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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT05269355




Registration number
NCT05269355
Ethics application status
Date submitted
25/02/2022
Date registered
8/03/2022
Date last updated
16/05/2024

Titles & IDs
Public title
A Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS)
Scientific title
A Phase 2/3 Study to Evaluate the Efficacy and Safety of Unesbulin in Unresectable or Metastatic, Relapsed or Refractory Leiomyosarcoma
Secondary ID [1] 0 0
2022-000073-12
Secondary ID [2] 0 0
PTC596-ONC-008-LMS
Universal Trial Number (UTN)
Trial acronym
SUNRISELMS
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Leiomyosarcoma 0 0
Condition category
Condition code
Cancer 0 0 0 0
Sarcoma (also see 'Bone') - soft tissue

Intervention/exposure
Study type
Interventional
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.
Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Progression-free Survival per Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 Assessed by an Independent Central Imaging Laboratory
Timepoint [1] 0 0
From the date of randomization to the date of the first documented tumor progression or death due to any cause, whichever occurs first (up to approximately 2 years)
Secondary outcome [1] 0 0
Overall Survival
Timepoint [1] 0 0
From the date of randomization to the date of death due to any cause (up to approximately 2 years)
Secondary outcome [2] 0 0
Objective Response Rate (ORR)
Timepoint [2] 0 0
From the date of randomization until the date of objectively documented progression or the date of initiation of subsequent therapy or palliative local therapy, whichever occurs first (up to approximately 2 years)
Secondary outcome [3] 0 0
Disease Control Rate (DCR)
Timepoint [3] 0 0
From the date of randomization until the date of the first documented tumor progression or the date of initiation of subsequent therapy or palliative local therapy, whichever occurs first (up to approximately 2 years)
Secondary outcome [4] 0 0
Duration of Response per RECIST 1.1 Assessed by an Independent Central Imaging Laboratory
Timepoint [4] 0 0
Time from the date of first confirmed response to the date of the first documented tumor progression or death due to any cause, whichever occurs first (up to approximately 2 years)
Secondary outcome [5] 0 0
Number of Participants with Treatment-emergent Adverse Events
Timepoint [5] 0 0
From the date of randomization up to approximately 2 years

Eligibility
Key inclusion criteria
Key

- 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

Key
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s

The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2/Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
23/05/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
30/06/2024
Actual
Sample size
Target
Accrual to date
Final
360
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Chris O'Brien Lifehouse - Camperdown
Recruitment hospital [2] 0 0
Peter MacCallum Cancer Institute - East Melbourne
Recruitment hospital [3] 0 0
Prince of Wales Hospital - Randwick
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
3000 - East Melbourne
Recruitment postcode(s) [3] 0 0
2031 - Randwick
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Connecticut
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United States of America
State/province [4] 0 0
Florida
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United States of America
State/province [5] 0 0
Illinois
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United States of America
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Maryland
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United States of America
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Michigan
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United States of America
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Missouri
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United States of America
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New York
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United States of America
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North Carolina
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United States of America
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Ohio
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United States of America
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Oregon
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United States of America
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Pennsylvania
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United States of America
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Texas
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Brazil
State/province [15] 0 0
Barretos
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Brazil
State/province [16] 0 0
Porto Alegre
Country [17] 0 0
Brazil
State/province [17] 0 0
Rio de Janeiro
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Brazil
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Salvador
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Brazil
State/province [19] 0 0
São José do Rio Preto
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Brazil
State/province [20] 0 0
São Paulo
Country [21] 0 0
Canada
State/province [21] 0 0
Ontario
Country [22] 0 0
Canada
State/province [22] 0 0
Quebec
Country [23] 0 0
France
State/province [23] 0 0
Bordeaux Cedex
Country [24] 0 0
France
State/province [24] 0 0
Lyon
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France
State/province [25] 0 0
Paris
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France
State/province [26] 0 0
Villejuif cedex
Country [27] 0 0
Germany
State/province [27] 0 0
Mannheim
Country [28] 0 0
Germany
State/province [28] 0 0
Munchen
Country [29] 0 0
Hungary
State/province [29] 0 0
Budapest
Country [30] 0 0
Italy
State/province [30] 0 0
Milano
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Italy
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Torino
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Netherlands
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Leiden
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Poland
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Poznan
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Poland
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Warszawa
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Spain
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Barcelona
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Spain
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Madrid
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United Kingdom
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Glasgow
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United Kingdom
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London
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United Kingdom
State/province [39] 0 0
Manchester

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
PTC Therapeutics
Address
Country

Ethics approval
Ethics application status

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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT05269355
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Mark Rance, MD
Address 0 0
PTC Therapeutics
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries