Facioscapulohumeral Muscular Dystrophy (FSHD)Phase 3NCT05397470
Efficacy and Safety of Losmapimod in Treating Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) (REACH)
A Phase 3 study testing Losmapimod in people with Facioscapulohumeral Muscular Dystrophy (FSHD), run by Fulcrum Therapeutics at 33 sites in 9 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Facioscapulohumeral Muscular Dystrophy (FSHD)
- What is being tested?
- Losmapimod
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 260 participants
- Where
- 33 sitesin 9 countries
- Study dates
- 16 Jun 2022to 19 Nov 2024
- Phase
- Phase 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 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Show all 12Show fewer
- 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.
Trial locations and contacts
33 sites in 9 countries.
33 sites in 9 countries
United States14 sites
University of Colorado Anschutz Medical Campus
Aurora, Colorado, 80045
Kennedy Krieger Institute
Baltimore, Maryland, 21205
Ohio State University Medical Center
Columbus, Ohio, 43210
University of Florida
Gainesville, Florida, 32610
University of California Irvine
Irvine, California, 92868
University of Kansas Medical Center
Kansas City, Kansas, 66160
University of California Los Angeles (UCLA)
Los Angeles, California, 90095
Virginia Commonwealth University
Richmond, Virginia, 23298
Mayo Clinic
Rochester, Minnesota, 55905
University of Rochester Medical Center
Rochester, New York, 14642
University of Utah
Salt Lake City, Utah, 84132
University of Washington Medical Center
Seattle, Washington, 98195
Washington University School of Medicine
St Louis, Missouri, 63110
University of Massachusetts Memorial Medical Center
Worcester, Massachusetts, 01655
Canada3 sites
University of Calgary
Calgary, Alberta, T2N 4Z6
Montreal Neurological Institute and Hospital
Montreal, Quebec, H3A 2B4
The Ottawa Hospital Research Institute
Ottawa, Ontario, K1Y 4E9
Germany3 sites
University Hospital Bonn
Bonn, 53127
LMU Klinikum Ludwig-Maximilians-Universität München
München, 80336
Universitätsklinikum Ulm
Ulm, 89081
Spain3 sites
Hospital Universitario Vall d'Hebron
Barcelona, 08035
Hospital Universitario Donostia
San Sebastián, Guipuzkoa, 20014
Hospital Universitari i Politecnic La Fe
Valencia, 46026
Denmark2 sites
Aarhus Universitetshospital
Aarhus, 8200
Rigshospitalet
Copenhagen, 2100
France2 sites
Nice University Hospital - CHU Nice
Nice, PACA, 06001
Institute de Myologie, Groupe Hospitalier Pitié-Salpêtrière
Paris, 75013
Italy2 sites
Fondazione Serena Onlus- Centro Clinico NEMO
Milan, Lombardy, 20162
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, 20133
Netherlands2 sites
Leiden University Medical Centre
Leiden, Southern Holland, 2333 ZA
Radboudumc
Nijmegen, Gelderland, 9101
United Kingdom2 sites
University College of London Hospitals
London, WC1N 3BG
Newcastle upon Tyne NHS Foundation Trust
Newcastle upon Tyne, NE1 3BZ
About this study
Official title: 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)
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.
- Being tested
- Losmapimod, Placebo oral tablet
- Keywords
- Facioscapulohumeral muscular dystrophy (FSHD), Facioscapulohumeral muscular dystrophy type 1 (FSHD 1), Facioscapulohumeral muscular dystrophy type 2 (FSHD 2), Muscular Dystrophy, Facioscapulohumeral Muscular Disorders, Musculoskeletal Diseases, Neuromuscular Diseases, REACH
- Medical subject headings
- Muscular Dystrophy, Facioscapulohumeral, Muscular Dystrophies, Musculoskeletal Diseases, Neuromuscular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LosmapimodDrug
Losmapimod 15 mg will be administered BID by mouth along with food.
Placebo oral tabletDrug
Placebo will be administered BID by mouth along with food.
| Study group | Role | What they receive |
|---|---|---|
| Part A: Placebo-controlled treatment period: Losmapimod | Experimental | Participants will be randomized to receive losmapimod. Receives: Drug: Losmapimod |
| Part A: Placebo-controlled treatment period: Placebo | Placebo comparator | Participants will be randomized to receive placebo Receives: Drug: Placebo oral tablet |
| Part B: Open-label extension | Experimental | Participants will receive losmapimod, upon completion of all assessments for Part A. Receives: Drug: Losmapimod |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Participants are instructed to complete a simple set of standardized movements of each arm centered around the shoulder joint. These arm movements are captured and quantitated with the use of a video camera. 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. Baseline is the last non-missing evaluation prior to first dose of study drug. Change from Baseline was calculated as the post-treatment value minus the value at Baseline.
Measured: Baseline (Day 1) and at Week 48
Part B: Number of Participants Reporting Serious Treatment Emergent Adverse Events (Serious TEAEs) and Non-serious TEAEs > 5%
Treatment-emergent adverse event is an AE that begins on or after the first dose of study drug and on or before the stop of study drug + 35 days or begins before the first dose of study drug and worsens on or after the first dose of study drug and on or before the stop of study drug + 35 days. A SAE is defined as an AE that results in any of the following outcomes: death; life-threatening adverse event; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions; congenital anomaly/birth defect.
Measured: Week 48 to Week 127
Part B: Number of Participants With Clinically Significant Changes in Chemistry Parameters
Blood samples were collected for the analysis of chemistry parameters: Glucose, sodium, potassium, calcium, inorganic phosphate, total protein, albumin, blood urea nitrogen, creatinine, total bilirubin, direct bilirubin, alkaline phosphatase, aspartate aminotransferase, alanine aminotransferase, gamma glutamyl transferase and creatine phosphokinase.
Measured: Week 48 to Week 127
Part B: Number of Participants With Clinically Significant Changes in Hematology Parameters
Blood samples were collected for the analysis of hematology parameters: hemoglobin (including mean corpuscular volume), mean corpuscular hemoglobin, mean corpuscular hemoglobin concentration, hematocrit, red blood cell count, total white blood cell count, platelet count. Differential blood counts, including basophils, eosinophils, neutrophils, lymphocytes, and monocytes
Measured: Week 48 to Week 127
Part B: Number of Participants With Clinically Significant Changes in Urinalysis
Urine samples were collected for the analysis of urinalysis parameters: Leucocytes, blood, nitrite, protein, urobilinogen, bilirubin, potential of Hydrogen (pH), specific gravity, ketones, glucose.
Measured: Week 48 to Week 127
Part B: Number of Participants With Clinically Significant Changes in Vital Parameters
Vital parameters including pulse rate, respiration rate, blood pressure, and temperature were measured in seated or recumbent for at least 5 minutes. Data for number of participants with abnormal clinically significant changes for vital signs have been presented.
Measured: Week 48 to Week 127
Part B: Number of Participants With Clinically Significant Changes in Electrocardiogram (ECG) Parameters
Twelve-lead ECGs were performed after participants has been recumbent for at least 5 minutes.
Measured: Week 48 to Week 127
Part B: Number of Participants With Clinically Significant Changes in Physical Examinations
Physical examinations included an evaluation of body systems, including but not limited to the following: skin; head, eyes, ears, nose, and throat; respiratory system; cardiovascular system; abdomen (liver, spleen); lymph nodes; neurological system; and musculoskeletal system.
Measured: Week 48 to Week 127
Secondary outcomes (11)
Part A: Change From Baseline in Quality of Life in Neurologic Disorders Upper Extremity (Neuro-QoL UE) Scale at Week 48
The Neuro-QoL Upper Extremity (UE) scale is a patient-reported outcome measure designed to assess upper limb function in individuals with neurologic conditions. It evaluates a participant's self-reported difficulty in performing activities for daily living (ADLs) involving digital, manual, and reach-related function and self-care. Responses are divided into 5 ordinal levels (1 = unable to do, 2 = with much difficulty, 3 = with some difficulty, 4 = with a little difficulty, 5 = without any difficulty). Lower scores indicate worse symptoms. Change from Baseline was calculated as the post-treatment value minus the value at Baseline.
Measured: Baseline (Day 1) and at Week 48
Part A: Number of Participants With Response to Patient's Global Impression of Change (PGIC) at Week 48
The Patient Global Impression of Change (PGIC) is a standard 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.
Measured: At Week 48
Part A: Change From Baseline in Whole Body (WB) Longitudinal Composite Muscle Fat Infiltration (MFI) of B Muscles at Week 48
The whole-body longitudinal composite muscle fat infiltration (MFI) of predefined B muscles is measured by musculoskeletal (MSK) magnetic resonance imaging (MRI). MFI quantifies the extent of fat replacement in muscle tissue, which is a key marker of disease progression in facioscapulohumeral muscular dystrophy (FSHD). The B muscles are a prespecified subset of muscles that are most relevant to FSHD progression. A decrease or smaller increase in MFI indicates slower disease progression or a potential treatment benefit. Change from Baseline was calculated as the post-treatment value minus the value at Baseline.
Measured: Baseline (Day 1) and at Week 48
Part A: Relative Change From Baseline in Average Shoulder Abductor Strength by Hand-held Quantitative Dynamometry at Week 48
Average shoulder abductor strength was assessed using hand-held dynamometry (HHD). Bilateral strength measurements were acquired from both upper limbs, with the average calculated for each participant. The relative change from baseline was expressed as a percentage. This evaluated the effect of losmapimod, relative to placebo, on muscle strength in individuals diagnosed with facioscapulohumeral muscular dystrophy (FSHD). Standardized procedures and equipment were employed across all study sites for strength testing, and trained personnel conducted all measurements to ensure consistency. Baseline is the last non-missing evaluation prior to first dose of study drug. Relative change from Baseline = 100 x (Post-baseline value - Baseline value) / (Baseline value).
Measured: Baseline (Day 1) and at Week 48
Part A: Number of Participants Serious TEAEs and TEAEs
Treatment-emergent adverse event is an AE that begins on or after the first dose of study drug and on or before the stop of study drug + 35 days or begins before the first dose of study drug and worsens on or after the first dose of study drug and on or before the stop of study drug + 35 days. A SAE is defined as an AE that results in any of the following outcomes: death; life-threatening adverse event; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions; congenital anomaly/birth defect.
Measured: Up to Week 48
Part A: Number of Participants With Clinically Significant Changes in Clinical Chemistry Parameters
Blood samples were collected for the analysis of clinical chemistry parameters including Glucose, sodium, potassium, calcium, inorganic phosphate, total protein, albumin, blood urea nitrogen, creatinine, total bilirubin, direct bilirubin, alkaline phosphatase, aspartate aminotransferase, alanine aminotransferase, gamma-glutamyltransferase and creatine phosphokinase
Measured: Up to Week 48
Part A: Number of Participants With Clinically Significant Changes in Hematology Parameters
Blood samples were collected for the analysis of hematology parameters: hemoglobin (including mean corpuscular volume), mean corpuscular hemoglobin, mean corpuscular hemoglobin concentration, hematocrit, red blood cell count, total white blood cell count, platelet count. Differential blood counts, including basophils, eosinophils, neutrophils, lymphocytes, and monocytes
Measured: Up to Week 48
Part A: Number of Participants With Clinically Significant Changes in Urinalysis
Urine samples were collected for the analysis of urinalysis parameters: Leucocytes, blood, nitrite, protein, urobilinogen, bilirubin, potential of Hydrogen (pH), specific gravity, ketones, glucose.
Measured: Up to Week 48
Part A: Number of Participants With Clinically Significant Changes in Vital Parameters
Vital parameters including pulse rate, respiration rate, blood pressure, and temperature were measured in seated or recumbent for at least 5 minutes. Data for number of participants with abnormal clinically significant changes for vital signs have been presented.
Measured: Up to Week 48
Part A: Number of Participants With Clinically Significant Changes in Electrocardiogram (ECG) Parameters
Twelve-lead ECGs was performed after participants has been recumbent for at least 5 minutes.
Measured: Up to Week 48
Part A: Number of Participants With Clinically Significant Changes in Physical Examinations
Physical examinations included an evaluation of body systems, including but not limited to the following: skin; head, eyes, ears, nose, and throat; respiratory system; cardiovascular system; abdomen (liver, spleen); lymph nodes; neurological system; and musculoskeletal system.
Measured: Up to Week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 May 2022
- Study start16 Jun 2022
- Primary completion19 Nov 2024
- Study completion19 Nov 2024
Why it stopped: Sponsor Decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Facioscapulohumeral Muscular Dystrophy (FSHD)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 260 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fulcrum Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05397470
- Sponsor's protocol ID
- 1821-FSH-301
- Status last verified
- Oct 2025
- First submitted
- 4 May 2022
- Last updated
- 10 Nov 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Upon publication of a manuscript describing the clinical trial data, Fulcrum will provide access to all collected, individual, de-identified participant data and related study documents (e.g., Study Protocol, Statistical Analysis Plan \[SAP\]) through a third party. Access will be granted upon request from qualified researchers and will be subject to specific criteria and conditions. Further details, including the IPD sharing URL, will be provided in an update to this posting.
Publications
Barbour AM, Sarov-Blat L, Cai G, Fossler MJ, Sprecher DL, Graggaber J, McGeoch AT, Maison J, Cheriyan J. Safety, tolerability, pharmacokinetics and pharmacodynamics of losmapimod following a single intravenous or oral dose in healthy volunteers. Br J Clin Pharmacol. 2013 Jul;76(1):99-106. doi: 10.1111/bcp.12063.
PubMed 23215699Han JJ, Kurillo G, Abresch RT, de Bie E, Nicorici A, Bajcsy R. Reachable workspace in facioscapulohumeral muscular dystrophy (FSHD) by Kinect. Muscle Nerve. 2015 Feb;51(2):168-75. doi: 10.1002/mus.24287. Epub 2014 Nov 19.
PubMed 24828906Mellion ML, Ronco L, Berends CL, Pagan L, Brooks S, van Esdonk MJ, van Brummelen EMJ, Odueyungbo A, Thompson LA, Hage M, Badrising UA, Raines S, Tracewell WG, van Engelen B, Cadavid D, Groeneveld GJ. Phase 1 clinical trial of losmapimod in facioscapulohumeral dystrophy: Safety, tolerability, pharmacokinetics, and target engagement. Br J Clin Pharmacol. 2021 Dec;87(12):4658-4669. doi: 10.1111/bcp.14884. Epub 2021 May 14.
PubMed 33931884Voermans NC, Statland JM, Hayward LJ, Rosenbohm A, Lopez de Munain A, Sacconi S, Leung DG, Badrising UA, Vissing J, Schoser B, Muelas N, Lochmuller H, Bugiardini E, Wang LH, Maggi L, Ragole T, Pestronk A, Hamel JI, Goyal NA, Korngut L, Naddaf E, Harper A, Shieh PB, Kornblum C, Sansone V, Genge A, Tasca G, Jiang J, Jouvin MH, Tawil R; REACH Investigators. A randomized, double-blind, placebo-controlled study of losmapimod in patients with facioscapulohumeral muscular dystrophy: Results of the REACH study. J Neuromuscul Dis. 2026 Feb 6:22143602261419558. doi: 10.1177/22143602261419558. Online ahead of print.
PubMed 41649965
Study at a glance
From the registry record, last updated by the sponsor on 10 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT05397470
- Acronym / sponsor ID
- 1821-FSH-301
- Keywords
- Facioscapulohumeral muscular dystrophy (FSHD), Facioscapulohumeral muscular dystrophy type 1 (FSHD 1), Facioscapulohumeral muscular dystrophy type 2 (FSHD 2), Muscular Dystrophy, Facioscapulohumeral Muscular Disorders, Musculoskeletal Diseases, Neuromuscular Diseases, REACH
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor Decision
- Enrolment
- 260 participants (actual)
- Lead sponsor
- Fulcrum TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 16 Jun 2022 (actual)
- Primary completion
- 19 Nov 2024 (actual)
- Study completion
- 19 Nov 2024 (actual)
- First posted
- 31 May 2022 (actual)
- Results
- Posted 10 Nov 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Part A: Placebo-controlled treatment period: Losmapimod | Experimental | Drug: LosmapimodParticipants will be randomized to receive losmapimod. |
| Part A: Placebo-controlled treatment period: Placebo | Placebo comparator | Drug: Placebo oral tabletParticipants will be randomized to receive placebo |
| Part B: Open-label extension | Experimental | Drug: LosmapimodParticipants will receive losmapimod, upon completion of all assessments for Part A. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (12 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LosmapimodLosmapimod 15 mg will be administered BID by mouth along with food. | Drug | Part A: Placebo-controlled treatment period: Losmapimod, Part B: Open-label extension |
| Placebo oral tabletPlacebo will be administered BID by mouth along with food. | Drug | Part A: Placebo-controlled treatment period: Placebo |
Primary outcome
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 (Day 1) and at Week 48
Participants are instructed to complete a simple set of standardized movements of each arm centered around the shoulder joint. These arm movements are captured and quantitated with the use of a video camera. 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. Baseline is the last non-missing evaluation prior to first dose of study drug. Change from Baseline was calculated as the post-treatment value minus the value at Baseline.
Part B: Number of Participants Reporting Serious Treatment Emergent Adverse Events (Serious TEAEs) and Non-serious TEAEs > 5%
Time frame: Week 48 to Week 127
Treatment-emergent adverse event is an AE that begins on or after the first dose of study drug and on or before the stop of study drug + 35 days or begins before the first dose of study drug and worsens on or after the first dose of study drug and on or before the stop of study drug + 35 days. A SAE is defined as an AE that results in any of the following outcomes: death; life-threatening adverse event; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions; congenital anomaly/birth defect.
Part B: Number of Participants With Clinically Significant Changes in Chemistry Parameters
Time frame: Week 48 to Week 127
Blood samples were collected for the analysis of chemistry parameters: Glucose, sodium, potassium, calcium, inorganic phosphate, total protein, albumin, blood urea nitrogen, creatinine, total bilirubin, direct bilirubin, alkaline phosphatase, aspartate aminotransferase, alanine aminotransferase, gamma glutamyl transferase and creatine phosphokinase.
Part B: Number of Participants With Clinically Significant Changes in Hematology Parameters
Time frame: Week 48 to Week 127
Blood samples were collected for the analysis of hematology parameters: hemoglobin (including mean corpuscular volume), mean corpuscular hemoglobin, mean corpuscular hemoglobin concentration, hematocrit, red blood cell count, total white blood cell count, platelet count. Differential blood counts, including basophils, eosinophils, neutrophils, lymphocytes, and monocytes
Part B: Number of Participants With Clinically Significant Changes in Urinalysis
Time frame: Week 48 to Week 127
Urine samples were collected for the analysis of urinalysis parameters: Leucocytes, blood, nitrite, protein, urobilinogen, bilirubin, potential of Hydrogen (pH), specific gravity, ketones, glucose.
Part B: Number of Participants With Clinically Significant Changes in Vital Parameters
Time frame: Week 48 to Week 127
Vital parameters including pulse rate, respiration rate, blood pressure, and temperature were measured in seated or recumbent for at least 5 minutes. Data for number of participants with abnormal clinically significant changes for vital signs have been presented.
Part B: Number of Participants With Clinically Significant Changes in Electrocardiogram (ECG) Parameters
Time frame: Week 48 to Week 127
Twelve-lead ECGs were performed after participants has been recumbent for at least 5 minutes.
Part B: Number of Participants With Clinically Significant Changes in Physical Examinations
Time frame: Week 48 to Week 127
Physical examinations included an evaluation of body systems, including but not limited to the following: skin; head, eyes, ears, nose, and throat; respiratory system; cardiovascular system; abdomen (liver, spleen); lymph nodes; neurological system; and musculoskeletal system.
11 secondary outcomes
Part A: Change From Baseline in Quality of Life in Neurologic Disorders Upper Extremity (Neuro-QoL UE) Scale at Week 48
Time frame: Baseline (Day 1) and at Week 48
Part A: Number of Participants With Response to Patient's Global Impression of Change (PGIC) at Week 48
Time frame: At Week 48
Part A: Change From Baseline in Whole Body (WB) Longitudinal Composite Muscle Fat Infiltration (MFI) of B Muscles at Week 48
Time frame: Baseline (Day 1) and at Week 48
Part A: Relative Change From Baseline in Average Shoulder Abductor Strength by Hand-held Quantitative Dynamometry at Week 48
Time frame: Baseline (Day 1) and at Week 48
Part A: Number of Participants Serious TEAEs and TEAEs
Time frame: Up to Week 48
Part A: Number of Participants With Clinically Significant Changes in Clinical Chemistry Parameters
Time frame: Up to Week 48
Part A: Number of Participants With Clinically Significant Changes in Hematology Parameters
Time frame: Up to Week 48
Part A: Number of Participants With Clinically Significant Changes in Urinalysis
Time frame: Up to Week 48
Part A: Number of Participants With Clinically Significant Changes in Vital Parameters
Time frame: Up to Week 48
Part A: Number of Participants With Clinically Significant Changes in Electrocardiogram (ECG) Parameters
Time frame: Up to Week 48
Part A: Number of Participants With Clinically Significant Changes in Physical Examinations
Time frame: Up to Week 48
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Nov 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 23215699Barbour AM, Sarov-Blat L, Cai G, Fossler MJ, Sprecher DL, Graggaber J, McGeoch AT, Maison J, Cheriyan J. Safety, tolerability, pharmacokinetics and pharmacodynamics of losmapimod following a single intravenous or oral dose in healthy volunteers. Br J Clin Pharmacol. 2013 Jul;76(1):99-106. doi: 10.1111/bcp.12063.
- Background: PubMed 24828906Han JJ, Kurillo G, Abresch RT, de Bie E, Nicorici A, Bajcsy R. Reachable workspace in facioscapulohumeral muscular dystrophy (FSHD) by Kinect. Muscle Nerve. 2015 Feb;51(2):168-75. doi: 10.1002/mus.24287. Epub 2014 Nov 19.
- Background: PubMed 33931884Mellion ML, Ronco L, Berends CL, Pagan L, Brooks S, van Esdonk MJ, van Brummelen EMJ, Odueyungbo A, Thompson LA, Hage M, Badrising UA, Raines S, Tracewell WG, van Engelen B, Cadavid D, Groeneveld GJ. Phase 1 clinical trial of losmapimod in facioscapulohumeral dystrophy: Safety, tolerability, pharmacokinetics, and target engagement. Br J Clin Pharmacol. 2021 Dec;87(12):4658-4669. doi: 10.1111/bcp.14884. Epub 2021 May 14.
- Derived from this study: PubMed 41649965Voermans NC, Statland JM, Hayward LJ, Rosenbohm A, Lopez de Munain A, Sacconi S, Leung DG, Badrising UA, Vissing J, Schoser B, Muelas N, Lochmuller H, Bugiardini E, Wang LH, Maggi L, Ragole T, Pestronk A, Hamel JI, Goyal NA, Korngut L, Naddaf E, Harper A, Shieh PB, Kornblum C, Sansone V, Genge A, Tasca G, Jiang J, Jouvin MH, Tawil R; REACH Investigators. A randomized, double-blind, placebo-controlled study of losmapimod in patients with facioscapulohumeral muscular dystrophy: Results of the REACH study. J Neuromuscul Dis. 2026 Feb 6:22143602261419558. doi: 10.1177/22143602261419558. Online ahead of print.
- Individual participant dataSharing planned: Yes. Upon publication of a manuscript describing the clinical trial data, Fulcrum will provide access to all collected, individual, de-identified participant data and related study documents (e.g., Study Protocol, Statistical Analysis Plan [SAP]) through a third party. Access will be granted upon request from qualified researchers and will be subject to specific criteria and conditions. Further details, including the IPD sharing URL, will be provided in an update to this posting.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
33 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Losmapimod in people with Facioscapulohumeral Muscular Dystrophy (FSHD), run by Fulcrum Therapeutics at 33 sites in 9 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 5 inclusion and 12 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 10 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 33 sites in United States, Canada, Germany, Spain, Denmark and 4 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 phase mean?
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 10 Nov 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?
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 (Day 1) and at Week 48). The study lists 8 primary outcomes in all. 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 NCT05397470, which also lists 4 linked publications.
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 10 Nov 2025.