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Efficacy and Safety of Losmapimod in Treating Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) (REACH)

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ClinicalTrials.gov Identifier: NCT05397470
Recruitment Status : Active, not recruiting
First Posted : May 31, 2022
Last Update Posted : March 12, 2024
Sponsor:
Information provided by (Responsible Party):
Fulcrum Therapeutics

Tracking Information
First Submitted Date  ICMJE May 4, 2022
First Posted Date  ICMJE May 31, 2022
Last Update Posted Date March 12, 2024
Actual Study Start Date  ICMJE June 16, 2022
Estimated Primary Completion Date October 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 7, 2024)
  • Part A: Change from Baseline in total Relative surface area (RSA) Quadrants 1 to 5 (Q1-Q5) with 500 grams (g) wrist weight averaged over both arms as assessed by Reachable workspace (RWS) at Week 48 [ Time Frame: Baseline and at Week 48 ]
    The RWS is a clinical outcome measure that measures the relative surface area that a participant may reach with an outstretched arm. Responses are rated on a scale of 0 (no reachable workspace) to 1.25 (maximal reachable workspace). Higher scores indicate better outcomes.
  • Part B: Number of participants reporting Adverse events (AEs) [ Time Frame: Up to Week 192 ]
  • Part B: Number of participants with clinically significant changes in clinical laboratory parameters, Electrocardiogram (ECG), vital signs and physical examinations [ Time Frame: Up to Week 192 ]
Original Primary Outcome Measures  ICMJE
 (submitted: May 25, 2022)
Reachable Workspace (RWS) [ Time Frame: Screening, Baseline and Weeks 4, 12, 24, 36, and 48 ]
The Reachable Workspace will be used to measure change(s) from baseline in the participants upper extremity function. The Reachable Workspace is a clinical outcome measure that measures the relative surface area that an individual may reach with an outstretched arm. Responses are rated on a scale of 0 (no reachable workspace) to 1.25 (maximal reachable workspace).
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: March 7, 2024)
  • Part A: Change from Baseline in Quality of Life in Neurologic Disorders upper extremity (Neuro-QoL UE) Scale at Week 48 [ Time Frame: Baseline and at Week 48 ]
    The Neuro-QoL UE will be used to measure change(s) from Baseline in the participants upper extremity function. The Neuro-QoL UE is a questionnaire that measures the participants self-reported upper extremity function including activities of daily living (ADLs) involving digital, manual, and reach-related function and self-care. Responses are rated from 1 (unable to do) to 5 (without any difficulty). Lower scores indicate worse symptoms.
  • Part A: Patient's Global Impression of Change (PGIC) at Week 48 [ Time Frame: At Week 48 ]
    The Patient Global Impression of Change (PGIC) is a standard and validated participant-report outcome that measures the participant's self-reported change in health status compared to the start of the study. The PGIC uses a single question and 7-point patient self-reporting scale of overall improvement during treatment ranging from 1 (very much improved) to 7 (very much worse). Higher scores indicate worse symptoms.
  • Part A: Change from Baseline in Whole body (WB) longitudinal composite Muscle Fat Infiltration (MFI) of B muscles at Week 48 [ Time Frame: Baseline and at Week 48 ]
    Change from Baseline in skeletal muscle tissue replacement by fat will be measured by WB musculoskeletal (MSK) magnetic resonance imaging (MRI).
  • Part A: Change from Baseline in average shoulder abductor strength by hand-held quantitative dynamometry at Week 48 [ Time Frame: Baseline and at Week 48 ]
    Quantitative: isometric dynamometry (hand-held dynamometer) will be used to assess the skeletal muscle strength. Isometric dynamometry measures the static muscle strength without any movement.
  • Part A: Number of participants reporting Adverse events (AEs) [ Time Frame: Up to Week 48 ]
  • Part A: Number of participants with clinically significant changes in clinical laboratory parameters, ECG, vital signs and physical examinations [ Time Frame: Up to Week 48 ]
Original Secondary Outcome Measures  ICMJE
 (submitted: May 25, 2022)
  • Neuro-QoL Upper Extremity (UE) Scale [ Time Frame: Screening, Baseline and Weeks 4, 12, 24, 36, and 48 ]
    The Neuro-QoL UE will be used to measure change(s) from baseline in the participants upper extremity function. The Neuro-QoL UE is a questionnaire that measures the participants self-reported upper extremity function including activities of daily living (ADLs) involving digital, manual, and reach-related function and self-care. Responses are rated from 1 (unable to do) to 5 (without any difficulty).
  • Patient's Global Impression of Change (PGIC) [ Time Frame: Weeks 4, 12, 24, 36, and 48 ]
    The Patient Global Impression of Change (PGIC) is a standard and validated patient-report outcome that measures the patient's self-reported change in health status compared to the start of the study. The PGIC uses a single question and 7-point patient self-reporting scale of overall improvement during treatment ranging from 1 (very much improved) to 7 (very much worse).
  • Muscle Fat Infiltration [ Time Frame: Week 48 ]
    Change from baseline in skeletal muscle tissue replacement by fat will be measured by whole body (WB) musculoskeletal (MSK) magnetic resonance imaging (MRI).
  • Treatment-Emergent Adverse Events [ Time Frame: Screening, Baseline and Weeks 4, 12, 24, 36, 48 and 7 days after the last dose of study drug ]
    Incidence of treatment-emergent adverse events (TEAEs) will be monitored continuously from screening to the end of safety follow-up visit. A treatment-emergent adverse event is defined as any event that was not present before exposure to the study drug or any event already present that worsens in either intensity or frequency after exposure to the study drug.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Efficacy and Safety of Losmapimod in Treating Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) (REACH)
Official Title  ICMJE A Phase 3 Global, Randomized, Double-Blind, Placebo-Controlled, 48-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) (REACH)
Brief Summary This is a study to evaluate the safety and efficacy of losmapimod in treating participants with Facioscapulohumeral Muscular Dystrophy (FSHD). Participants diagnosed with Facioscapulohumeral muscular dystrophy type 1 (FSHD1) or Facioscapulohumeral muscular dystrophy type 2 (FSHD2) will participate in Part A (Placebo-controlled treatment period) and will be randomized in a 1:1 ratio to receive losmapimod 15 milligrams (mg) or placebo orally twice daily (BID). Upon completion of Part A, participants will have the option to rollover into Part B (open-label extension) to evaluate the long-term safety, tolerability, and efficacy of losmapimod and will receive losmapimod 15 mg orally BID.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description:
Part A of this study will be performed in a double-blind fashion. Part B of the study will be performed in an open-label fashion. The investigator, study staff, subjects, sponsor, and monitor will remain blinded to the treatment in Part A until study closure (i.e., until closure of Part B).
Primary Purpose: Treatment
Condition  ICMJE Facioscapulohumeral Muscular Dystrophy (FSHD)
Intervention  ICMJE
  • Drug: Losmapimod
    Losmapimod 15 mg will be administered BID by mouth along with food.
  • Drug: Placebo oral tablet
    Placebo will be administered BID by mouth along with food.
Study Arms  ICMJE
  • Experimental: Part A: Placebo-controlled treatment period: Losmapimod
    Participants will be randomized to receive losmapimod.
    Intervention: Drug: Losmapimod
  • Placebo Comparator: Part A: Placebo-controlled treatment period: Placebo
    Participants will be randomized to receive placebo
    Intervention: Drug: Placebo oral tablet
  • Experimental: Part B: Open-label extension
    Participants will receive losmapimod, upon completion of all assessments for Part A.
    Intervention: Drug: Losmapimod
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: September 27, 2023)
260
Original Estimated Enrollment  ICMJE
 (submitted: May 25, 2022)
230
Estimated Study Completion Date  ICMJE January 2026
Estimated Primary Completion Date October 2024   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Participants must be between 18 and 65 years of age, inclusive.
  • Genetically confirmed diagnosis of FSHD 1 or FSHD 2.
  • Clinical severity score of 2 to 4 (Ricci Score; Range 0-5), at screening. Participants who are wheelchair-dependent or dependent on walker or wheelchair for activities are not permitted to enroll in the study.
  • Screening total RSA (Q1-Q4) without weight in the dominant UE assessed by RWS ≥ 0.2 and ≤ 0.7.
  • No contraindications to MRI.

Exclusion Criteria:

  • Previously diagnosed cancer that has not been in complete remission for at least 5 years. Localized carcinomas of the skin and carcinoma in situ of the cervix that have been resected or ablated for cure are not exclusionary.
  • Participants who are on drug(s) or supplements that may affect muscle function, as determined by the Investigator: participants must be on a stable dose of that drug(s) or supplement for at least 3 months prior to the first dose of study drug and remain on that stable dose for the duration of the study.
  • Known active opportunistic or life-threatening infections including Human Immunodeficiency virus (HIV) and hepatitis B or C.
  • Known active or inactive tuberculosis infection.
  • Acute or chronic history of liver disease.
  • Known severe renal impairment.
  • History of cardiac dysrhythmias requiring anti-arrhythmia treatment(s); or history or evidence of abnormal ECGs.
  • Use of another investigational product within 30 days or 5 half-lives (whichever is longer) or currently participating in a study of an investigational device.
  • Current or anticipated participation in a natural history study. Previous participation is allowed but participants cannot continue after enrollment in Study 1821-FSH-301.
  • Known hypersensitivity to losmapimod or any of its excipients.
  • Previous participation in a Fulcrum-sponsored FSHD losmapimod study (FIS-001-2019 or FIS-002-2019).

Note that all other inclusion and exclusion criteria are listed in the protocol and only key are presented.

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 65 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Canada,   Denmark,   France,   Germany,   Italy,   Netherlands,   Spain,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT05397470
Other Study ID Numbers  ICMJE 1821-FSH-301
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Current Responsible Party Fulcrum Therapeutics
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Fulcrum Therapeutics
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Fulcrum Therapeutics
Verification Date March 2024

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP