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Sponsor Decision
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Part A: Placebo-controlled treatment period: Losmapimod | Experimental | Participants will be randomized to receive losmapimod. |
|
| Part A: Placebo-controlled treatment period: Placebo | Placebo Comparator | Participants will be randomized to receive placebo |
|
| Part B: Open-label extension | Experimental | Participants will receive losmapimod, upon completion of all assessments for Part A. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Losmapimod | Drug | Losmapimod 15 mg will be administered BID by mouth along with food. |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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. | 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. | Week 48 to Week 127 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Key Inclusion Criteria:
Key Exclusion Criteria:
Note that all other inclusion and exclusion criteria are listed in the protocol and only key are presented.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California Irvine | Irvine | California | 92868 | United States | ||
| University of California Los Angeles (UCLA) |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 23215699 | Background | 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. | |
| 24828906 | Background |
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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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Participants who completed 48 Weeks treatment period in Part A were enrolled in Part B. Part A of the study was completed as planned; however, Part B was terminated prematurely due to a Sponsor decision.
This was a two-part study. Part A was a global, randomized, double-blind, placebo-controlled, parallel-group, multicenter study that enrolled up to 260 participants. Part B was an open-label extension phase in which approximately 249 participants from Part A rolled over to receive Losmapimod treatment.
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| ID | Title | Description |
|---|---|---|
| FG000 | Part A: Losmapimod 15 Milligrams (mg) | Participants received Losmapimod 15 mg orally twice daily (BID) with food for 48 weeks. |
| FG001 | Part A: Placebo | Participants received matching placebo orally twice daily (BID) with food for 48 weeks. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Part A: PCT Period (Up to 48 Weeks) |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Dec 21, 2023 | Sep 2, 2025 |
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Part B of the study will be performed in an open-label fashion.
| Placebo oral tablet | Drug | Placebo will be administered BID by mouth along with food. |
|
| 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 |
| 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. | 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. | 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. | 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. | Week 48 to Week 127 |
| 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. | 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. | 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). | 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. | 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 | 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 | 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. | 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. | 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. | 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. | Up to Week 48 |
| Los Angeles |
| California |
| 90095 |
| United States |
| University of Colorado Anschutz Medical Campus | Aurora | Colorado | 80045 | United States |
| University of Florida | Gainesville | Florida | 32610 | United States |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | United States |
| Kennedy Krieger Institute | Baltimore | Maryland | 21205 | United States |
| University of Massachusetts Memorial Medical Center | Worcester | Massachusetts | 01655 | United States |
| Mayo Clinic | Rochester | Minnesota | 55905 | United States |
| Washington University School of Medicine | St Louis | Missouri | 63110 | United States |
| University of Rochester Medical Center | Rochester | New York | 14642 | United States |
| Ohio State University Medical Center | Columbus | Ohio | 43210 | United States |
| University of Utah | Salt Lake City | Utah | 84132 | United States |
| Virginia Commonwealth University | Richmond | Virginia | 23298 | United States |
| University of Washington Medical Center | Seattle | Washington | 98195 | United States |
| University of Calgary | Calgary | Alberta | T2N 4Z6 | Canada |
| The Ottawa Hospital Research Institute | Ottawa | Ontario | K1Y 4E9 | Canada |
| Montreal Neurological Institute and Hospital | Montreal | Quebec | H3A 2B4 | Canada |
| Aarhus Universitetshospital | Aarhus | 8200 | Denmark |
| Rigshospitalet | Copenhagen | 2100 | Denmark |
| Nice University Hospital - CHU Nice | Nice | PACA | 06001 | France |
| Institute de Myologie, Groupe Hospitalier Pitié-Salpêtrière | Paris | 75013 | France |
| University Hospital Bonn | Bonn | 53127 | Germany |
| LMU Klinikum Ludwig-Maximilians-Universität München | München | 80336 | Germany |
| Universitätsklinikum Ulm | Ulm | 89081 | Germany |
| Fondazione Serena Onlus- Centro Clinico NEMO | Milan | Lombardy | 20162 | Italy |
| Fondazione IRCCS Istituto Neurologico Carlo Besta | Milan | 20133 | Italy |
| Radboudumc | Nijmegen | Gelderland | 9101 | Netherlands |
| Leiden University Medical Centre | Leiden | Southern Holland | 2333 ZA | Netherlands |
| Hospital Universitario Donostia | San Sebastián | Guipuzkoa | 20014 | Spain |
| Hospital Universitario Vall d'Hebron | Barcelona | 08035 | Spain |
| Hospital Universitari i Politecnic La Fe | Valencia | 46026 | Spain |
| University College of London Hospitals | London | WC1N 3BG | United Kingdom |
| Newcastle upon Tyne NHS Foundation Trust | Newcastle upon Tyne | NE1 3BZ | United Kingdom |
| Han 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. |
| 33931884 | Background | Mellion 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. |
| 41649965 | Derived | Voermans 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. |
| FG002 | Part B: Losmapimod 15 mg OLE | Participants who completed Part A rolled over into Part B and received Losmapimod 15 mg orally twice daily (BID) with food. |
| COMPLETED |
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| NOT COMPLETED |
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| Part B: OLE Period (Up to Week 127) |
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Full Analysis Set comprised of all participants with FSHD1 or FSHD2 who are randomized and receive at least 1 dose of study drug in the placebo-controlled treatment period.
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| ID | Title | Description |
|---|---|---|
| BG000 | Losmapimod 15 mg | Participants received Losmapimod 15 mg orally twice daily (BID) with food for 48 weeks. |
| BG001 | Matching Placebo | Participants received matching placebo orally twice daily (BID) with food for 48 weeks. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
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| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Full Analysis Set comprised of all participants with FSHD1 or FSHD2 who are randomized and receive at least 1 dose of study drug in the placebo-controlled treatment period. Only those participants with data available at specified timepoints has been presented. | Posted | Least Squares Mean | Standard Error | Scores on a scale | Baseline (Day 1) and at Week 48 |
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| Primary | 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. | Open-Label Analysis Set comprises of all participants who received at least 1 dose of losmapimod in Part B. | Posted | Count of Participants | Participants | Week 48 to Week 127 |
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| Primary | 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. | Open-label Analysis Set. | Posted | Count of Participants | Participants | Week 48 to Week 127 |
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| Primary | 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 | Open-label Analysis Set. | Posted | Count of Participants | Participants | Week 48 to Week 127 |
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| Primary | 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. | Open-label Analysis Set | Posted | Count of Participants | Participants | Week 48 to Week 127 |
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| Primary | 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. | Open-label Analysis Set. | Posted | Count of Participants | Participants | Week 48 to Week 127 |
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| Primary | 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. | Open-label Analysis Set. | Posted | Count of Participants | Participants | Week 48 to Week 127 |
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| Primary | 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. | Open-label Analysis Set. | Posted | Count of Participants | Participants | Week 48 to Week 127 |
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| Secondary | 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. | Full Analysis Set. Only those participants with data available at specified timepoints has been presented. | Posted | Mean | Standard Deviation | Scores on a scale | Baseline (Day 1) and at Week 48 |
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| Secondary | 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. | Full Analysis Set. | Posted | Count of Participants | Participants | At Week 48 |
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| Secondary | 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. | Full Analysis Set. Only those participants with data available at specified timepoints has been presented. | Posted | Mean | Standard Deviation | Percent | Baseline (Day 1) and at Week 48 |
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| Secondary | 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). | Full Analysis Set. Only those participants with data available at specified timepoints has been presented. | Posted | Mean | Standard Deviation | Percent | Baseline (Day 1) and at Week 48 |
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| Secondary | 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. | Safety Analysis Set comprises all participants who received any study drug in the placebo-controlled treatment period. | Posted | Count of Participants | Participants | Up to Week 48 |
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| Secondary | 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 | Safety Analysis Set. | Posted | Count of Participants | Participants | Up to Week 48 |
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| Secondary | 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 | Safety Analysis Set. | Posted | Count of Participants | Participants | Up to Week 48 |
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| Secondary | 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. | Safety Analysis Set | Posted | Count of Participants | Participants | Up to Week 48 |
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| Secondary | 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. | Safety Analysis Set. | Posted | Count of Participants | Participants | Up to Week 48 |
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| Secondary | 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. | Safety Analysis Set. | Posted | Count of Participants | Participants | Up to Week 48 |
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| Secondary | 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. | Safety Analysis Set. | Posted | Count of Participants | Participants | Up to Week 48 |
|
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Part A: Up to 48 Weeks; Part B (OLE Period): Up to Week 127
Serious TEAE and TEAE > 5% has been presented for Safety Analysis Set in Part A and Open-label Analysis Set in Part B. Safety Analysis Set comprises of all participants who received any study drug in the placebo-controlled treatment period. Open-Label Analysis Set comprises of all participants who received at least 1 dose of losmapimod in Part B.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Part A: Placebo-controlled Treatment Period: Losmapimod | Participants received Losmapimod 15 mg orally twice daily (BID) with food for 48 weeks. | 0 | 130 | 5 | 130 | 122 | 130 |
| EG001 | Part A: Placebo-controlled Treatment Period: Placebo | Participants received matching placebo orally twice daily (BID) with food for 48 weeks. | 1 | 130 | 8 | 130 | 112 | 130 |
| EG002 | Part B: Open Label Treatment Period: Losmapimod | Participants who completed Part A rolled over into Part B and received Losmapimod 15 mg orally twice daily (BID) with food. | 0 | 249 | 7 | 249 | 94 | 249 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Atrial fibrillation | Cardiac disorders | MedDRA (26.1) | Non-systematic Assessment |
| |
| Extrasystoles | Cardiac disorders | MedDRA (26.1) | Non-systematic Assessment |
| |
| Supraventricular tachycardia | Cardiac disorders | MedDRA (26.1) | Non-systematic Assessment |
| |
| Retinal detachment | Eye disorders | MedDRA (26.1) | Non-systematic Assessment |
| |
| Colitis ischaemic | Gastrointestinal disorders | MedDRA (26.1) | Non-systematic Assessment |
| |
| Appendicitis | Infections and infestations | MedDRA (26.1) | Non-systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA (26.1) | Non-systematic Assessment |
| |
| Diverticulitis | Infections and infestations | MedDRA (26.1) | Non-systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (26.1) | Non-systematic Assessment |
| |
| Urosepsis | Infections and infestations | MedDRA (26.1) | Non-systematic Assessment |
| |
| Accidental exposure to product by child | Injury, poisoning and procedural complications | MedDRA (26.1) | Non-systematic Assessment |
| |
| Ankle fracture | Injury, poisoning and procedural complications | MedDRA (26.1) | Non-systematic Assessment |
| |
| Meniscus injury | Injury, poisoning and procedural complications | MedDRA (26.1) | Non-systematic Assessment |
| |
| Peroneal nerve injury | Injury, poisoning and procedural complications | MedDRA (26.1) | Non-systematic Assessment |
| |
| Post ablation syndrome | Injury, poisoning and procedural complications | MedDRA (26.1) | Non-systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA (26.1) | Non-systematic Assessment |
| |
| Lumbar spinal stenosis | Musculoskeletal and connective tissue disorders | MedDRA (26.1) | Non-systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (26.1) | Non-systematic Assessment |
| |
| Spinal stenosis | Musculoskeletal and connective tissue disorders | MedDRA (26.1) | Non-systematic Assessment |
| |
| Small cell lung cancer metastatic | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (26.1) | Non-systematic Assessment |
| |
| Lumbar radiculopathy | Nervous system disorders | MedDRA (26.1) | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| COVID-19 | Infections and infestations | MedDRA (26.1) | Non-systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (26.1) | Non-systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (26.1) | Non-systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (26.1) | Non-systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (26.1) | Non-systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (26.1) | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (26.1) | Non-systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (26.1) | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (26.1) | Non-systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA (26.1) | Non-systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (26.1) | Non-systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (26.1) | Non-systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA (26.1) | Non-systematic Assessment |
|
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Call Center | Fulcrum Therapeutics | 617-651-8853 | clinicaltrials@fulcrumtx.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Aug 7, 2024 | Sep 2, 2025 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D020391 | Muscular Dystrophy, Facioscapulohumeral |
| D009136 | Muscular Dystrophies |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| ID | Term |
|---|---|
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009422 | Nervous System Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
Not provided
Not provided
| ID | Term |
|---|---|
| C543534 | 6-(5-((cyclopropylamino)carbonyl)-3-fluoro-2-methylphenyl)-N-(2,2-dimethylprpyl)-3-pyridinecarboxamide |
Not provided
Not provided
Not provided
| Study closed/terminated |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
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