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LeiomyosarcomaPhase 2/3SUNRISELMSNCT05269355

A Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS)

A Phase 2/3 study testing Unesbulin and Dacarbazine in people with Leiomyosarcoma, run by PTC Therapeutics at 54 sites in 12 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Leiomyosarcoma
What is being tested?
Unesbulin and Dacarbazine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
359 participants
Where
54 sitesin 12 countries
Study dates
23 May 2022to 17 Jul 2024
Phase
Phase 2/3
Are there results?
Yes, posted in the registrySummary in the doctors' view

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 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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
Show all 10Show fewer
  • 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.

Trial locations and contacts

54 sites in 12 countries.

54 sites in 12 countries

United States23 sites

  • University of Michigan

    Ann Arbor, Michigan, 48109

  • Johns Hopkins Oncology Group

    Baltimore, Maryland, 21231

  • Northwestern Memorial Hospital

    Chicago, Illinois, 60611

  • The Ohio State University (OSU)

    Columbus, Ohio, 43210

  • University of Colorado Denver

    Denver, Colorado, 80045

  • City of Hope

    Duarte, California, 91010

  • Duke University Medical Center

    Durham, North Carolina, 27710

  • MD Anderson Cancer Center

    Houston, Texas, 77030

  • Mayo Clinic

    Jacksonville, Florida, 32224

  • University of California, Los Angeles (UCLA) - Jonsson Comprehensive Cancer Center

    Los Angeles, California, 90024

  • Yale University

    New Haven, Connecticut, 06511

  • The Trustees of Columbia University

    New York, New York, 10027

  • David H. Koch Center for Cancer Care at Memorial Sloan Kettering Cancer Center

    New York, New York, 11101

  • University of Florida (UF) Health Cancer Center - Orlando Health

    Orlando, Florida, 32806

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104

  • Thomas Jefferson University

    Philadelphia, Pennsylvania, 19144

  • University of Pittsburgh Medical Center

    Pittsburgh, Pennsylvania, 15232

  • Oregon Health & Science University

    Portland, Oregon, 97239

  • Sarcoma Oncology Research Center

    Santa Monica, California, 90403

  • Washington University

    St Louis, Missouri, 63110

  • Stanford Cancer Center

    Stanford, California, 94305

  • Moffitt

    Tampa, Florida, 33612

  • Northwestern Medicine - Warrenville Cancer Center

    Warrenville, Illinois, 60555

Other trials in United States

Brazil6 sites

  • Fundacao PIO XII - Hospital de Amor

    Barretos

  • Santa Casa de Misericordia de Porto Alegre

    Porto Alegre

  • INCA I - Instituto Nacional de Cancer

    Rio de Janeiro, 20220-410

  • Hospital Sao Rafael - Instituto D'Or da Bahia

    Salvador

  • CIP - Centro Integrado de Pesquisas do Hospital de Base de Sao Jose do Rio Preto

    São José do Rio Preto

  • Instituto do Cancer do Estado de São Paulo (ICESP)

    São Paulo

Other trials in Brazil

France4 sites

  • Institut Bergonie

    Bordeaux, 33076

  • Centre Leon Berard

    Lyon, 69008

  • Institut Curie

    Paris, 75005

  • Gustave Roussy

    Villejuif

Other trials in France

Spain4 sites

  • Institut Catala d'Oncologia (Hospital Duran y Reynals)

    Barcelona, 08908

  • Hospital Universitario Vall d'Hebron

    Barcelona

  • Hospital Fundacion Jimenez Diaz

    Madrid, 28040

  • Hospital Universitario 12 de Octubre

    Madrid

Other trials in Spain

Australia3 sites

  • Chris O'Brien Lifehouse

    Camperdown, 2050

  • Peter MacCallum Cancer Institute

    East Melbourne, 3000

  • Prince of Wales Hospital

    Randwick, 2031

Other trials in Australia

Canada3 sites

  • Universite de Montreal - Hopital Maisonneuve-Rosemont (HMR)

    Montreal, Quebec

  • The Ottawa Hospital Cancer Centre

    Ottawa, Ontario, K1H8L6

  • Princess Margaret Hospital

    Toronto, Ontario

Other trials in Canada

United Kingdom3 sites

  • Beatson, West of Scotland Cancer Centre

    Glasgow, G12 0YN

  • Royal Marsden Hospital

    London, SW3 6JJ

  • The Christie NHS Foundation Trust

    Manchester

Other trials in United Kingdom

Germany2 sites

  • Universitaetsmedizin Mannheim

    Mannheim, 68167

  • Klinikum der Ludwig-Maximilians-Universitaet Muenchen

    München, 81377

Other trials in Germany

Italy2 sites

  • Fondazione IRCCS Istituto Nazionale dei Tumori

    Milan, 20133

  • La Fondazione e l'Istituto di Candiolo

    Torino, 10060

Other trials in Italy

Poland2 sites

  • Niepubliczny Zaklad Opieki Zdrowotnej Zespól Poradni Specjalistycznych "TERMEDICA"

    Poznan

  • Narodowy Instytut Onkologii im. Marii Sklodowskiej-Curie - Panstwowy Instytut Badawczy, Klinika Nowotworow Tkanek Miekkich, Kosci i Czerniakow

    Warsaw, 02-781

Other trials in Poland

Hungary1 site

  • Eszak-Pesti Centrumkorhaz - Honvedkorhaz

    Budapest

Other trials in Hungary

Netherlands1 site

  • Leids Universitair Medisch Centrum

    Leiden

Other trials in Netherlands

About this study

Official title: A Phase 2/3 Study to Evaluate the Efficacy and Safety of Unesbulin in Unresectable or Metastatic, Relapsed or Refractory Leiomyosarcoma

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.

Conditions
Leiomyosarcoma
Being tested
Unesbulin, Dacarbazine, Placebo
Medical subject headings
Leiomyosarcoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • UnesbulinDrug

    Also called PTC596

    Unesbulin will be administered as per the dose and schedule specified in the arm description.

  • DacarbazineDrug

    Also called DTIC

    Dacarbazine will be administered as per the dose and schedule specified in the arm description.

  • PlaceboOther

    Placebo will be administered as per the schedule specified in the arm description.

Study groups in NCT05269355
Study groupRoleWhat they receive
Unesbulin and DacarbazineExperimentalParticipants 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.
Receives: Drug: Unesbulin; Drug: Dacarbazine
Placebo and DacarbazinePlacebo comparatorParticipants 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.
Receives: Drug: Dacarbazine; Other: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Progression Free Survival (PFS) Per Independent Central Review Using Response Evaluation Criteria in Solid Tumors (RECIST) V1.1

    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.

    Measured: Up to approximately 2 years

Secondary outcomes (5)

  1. Overall Survival

    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.

    Measured: Up to approximately 2 years

  2. Objective Response Rate (ORR) Per Independent Central Review Using RECIST V1.1

    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.

    Measured: Up to approximately 2 years

  3. Disease Control Rate (DCR) Per Independent Central Review Using RECIST V1.1

    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.

    Measured: Up to approximately 2 years

  4. Duration of Response Per Independent Central Review Using RECIST V1.1

    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.

    Measured: Up to approximately 2 years

  5. Number of Participants With Treatment-emergent Adverse Events (TEAEs)

    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'.

    Measured: From first dose of study drug up to approximately 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted8 Mar 2022
  2. Study start23 May 2022
  3. Primary completion17 Jun 2024
  4. Study completion17 Jul 2024

Why it stopped: Business decision

Resources

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 2/3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Triple blind
Who is masked
Participant, Investigator, Outcomes assessor
Enrolment
359 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05269355
Sponsor's protocol ID
PTC596-ONC-008-LMS
Other IDs
2022-000073-12
Acronym
SUNRISELMS
Status last verified
May 2025
First submitted
25 Feb 2022
Last updated
13 Jun 2025
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing Unesbulin and Dacarbazine in people with Leiomyosarcoma, run by PTC Therapeutics at 54 sites in 12 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 7 inclusion and 10 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: stopped early, as of the sponsor's last update on 13 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 54 sites in United States, Brazil, France, Spain, Australia and 7 more countries. 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. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 13 Jun 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Progression Free Survival (PFS) Per Independent Central Review Using Response Evaluation Criteria in Solid Tumors (RECIST) V1.1 (time frame: Up to approximately 2 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05269355.

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 13 Jun 2025.

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