Trial · NCT04004000
Evaluation of Safety, Tolerability, and Changes in Biomarker and Clinical Outcome Assessments of Losmapimod for FSHD1 With Extension
NCT04004000, Phase 2, Terminated, Fulcrum Therapeutics; results first posted 2025-12-02
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Evaluation of Safety, Tolerability, and Changes in Biomarker and Clinical Outcome Assessments of Losmapimod for FSHD1 With Extension. Trials, record NCT04004000. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04004000
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Evaluation of Safety, Tolerability, and Changes in Biomarker and Clinical Outcome Assessments of Losmapimod for FSHD1 With Extension
- NCT number
- NCT04004000
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 14 participants, actual
- Lead sponsor
- Fulcrum Therapeutics
- Conditions
- Facioscapulohumeral Muscular Dystrophy 1
- Interventions
- Losmapimod
- First posted
- 2019-07-01
- Last updated
- 2025-12-02
- Results first posted
- 2025-12-02
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants Reporting Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | Up to 68 Weeks | 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 TEAE is an AE that begins 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 worsens on or after the first dose of study drug and before the stop of study drug + 7 days. 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. |
| Primary | Number of Participants With Clinically Significant Changes in Chemistry Parameters | Up to 68 Weeks | Blood samples were collected for the analysis of chemistry parameters: Glucose, sodium, potassium, calcium, inorganic phosphate, total protein, albumin, blood urea nitrogen, creatinine, uric acid, total bilirubin, alkaline phosphatase, aspartate aminotransferase, alanine aminotransferase, gamma glutamyl transferase, lactate dehydrogenase and creatine phosphokinase. |
| Primary | Number of Participants With Clinically Significant Changes in Hematology Parameters | Up to 68 Weeks | 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 |
| Primary | Number of Participants With Clinically Significant Changes in Serum Coagulation Parameters | Up to 68 Weeks | Blood samples were collected for the analysis of Serum coagulation parameters: International normalized ratio, prothrombin time, activated partial thromboplastin time and fibrinogen. |
| Secondary | Percent Change From Baseline in the Ratio of Phosphorylated HSP27 (pHSP27) /Total HSP27 in Sorbitol-stimulated Whole Blood | Baseline and at Week 44 | Phosphorylated HSP27 in peripheral whole blood with ex-vivo sorbitol stimulation and in skeletal muscle (without sorbitol stimulation) was measured at specified time points. The pre-treatment value was considered as Baseline for change from Baseline calculations. The geometric mean for fold percent change from Baseline (and 95% confidence interval) in pHSP27/total HSP27 was calculated. Percent change from baseline = 100\*(exp(mean of change from baseline on the log-transformed scale) - 1). |
| Secondary | Percent Change From Baseline in Ratio of pHSP27/Total HSP27 in Muscle | Baseline and at Week 8 | Phosphorylated HSP27 in skeletal muscle (without sorbitol stimulation) was measured at specified time points. The pre-treatment value was considered as Baseline for change from Baseline calculations. The geometric mean for fold percent change from Baseline (and 95% confidence interval) in pHSP27/total HSP27 was calculated. Percent change from baseline = 100\*(exp(mean of change from baseline on the log-transformed scale) - 1). |
| Secondary | Plasma Concentration of Losmapimod | Post-dose at Baseline, Week 4, Week 8, Week 20, Week 32, Week 44, and Week 56 | Blood samples were collected to measure the plasma concentration of losmapimod at specified timepoints. |
| Secondary | Muscle Concentration of Losmapimod | Post dose at Baseline, Week 4, Week 8 and Week 44 | Muscle biopsies were collected to measure the concentration of losmapimod at specified timepoints. |
Source ClinicalTrials.gov
Outcomes8
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants Reporting Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | Up to 68 Weeks | Safety Analysis Set. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Clinically Significant Changes in Chemistry Parameters | Up to 68 Weeks | Safety Analysis Set. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Clinically Significant Changes in Hematology Parameters | Up to 68 Weeks | Safety Analysis Set | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Clinically Significant Changes in Serum Coagulation Parameters | Up to 68 Weeks | Safety Analysis Set | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Percent Change From Baseline in the Ratio of Phosphorylated HSP27 (pHSP27) /Total HSP27 in Sorbitol-stimulated Whole Blood | Baseline and at Week 44 | Safety Analysis Set. Only those participants with data available at specified time points has been presented. | Percent change | GEOMETRIC_MEAN |
| SECONDARY | Percent Change From Baseline in Ratio of pHSP27/Total HSP27 in Muscle | Baseline and at Week 8 | Safety Analysis Set. Only those participants with data available at specified time points has been presented. | Percent change | GEOMETRIC_MEAN |
| SECONDARY | Plasma Concentration of Losmapimod | Post-dose at Baseline, Week 4, Week 8, Week 20, Week 32, Week 44, and Week 56 | Safety Analysis Set. Only those participants with data available at specified timepoints has been presented. | Nanograms per milliliter | GEOMETRIC_MEAN |
| SECONDARY | Muscle Concentration of Losmapimod | Post dose at Baseline, Week 4, Week 8 and Week 44 | Safety Analysis Set. Only those participants with data available at specified time points has been presented. | Nanograms per milliliter | GEOMETRIC_MEAN |
Source ClinicalTrials.gov
Results by group18
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants Reporting Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | Any TEAE | 14 | Participants | |
| Number of Participants Reporting Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | Any serious TEAE | 2 | Participants | |
| Number of Participants With Clinically Significant Changes in Chemistry Parameters | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 0 | Participants | ||
| Number of Participants With Clinically Significant Changes in Hematology Parameters | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 0 | Participants | ||
| Number of Participants With Clinically Significant Changes in Serum Coagulation Parameters | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 0 | Participants | ||
| Percent Change From Baseline in the Ratio of Phosphorylated HSP27 (pHSP27) /Total HSP27 in Sorbitol-stimulated Whole Blood | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | -48.3 | 95% Confidence Interval -55.4 to -40.1 | Percent change | |
| Percent Change From Baseline in Ratio of pHSP27/Total HSP27 in Muscle | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 33.9 | 95% Confidence Interval -78.3 to 726.5 | Percent change | |
| Plasma Concentration of Losmapimod | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | Baseline | 58.923 | Geometric Coefficient of Variation 0.488 | Nanograms per milliliter |
| Plasma Concentration of Losmapimod | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | Week 4 | 92.737 | Geometric Coefficient of Variation 0.220 | Nanograms per milliliter |
| Plasma Concentration of Losmapimod | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | Week 8 | 83.006 | Geometric Coefficient of Variation 0.356 | Nanograms per milliliter |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Main Study Phase (Up to 52 Weeks) | STARTED | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 14 | |
| Main Study Phase (Up to 52 Weeks) | COMPLETED | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 14 | |
| Main Study Phase (Up to 52 Weeks) | NOT COMPLETED | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 0 | |
| Extension Phase (Up to 68 Weeks) | STARTED | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 12 | |
| Extension Phase (Up to 68 Weeks) | COMPLETED | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 0 | |
| Extension Phase (Up to 68 Weeks) | NOT COMPLETED | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 12 |
Source ClinicalTrials.gov
Adverse events107
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 | Palpitations | Cardiac disorders | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 2 | 14 | |
| other | Middle ear effusion | Ear and labyrinth disorders | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 1 | 14 | |
| other | Tinnitus | Ear and labyrinth disorders | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 1 | 14 | |
| other | Dry eye | Eye disorders | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 3 | 14 | |
| other | Eczema eyelids | Eye disorders | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 1 | 14 | |
| other | Eye irritation | Eye disorders | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 1 | 14 | |
| other | Lacrimation increased | Eye disorders | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 2 | 14 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 3 | 14 | |
| other | Colitis microscopic | Gastrointestinal disorders | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 1 | 14 | |
| other | Diarrhoea | Gastrointestinal disorders | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 2 | 14 |
1 to 10 of 107Source ClinicalTrials.gov
See all 107 adverse eventsAdverse event totals3
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 | 14 |
| Total, other adverse events | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 14 | 14 |
| Total, serious adverse events | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | 2 | 14 |
Source ClinicalTrials.gov
Papers5
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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/NCT04004000
GET https://conductscience.org/api/trials/v1/NCT04004000/events?page=2