. Treatment will continue for each participant until evidence of unacceptable toxicity, disease progression, or treatment discontinuation for another reason.
Placebo comparator: Placebo and Dacarbazine - Participants will receive placebo matching to unesbulin tablets administered orally twice weekly in each 3-week treatment cycle in combination with dacarbazine 1000 mg/m\^2 IV once every 21 days. Treatment will continue for each participant until evidence of unacceptable toxicity, disease progression, or treatment discontinuation for another reason.
Treatment: Drugs: Unesbulin
Unesbulin will be administered as per the dose and schedule specified in the arm description.
Treatment: Drugs: Dacarbazine
Dacarbazine will be administered as per the dose and schedule specified in the arm description.
Other interventions: Placebo
Placebo will be administered as per the schedule specified in the arm description.
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Intervention code [1]
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Treatment: Drugs
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Intervention code [2]
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Other interventions
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Progression Free Survival (PFS) Per Independent Central Review Using Response Evaluation Criteria in Solid Tumors (RECIST) V1.1
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Assessment method [1]
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PFS was defined as the time from randomization to the documented disease progression or death due to any cause, whichever occurred first. Disease progression was defined as at least a 20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study. In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 millimeters (mm). The appearance of one or more new lesions was also considered progression.
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Timepoint [1]
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Up to approximately 2 years
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Secondary outcome [1]
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Overall Survival
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Assessment method [1]
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Overall survival was defined as the time in months from the randomization date to the date of death from any cause or date last known alive for those who did not die.
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Timepoint [1]
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Up to approximately 2 years
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Secondary outcome [2]
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Objective Response Rate (ORR) Per Independent Central Review Using RECIST V1.1
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Assessment method [2]
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ORR was defined as percentage of participants who achieved a confirmed best overall response (BOR) of complete response (CR) or partial response (PR). CR: Disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \<10 mm. PR: At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters.
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Timepoint [2]
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Up to approximately 2 years
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Secondary outcome [3]
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Disease Control Rate (DCR) Per Independent Central Review Using RECIST V1.1
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Assessment method [3]
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DCR was defined as percentage of participants who achieved a confirmed BOR of CR, PR, or at least 3 months of stable disease (SD). CR: Disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \<10 mm. PR: At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters. SD: Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study.
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Timepoint [3]
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Up to approximately 2 years
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Secondary outcome [4]
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Duration of Response Per Independent Central Review Using RECIST V1.1
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Assessment method [4]
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Duration of response was defined as the time from the date of first confirmed response of CR or PR to the date of the first documented disease progression or death due to any cause, whichever occurred first. CR: Disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \<10 mm. PR: At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters. Disease progression: At least a 20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study. In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm. The appearance of one or more new lesions was also considered progression.
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Timepoint [4]
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Up to approximately 2 years
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Secondary outcome [5]
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Number of Participants With Treatment-emergent Adverse Events (TEAEs)
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Assessment method [5]
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An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A TEAE was defined as an AE that had an onset date on or after the first dose of study drug until 30 days after last dose or occurred prior to first dose of study drug and worsened in severity after first dose of study drug. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.
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Timepoint [5]
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From first dose of study drug up to approximately 2 years
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Eligibility
Key inclusion criteria
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* Histological or cytological confirmation of LMS arising at any anatomic site except bone sarcoma, unresectable or metastatic, relapsed or refractory disease measurable per RECIST 1.1 criteria
* Disease progression on previous treatment before screening or intolerability to other oncology treatments
* Participants with liver metastases may be enrolled
* Participants with well-controlled asthma or chronic obstructive pulmonary disease may be enrolled.
* Toxicity from prior therapies recovered to Grade =1 or participant's baseline, except for alopecia. In addition, endocrinopathies associated with prior immunotherapy-based treatments that are well controlled on replacement medication are not exclusionary.
* At least 1 prior systemic cytotoxic or targeted therapy regimen for LMS, which may include but is not limited to single-agent doxorubicin or other anthracycline, doxorubicin plus ifosfamide, trabectedin, pazopanib, or gemcitabine with or without docetaxel.
* At least 4 weeks since prior surgery and recovered in the opinion of investigator
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Minimum age
18
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
* Received temozolomide or dacarbazine at any time
* Any other systemic anticancer therapy including investigational agents =3 weeks before initiation of study treatment. Additionally, participants may not have received radiation =3 weeks before initiation of study treatment.
* Known intolerance to dacarbazine or one or more of the excipients in unesbulin.
* Co-existing active infection or any co-existing medical condition likely to interfere with study procedures
* Gastrointestinal disease or other conditions that could affect absorption. Active peptic ulcer disease, active gastritis, or previous history of gastric perforation within the last 2 years
* Major surgery, open biopsy, or significant traumatic injury that has not recovered, in the opinion of the investigator, within 28 days of baseline
* Immunization with a live vaccine within 30 days before starting study drug due to the risk of serious and life-threatening infections.
* Prior malignancies, other than LMS, that required treatment or have shown evidence of recurrence (except for non-melanoma skin cancer or adequately treated cervical carcinoma in situ, prostate cancer in situ or any other low risk malignancy that is approved by the medical monitor) during the 5 years before initiation.
* Prior or ongoing clinically significant illness, medical or psychiatric condition, medical history, physical findings, electrocardiogram (ECG) findings, or laboratory abnormality that, in the investigator's opinion, could affect the safety of the participant, or alter the absorption, distribution, metabolism, or excretion of the study drugs, or could impair the assessment of study results.
Note: Other inclusion and exclusion criteria may apply.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Stopped early
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
23/05/2022
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
17/07/2024
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Sample size
Target
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Accrual to date
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Final
359
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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Chris O'Brien Lifehouse - Camperdown
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Recruitment hospital [2]
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Peter MacCallum Cancer Institute - East Melbourne
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Recruitment hospital [3]
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Prince of Wales Hospital - Randwick
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Recruitment postcode(s) [1]
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2050 - Camperdown
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Recruitment postcode(s) [2]
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3000 - East Melbourne
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Recruitment postcode(s) [3]
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2031 - Randwick
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Recruitment outside Australia
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United States of America
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California
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Colorado
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Rio de Janeiro
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Salvador
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São José do Rio Preto
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Ontario
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Lyon
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Mannheim
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Madrid
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United Kingdom
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Manchester
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
PTC Therapeutics
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Address
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Ethics approval
Ethics application status
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Summary
Brief summary
This study will compare the efficacy and safety of unesbulin plus dacarbazine versus placebo plus dacarbazine in participants with unresectable or metastatic, relapsed or refractory LMS who have received at least 1 prior line of systemic therapy.
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Trial website
https://clinicaltrials.gov/study/NCT05269355
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Mark Rance, MD
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Address
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PTC Therapeutics
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Contact person for scientific queries
Data sharing statement
What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/55/NCT05269355/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/55/NCT05269355/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT05269355
Download to PDF