Trial · NCT04003974
Efficacy and Safety of Losmapimod in Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD)
NCT04003974, Phase 2, Completed, Fulcrum Therapeutics; results first posted 2024-07-10
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Efficacy and Safety of Losmapimod in Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD). Trials, record NCT04003974. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04003974
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Efficacy and Safety of Losmapimod in Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD)
- NCT number
- NCT04003974
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 80 participants, actual
- Lead sponsor
- Fulcrum Therapeutics
- Conditions
- Facioscapulohumeral Muscular Dystrophy (FSHD)
- Interventions
- Losmapimod oral tablet, Placebo oral tablet
- First posted
- 2019-07-01
- Last updated
- 2024-07-10
- Results first posted
- 2024-07-10
Source ClinicalTrials.gov
Registered outcomes19
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in Double Homeobox 4 (DUX4) Activity in Affected Skeletal Muscle | Baseline and Week 16 to Week 36 | Skeletal muscle biopsies were collected at Baseline and post-Baseline. DUX4 activity in skeletal muscle biopsies was assessed by measuring expression levels of a panel of 6 genes known to be regulated by DUX4. Expression levels of genes were measured using a validated quantitative RT-PCR assay and expressed as Ct (PCR cycles). Raw Ct for each of the 6 genes was normalized to the specified reference genes to generate a normalized Ct. The DUX4 activity is the average of the normalized Cts of each of the identified 6 genes, where the Ct for each of the 6 genes is first normalized to reference genes before the average is generated. Change in Baseline is calculated as \[average delta Ct across the 6 genes post-baseline\] minus \[average delta Ct across the 6 genes at baseline\]. |
| Secondary | Number of Participants With Type of Adverse Events (AEs) to Losmapimod | Up to Week 48 | An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of a study intervention, whether or not considered related to the study intervention. A Serious adverse event (SAE) is any untoward medical occurrence that, at any dose results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent disability/incapacity, is a congenital anomaly/birth defect or any other situation according to medical or scientific judgment. A treatment emergent adverse events (TEAE) is defined as any event that was not present before exposure to study drug or any event that was already present but worsens in either intensity or frequency after exposure to study drug. Number of participants with type of AEs to losmapimod has been presented which included: TEAEs and SAEs. |
| Secondary | Number of Participants With Severity of AEs to Losmapimod | Up to Week 48 | An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of a study intervention, whether or not considered related to the study intervention. An SAE is any untoward medical occurrence that, at any dose results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent disability/incapacity, is a congenital anomaly/birth defect or any other situation according to medical or scientific judgment. Number of participants with severity grading of AEs to losmapimod has been presented: Mild (An event that is usually transient in nature and generally does not interfere with normal activities), Moderate (An AE that is sufficiently discomforting to interfere with normal activities) and Severe (An AE that is incapacitating and prevents normal activities). The higher the grade, the more severe the symptoms. |
| Secondary | Number of Participants With Relationship of AEs to Losmapimod | Up to Week 48 | An AE is any untoward medical occurrence in a clinical study participant,temporally associated with use of a study intervention, whether or not considered related to study intervention. An SAE is any untoward medical occurrence that, at any dose results in death,is life-threatening,requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent disability/incapacity, is a congenital anomaly/birth defect or any other situation according to medical or scientific judgment. Number of participants with relationship of AEs to losmapimod has been presented:Unlikely related(most likely produced by other factors and temporal relationship of AE to drug makes a causal relationship unlikely),not related (no association between drug and AE), possibly related (treatment with drug caused/contributed to AE),probably related (reasonable temporal sequence of event with drug exists) and definitely related (definite causal relationship exists between drug and AE) |
| Secondary | Number of Participants With Adverse Events of Special Interest (AESIs) | Up to Week 48 | An AESI (serious or non-serious) is one of scientific and medical concern for which ongoing monitoring and rapid communication by the investigator to the sponsor can be appropriate. Adverse events of special interest for this study included liver tests that met the criteria for potential drug-induced liver injury (DILI), in accordance with the Unites States (US) Food and Drug Administration (FDA) Guidance for Industry-Drug-Induced Liver Injury: Premarketing Clinical Evaluation. Number of participants with AESIs has been presented. |
| Secondary | Number of Participants Who Prematurely Discontinued Study Drug Due to a Treatment Emergent Adverse Event (TEAE) | Up to Week 48 | TEAE was an AE that began on or after the first dose of study drug and before the stop of study drug + 7 days or begins before the first dose of study drug and worsened in severity on or after the first dose of study drug and before the stop of study drug + 7 days. An AE with completely missing onset and end dates were considered as treatment-emergent AE. Number of participants who prematurely discontinued study drug due to a TEAE has been presented. |
| Secondary | Change From Baseline in Muscle Fat Fraction (MFF) at Week 12, Week 24 and Week 48 | Baseline and at Week 12, Week 24 and Week 48 | Measurement of the extent of skeletal muscle tissue replacement by fat in FSHD participants were done through automatic skeletal muscle segmentation for the 3D muscle volumes and fat fraction analysis via robust algorithms using Dixon imaging. Composite variables, incorporating pre-selected muscles, were derived for longitudinal analysis of MFF. Baseline was defined as the last non-missing evaluation on or before the day of first dose of study drug. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value. |
| Secondary | Change From Baseline in Lean Muscle Volume (LMV) at Week 12, Week 24 and Week 48 | Baseline and at Week 12, Week 24 and Week 48 | Measurement of the extent of skeletal muscle tissue replacement by fat in FSHD participants were done through automatic skeletal muscle segmentation for the 3D muscle volumes and fat fraction analysis via robust algorithms using Dixon imaging. Composite variables, incorporating pre-selected muscles, were derived for longitudinal analysis of LMV. Baseline was defined as the last non-missing evaluation on or before the day of first dose of study drug. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value. |
| Secondary | Change From Baseline in Muscle Fat Infiltration (MFI) at Week 12, Week 24 and Week 48 | Baseline and at Week 12, Week 24 and Week 48 | Measurement of the extent of skeletal muscle tissue replacement by fat in FSHD participants were done through automatic skeletal muscle segmentation for the 3D muscle volumes and fat fraction analysis via robust algorithms using Dixon imaging. Composite variables, incorporating pre-selected muscles, were derived for longitudinal analysis of MFI. Baseline was defined as the last non-missing evaluation on or before the day of first dose of study drug. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value. |
| Secondary | Plasma Concentration After Administration of Losmapimod | Pre dose, 4 hours post dose, Week 4 pre dose, Week 4: 4 hours post dose, Week 12, Week 16 pre dose, Week 16: 4 hours post dose, Week 24, Week 36 pre dose, Week 36: 4 hours post dose, Week 48 | Blood samples were collected at indicated time points for pharmacokinetic (PK) analysis of losmapimod. |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 registered outcomesOutcomes19
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in Double Homeobox 4 (DUX4) Activity in Affected Skeletal Muscle | Baseline and Week 16 to Week 36 | Full Analysis Set. Only those participants with data available at the specified data points were analyzed. | Delta threshold cycle (Ct) | LEAST_SQUARES_MEAN |
| SECONDARY | Number of Participants With Type of Adverse Events (AEs) to Losmapimod | Up to Week 48 | Safety Analysis Set: included all participants who received any study drug. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Severity of AEs to Losmapimod | Up to Week 48 | Safety Analysis Set | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Relationship of AEs to Losmapimod | Up to Week 48 | Safety Analysis Set | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Adverse Events of Special Interest (AESIs) | Up to Week 48 | Safety Analysis Set | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants Who Prematurely Discontinued Study Drug Due to a Treatment Emergent Adverse Event (TEAE) | Up to Week 48 | Safety Analysis Set | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in Muscle Fat Fraction (MFF) at Week 12, Week 24 and Week 48 | Baseline and at Week 12, Week 24 and Week 48 | Full Analysis Set. Only those participants with data available at the specified data points were analyzed. | Percentage point | MEAN |
| SECONDARY | Change From Baseline in Lean Muscle Volume (LMV) at Week 12, Week 24 and Week 48 | Baseline and at Week 12, Week 24 and Week 48 | Full Analysis Set. Only those participants with data available at the specified data points were analyzed. | Liter | MEAN |
| SECONDARY | Change From Baseline in Muscle Fat Infiltration (MFI) at Week 12, Week 24 and Week 48 | Baseline and at Week 12, Week 24 and Week 48 | Full Analysis Set. Only those participants with data available at the specified data points were analyzed. | Percentage point | MEAN |
| SECONDARY | Plasma Concentration After Administration of Losmapimod | Pre dose, 4 hours post dose, Week 4 pre dose, Week 4: 4 hours post dose, Week 12, Week 16 pre dose, Week 16: 4 hours post dose, Week 24, Week 36 pre dose, Week 36: 4 hours post dose, Week 48 | Pharmacokinetics Analysis Set: included all participants who received at least 1 dose of losmapimod and had an evaluable PK data for losmapimod. Only those participants with data available at the specified data points were analyzed. | Nanogram per milliliter | MEDIAN |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 outcomesResults by group126
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in Double Homeobox 4 (DUX4) Activity in Affected Skeletal Muscle | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 0.8292 | Standard Error 0.6138 | Delta threshold cycle (Ct) | |
| Change From Baseline in Double Homeobox 4 (DUX4) Activity in Affected Skeletal Muscle | Placebo | 0.4008 | Standard Error 0.6491 | Delta threshold cycle (Ct) | |
| Number of Participants With Type of Adverse Events (AEs) to Losmapimod | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | Type: TEAEs | 29 | Participants | |
| Number of Participants With Type of Adverse Events (AEs) to Losmapimod | Placebo | Type: TEAEs | 23 | Participants | |
| Number of Participants With Type of Adverse Events (AEs) to Losmapimod | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | Type: SAEs | 2 | Participants | |
| Number of Participants With Type of Adverse Events (AEs) to Losmapimod | Placebo | Type: SAEs | 0 | Participants | |
| Number of Participants With Severity of AEs to Losmapimod | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | Severity: Mild | 18 | Participants | |
| Number of Participants With Severity of AEs to Losmapimod | Placebo | Severity: Mild | 15 | Participants | |
| Number of Participants With Severity of AEs to Losmapimod | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | Severity: Moderate | 9 | Participants | |
| Number of Participants With Severity of AEs to Losmapimod | Placebo | Severity: Moderate | 8 | Participants |
1 to 10 of 126Source ClinicalTrials.gov
See all 126 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 40 | |
| Overall Study | STARTED | Placebo | 40 | |
| Overall Study | COMPLETED | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 39 | |
| Overall Study | COMPLETED | Placebo | 38 | |
| Overall Study | NOT COMPLETED | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 1 | |
| Overall Study | NOT COMPLETED | Placebo | 2 |
Source ClinicalTrials.gov
Adverse events194
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Alcohol poisoning | Injury, poisoning and procedural complications | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 1 | 40 | |
| other | Alcohol poisoning | Injury, poisoning and procedural complications | Placebo | 0 | 40 | |
| other | Arthropod bite | Injury, poisoning and procedural complications | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 1 | 40 | |
| other | Arthropod bite | Injury, poisoning and procedural complications | Placebo | 0 | 40 | |
| other | Contusion | Injury, poisoning and procedural complications | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 1 | 40 | |
| other | Contusion | Injury, poisoning and procedural complications | Placebo | 3 | 40 | |
| other | Fall | Injury, poisoning and procedural complications | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 6 | 40 | |
| other | Fall | Injury, poisoning and procedural complications | Placebo | 2 | 40 | |
| other | Foot fracture | Injury, poisoning and procedural complications | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 1 | 40 | |
| other | Foot fracture | Injury, poisoning and procedural complications | Placebo | 0 | 40 |
1 to 10 of 194Source ClinicalTrials.gov
See all 194 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 0 | 40 |
| Total, all-cause mortality | Placebo | 0 | 40 |
| Total, other adverse events | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 29 | 40 |
| Total, other adverse events | Placebo | 23 | 40 |
| Total, serious adverse events | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 2 | 40 |
| Total, serious adverse events | Placebo | 0 | 40 |
Source ClinicalTrials.gov
Papers9
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons4
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Losmapimod 15 Milligrams (mg) Twice Daily (BID) vs Placebo: Number of Participants With Adverse Events of Special Interest (AESIs) | Trial comparisons |
| Losmapimod 15 Milligrams (mg) Twice Daily (BID) vs Placebo: deaths from any cause | Trial comparisons |
| Losmapimod 15 Milligrams (mg) Twice Daily (BID) vs Placebo: Number of Participants Who Prematurely Discontinued Study Drug Due to a Treatment Emergent Adverse Event (TEAE) | Trial comparisons |
| Losmapimod 15 Milligrams (mg) Twice Daily (BID) vs Placebo: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04003974
GET https://conductscience.org/api/trials/v1/NCT04003974/results?page=2