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

March 28, 2024 updated by: PTC Therapeutics

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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

360

Phase

  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Australia
      • Camperdown, Australia, 2050
        • Chris O'Brien Lifehouse
      • East Melbourne, Australia, 3000
        • Peter MacCallum Cancer Institute
      • Randwick, Australia, 2031
        • Prince of Wales Hospital
  • Brazil
      • Barretos, Brazil
        • Fundacao PIO XII - Hospital de Amor
      • Porto Alegre, Brazil
        • Santa Casa de Misericordia de Porto Alegre
      • Rio de Janeiro, Brazil, 20220-410
        • INCA I - Instituto Nacional de Cancer
      • Salvador, Brazil
        • Hospital Sao Rafael - Instituto D'Or da Bahia
      • São José do Rio Preto, Brazil
        • CIP - Centro Integrado de Pesquisas do Hospital de Base de Sao Jose do Rio Preto
      • São Paulo, Brazil
        • Instituto do Cancer do Estado de São Paulo (ICESP)
  • Canada
    • Ontario
      • Ottawa, Ontario, Canada, K1H8L6
        • The Ottawa Hospital Cancer Centre
      • Toronto, Ontario, Canada
        • Princess Margaret Hospital
    • Quebec
      • Montreal, Quebec, Canada
        • Universite de Montreal - Hopital Maisonneuve-Rosemont (HMR)
  • France
      • Bordeaux Cedex, France, 33076
        • Institut Bergonie
      • Lyon, France, 69008
        • Centre LEON BERARD
      • Paris, France, 75005
        • Institut Curie
      • Villejuif cedex, France
        • Gustave Roussy
  • Germany
      • Mannheim, Germany, 68167
        • Universitaetsmedizin Mannheim
      • Munchen, Germany, 81377
        • Klinikum der Ludwig-Maximilians-Universitaet Muenchen
  • Hungary
      • Budapest, Hungary
        • Eszak-Pesti Centrumkorhaz - Honvedkorhaz
  • Italy
      • Milano, Italy, 20133
        • Fondazione IRCCS Istituto Nazionale dei Tumori
      • Torino, Italy, 10060
        • La Fondazione e l'Istituto di Candiolo
  • Netherlands
      • Leiden, Netherlands
        • Leids Universitair Medisch Centrum
  • Poland
      • Poznan, Poland
        • Niepubliczny Zaklad Opieki Zdrowotnej Zespól Poradni Specjalistycznych "TERMEDICA"
      • Warszawa, Poland, 02-781
        • Narodowy Instytut Onkologii im. Marii Sklodowskiej-Curie - Panstwowy Instytut Badawczy, Klinika Nowotworow Tkanek Miekkich, Kosci i Czerniakow
  • Spain
      • Barcelona, Spain
        • Hospital Universitario Vall d'Hebron
      • Barcelona, Spain, 08908
        • Institut Catala d'Oncologia (Hospital Duran y Reynals)
      • Madrid, Spain
        • Hospital Universitario 12 de octubre
      • Madrid, Spain, 28040
        • Hospital Fundación Jiménez Díaz
  • United Kingdom
      • Glasgow, United Kingdom, G12 0YN
        • Beatson, West of Scotland Cancer Centre
      • London, United Kingdom, SW3 6JJ
        • Royal Marsden Hospital
      • Manchester, United Kingdom
        • The Christie NHS Foundation Trust
  • United States
    • California
      • Duarte, California, United States, 91010
        • City of Hope
      • Los Angeles, California, United States, 90024
        • University of California, Los Angeles (UCLA) - Jonsson Comprehensive Cancer Center
      • Santa Monica, California, United States, 90403
        • Sarcoma Oncology Research Center
      • Stanford, California, United States, 94305
        • Stanford Cancer Center
    • Colorado
      • Denver, Colorado, United States, 80045
        • University of Colorado Denver
    • Connecticut
      • New Haven, Connecticut, United States, 06511
        • Yale University
    • Florida
      • Jacksonville, Florida, United States, 32224
        • Mayo Clinic
      • Orlando, Florida, United States, 32806
        • University of Florida (UF) Health Cancer Center - Orlando Health
      • Tampa, Florida, United States, 33612
        • Moffitt
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Northwestern Memorial Hospital
      • Warrenville, Illinois, United States, 60555
        • Northwestern Medicine - Warrenville Cancer Center
    • Maryland
      • Baltimore, Maryland, United States, 21231
        • Johns Hopkins Oncology Group
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • University of Michigan
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Washington University
    • New York
      • New York, New York, United States, 10027
        • The Trustees of Columbia University
      • New York, New York, United States, 11101
        • David H. Koch Center for Cancer Care at Memorial Sloan Kettering Cancer Center
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Duke University Medical Center
    • Ohio
      • Columbus, Ohio, United States, 43210
        • The Ohio State University (OSU)
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health & Science University
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • University of Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19144
        • Thomas Jefferson University
      • Pittsburgh, Pennsylvania, United States, 15232
        • University of Pittsburgh Medical Center
    • Texas
      • Houston, Texas, United States, 77030
        • MD Anderson Cancer Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria:

  • 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 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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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.
Drug: Unesbulin
Unesbulin will be administered as per the dose and schedule specified in the arm description.
Other Names:
  • PTC596
Drug: Dacarbazine
Dacarbazine will be administered as per the dose and schedule specified in the arm description.
Other Names:
  • DTIC
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.
Drug: Dacarbazine
Dacarbazine will be administered as per the dose and schedule specified in the arm description.
Other Names:
  • DTIC
Other: Placebo
Placebo will be administered as per the schedule specified in the arm description.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Progression-free Survival per Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 Assessed by an Independent Central Imaging Laboratory
Time Frame: 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)
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 Measures

Outcome Measure
Measure Description
Time Frame
Overall Survival
Time Frame: From the date of randomization to the date of death due to any cause (up to approximately 2 years)
From the date of randomization to the date of death due to any cause (up to approximately 2 years)
Objective Response Rate (ORR)
Time Frame: 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)
ORR is defined as the number of participants who achieve a confirmed best overall response (BOR) of complete response (CR) or partial response (PR) using RECIST 1.1 as per independent radiologist assessment.
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)
Duration of Response per RECIST 1.1 Assessed by an Independent Central Imaging Laboratory
Time Frame: 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)
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)
Number of Participants with Treatment-emergent Adverse Events
Time Frame: From the date of randomization up to approximately 2 years
From the date of randomization up to approximately 2 years
Disease Control Rate (DCR)
Time Frame: 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)
DCR is defined as the number of participants with BOR of CR, PR, or at least 3 months of stable disease using RECIST 1.1 as per independent radiologist assessment.
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)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

PTC Therapeutics

Investigators

  • Study Director: Mark Rance, MD, PTC Therapeutics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 23, 2022

Primary Completion (Estimated)

June 30, 2024

Study Completion (Estimated)

June 30, 2024

Study Registration Dates

First Submitted

February 25, 2022

First Submitted That Met QC Criteria

February 25, 2022

First Posted (Actual)

March 8, 2022

Study Record Updates

Last Update Posted (Actual)

March 29, 2024

Last Update Submitted That Met QC Criteria

March 28, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • PTC596-ONC-008-LMS
  • 2022-000073-12 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Leiomyosarcoma

Clinical Trials on Unesbulin

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Malta

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe