Facioscapulohumeral Muscular Dystrophy 1Phase 2FSHDNCT04004000
Evaluation of Safety, Tolerability, and Changes in Biomarker and Clinical Outcome Assessments of Losmapimod for FSHD1 With Extension
A Phase 2 study testing Losmapimod in people with Facioscapulohumeral Muscular Dystrophy 1, run by Fulcrum Therapeutics at 1 site in Netherlands.
Trial summary
This clinical trial is a study to evaluate the safety, tolerability, and changes in biomarker and clinical outcome assessments of Losmapimod for patients with Facioscapulohumeral Muscular Dystrophy 1 (FSHD1) with an open-label extension. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Facioscapulohumeral Muscular Dystrophy 1
- What is being tested?
- Losmapimod
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 1 siteNetherlands
- Study dates
- 23 Aug 2019to 31 Oct 2024
- Phase
- Phase 2
- 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
- FSHD1 subjects age 18-65 years.
- Subject will sign and date an informed consent form (ICF).
- Confirmed diagnosis of FSHD1 with 1 to 9 repeats via assessment of the size of the D4Z4 array on chromosome 4. Genetic confirmation must be obtained prior to the screening MRI and baseline muscle biopsy; genetic confirmation can come from previous testing if verified with appropriate documentation.
- Must be willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, scheduled needle muscle biopsies, and other study procedures.
- Both male and female subjects must be willing to practice an approved method of birth control.
- Clinical Severity Score between 2 and 4 on Ricci's Scale (scale range is from 0 to 5). Subjects that use a wheelchair or walker for any activity are not permitted to enroll in the study.
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- Commitment to complete the 2 visits for skeletal muscle needle biopsy and all visits for whole-body MRI.
- Able to complete the RWS, TUG, and FSHD PROs (FSHD-RODS and FSHD-HI) at the screening visit.
- Must have an MRI-eligible muscle for biopsy as determined by the central reader.
- Subject must complete the main study through the Week 60 visit in order to participate in the open-label extension study.
Who cannot join
- History of any illness or any clinical condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject. This may include, but is not limited to, history of relevant drug or food allergies; history of cardiovascular or central nervous system disease; history or presence of clinically significant pathology; clinically significant history of mental disease; and history of cancer, except for squamous cell skin cancer, basal cell skin cancer, and Stage 0 cervical carcinoma in situ (all 3 with no recurrence for the last 5 years).
- Subject has a known or clinically suspected infection with human immunodeficiency virus or hepatitis B or C viruses.
- Subject has current clinically significant liver (alanine aminotransferase > 2X upper limit of normal or total bilirubin >1.5 X upper limit of normal) or kidney (GFR < 30 mL/min/1.73m2) dysfunction.
- Subject screens positive for hepatitis B surface antigen, hepatitis C virus (HCV) antibody, or antibodies against human immunodeficiency viruses 1 and 2 (HIV 1/HIV 2 antibodies).
- Subject has any condition possibly affecting drug absorption (eg, gastrectomy, cholecystectomy, or other gastrointestinal tract surgery, except appendectomy).
- Subject has a standard 12-lead ECG demonstrating QT interval by Fredericia (QTcF) >450 msec for male subjects and QTcF >470 msec for female subjects at Screening. If QTcF exceeds 450 msec for males or 470 msec for females, the ECG will be repeated 2 more times, and the average of the 3 QTcF values will be used to determine the subject's eligibility.
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- Subject has a history of cardiac dysrhythmias requiring anti-arrhythmia treatment(s); or history or evidence of abnormal ECGs that, in the opinion of the investigator or Medical Monitor, would preclude the subject's participation in the study.
- Male subject has a female partner who is planning to become pregnant during the study or within 90 days after the last study drug dose.
- Subject has donated blood (of approximately 1 pint [500 mL] or more) or has had any significant loss of blood within 90 days before the first study drug dose, as determined by the investigator.
- Vaccination with a live attenuated vaccine within 6 weeks of randomisation.
- Subject has a history of alcohol, analgesic/opioid, and/or illicit drug abuse as defined by the American Psychiatric Association Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition, in the last 6 months before screening, or a positive test for drugs of abuse at screening.
- Subject has participated in a clinical trial in which they have received an investigational product within the following time period prior to enrolment in the current study: 30 days, 5 half-lives or twice the duration of the biological effect of the investigational product (whichever was longer).
- For subjects that are on drug(s) or supplements that may affect muscle function as determined by the treating physician or included in the list of drugs presented in Section 15: subjects must be on a stable dose of that drug(s) or supplement for at least 3 months prior to enrollment in the study and remain on that stable dose for the duration of the study (list of drugs presented in Section 15). Changes to the dose or treatment discontinuation during the study can only be done for strict medical reasons by the treating physician with clear documentation and notification to the Sponsor.
- Subject has a history of sensitivity to any of the study medications or components thereof, or a history of drug or other allergy that, in the opinion of the investigator or Medical Monitor, contraindicated their participation.
- Female subject is pregnant as determined by positive urine Human Chorionic Gonadotropin (HCG) test at Screening or prior to dosing.
- Female subject is lactating.
- Subject is unwilling or unable to follow the procedures outlined in the protocol.
- Subject has any contraindication for MRI (including severe claustrophobia and any shrapnel or metal implants in the body that are not MRI compatible).
- Subject was mentally or legally incapacitated up to 2 years prior to enrollment.
- Subject has abnormal laboratory results indicative of any significant medical disease that, in the opinion of the investigator or the medical monitor, would preclude the subject's participation in the study.
- Subject, or close relative of the subject, is the investigator or a subinvestigator, research assistant, pharmacist, study coordinator, or other staff directly involved with the conduct of the study at that site.
- Subject has taken any anticoagulants for at least 1 month and anti-platelet agents for at least 1 week before each muscle biopsy.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: An Open-Label Pilot Study of Losmapimod to Evaluate the Safety, Tolerability, and Changes in Biomarker and Clinical Outcome Assessments in Subjects With Facioscapulohumeral Muscular Dystrophy 1 (FSHD1) With Extension
This clinical trial is a study to evaluate the safety, tolerability, and changes in biomarker and clinical outcome assessments of Losmapimod for patients with Facioscapulohumeral Muscular Dystrophy 1 (FSHD1) with an open-label extension.
Read the detailed description
This study is a single-centre, open-label pilot study that will investigate the safety, tolerability, pharmacokinetics (PK), and target engagement during long-term dosing with losmapimod tablets in adult subjects with FSHD1. Subjects will be evaluated during an 8- week pre-treatment period (Visits 1 through 3) to establish pre-treatment baseline assessments. Subjects will then be treated with losmapimod for approximately 1 year (Visits 4 through 9) and assessed at relatively regular intervals for change from pre-treatment assessments. All subjects will undergo two muscle biopsies; one at baseline, pre-treatment (Visit 4, Week 8 ± 1 week) and the second on-treatment muscle biopsy approximately 4 or 8 weeks later (Visit 5, Week 14 ± 2 weeks). Up to 8 subjects will have an on-treatment biopsy at 4 weeks and up to 8 subjects will have the on-treatment biopsy at 8 weeks.
Only subjects who participated in and competed all study procedures in the OLS Study treatment period (Week 60) will be eligible to participate in the open-label extension study.
The extension of this study will enable continued investigation of the safety and tolerability of long-term dosing with losmapimod tablets in adult subjects with FSHD1. During the extension study, subjects will receive 15 mg of losmapimod by mouth twice daily for a total of 30 mg by mouth daily. All subjects will attend clinic visits approximately every 24 weeks and have a safety phone call 12 weeks between in-person clinic visits until 90 days after commercial drug is available post regulatory approval or until study termination.
The primary endpoint of the main study is to evaluate the safety and tolerability of long-term dosing of losmapimod tablets in subjects with FSHD1. Secondary endpoints include assessment of target engagement of losmapimod in blood and skeletal muscle and repeated dose pharmacokinetics in subjects with FSHD1 over long-term dosing.
The extension will continue investigation of efficacy with assessment of skeletal muscle by ultrasound as well as the safety, tolerability, pharmacokinetics (PK), and exploration of efficacy measures including whole body skeletal muscle MRI and selected clinical outcome assessments during long- term dosing with losmapimod tablets in adult subjects with FSHD1. Secondary endpoints include assessment of efficacy as evaluated by whole body skeletal muscle MRI parameters, safety and tolerability of long-term dosing, target engagement of losmapimod in blood and skeletal muscle and repeated dose pharmacokinetics in subjects with FSHD1 over long-term dosing.
- Conditions
- Facioscapulohumeral Muscular Dystrophy 1
- Being tested
- Losmapimod
- Keywords
- FSHD, FSHD1, Muscular Dystrophies, Facioscapulohumeral Muscular Disorders, Atrophic Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases
- Medical subject headings
- 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
The main study includes a treatment period of approximately one year. Subjects will receive 15 mg of losmapimod twice daily by mouth; for a total of 30 mg daily. The study drug should be taken with food and the date and time of each dose taken recorded in the subject diary.
Only subjects who participated in and completed all procedures for the main study (Week 60) will be eligible to participate in the extension.
For the extension, subjects will receive 15 mg of losmapimod by mouth twice daily for a total of 30 mg by mouth daily. Participation will continue until 90 days after commercial drug is available post regulatory approval or study termination.
| Study group | Role | What they receive |
|---|---|---|
| Treatment | Experimental | FSHD1 subjects with genetic confirmation will receive 15 mg of losmapimod twice daily given by mouth; for a total of 30 mg daily until 90 days after commercial drug is available post regulatory approval or study termination. Receives: Drug: Losmapimod |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Reporting Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs
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.
Measured: Up to 68 Weeks
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, uric acid, total bilirubin, alkaline phosphatase, aspartate aminotransferase, alanine aminotransferase, gamma glutamyl transferase, lactate dehydrogenase and creatine phosphokinase.
Measured: Up to 68 Weeks
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 68 Weeks
Number of Participants With Clinically Significant Changes in Serum Coagulation Parameters
Blood samples were collected for the analysis of Serum coagulation parameters: International normalized ratio, prothrombin time, activated partial thromboplastin time and fibrinogen.
Measured: Up to 68 Weeks
Secondary outcomes (4)
Percent Change From Baseline in the Ratio of Phosphorylated HSP27 (pHSP27) /Total HSP27 in Sorbitol-stimulated Whole Blood
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).
Measured: Baseline and at Week 44
Percent Change From Baseline in Ratio of pHSP27/Total HSP27 in Muscle
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).
Measured: Baseline and at Week 8
Plasma Concentration of Losmapimod
Blood samples were collected to measure the plasma concentration of losmapimod at specified timepoints.
Measured: Post-dose at Baseline, Week 4, Week 8, Week 20, Week 32, Week 44, and Week 56
Muscle Concentration of Losmapimod
Muscle biopsies were collected to measure the concentration of losmapimod at specified timepoints.
Measured: Post dose at Baseline, Week 4, Week 8 and Week 44
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jul 2019
- Study start23 Aug 2019
- Primary completion31 Oct 2024
- Study completion31 Oct 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 (5)Papers the sponsor has linked to this study.
- Other trials for Facioscapulohumeral Muscular Dystrophy 1Studies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- This is a single-centre, open-label pilot study with open-label extension
- Masking
- None (open label)
- Enrolment
- 14 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
- NCT04004000
- Sponsor's protocol ID
- FIS-001-2019
- Acronym
- FSHD
- Status last verified
- Nov 2025
- First submitted
- 25 Jun 2019
- Last updated
- 2 Dec 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
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 23215699Cheriyan J, Webb AJ, Sarov-Blat L, Elkhawad M, Wallace SM, Maki-Petaja KM, Collier DJ, Morgan J, Fang Z, Willette RN, Lepore JJ, Cockcroft JR, Sprecher DL, Wilkinson IB. Inhibition of p38 mitogen-activated protein kinase improves nitric oxide-mediated vasodilatation and reduces inflammation in hypercholesterolemia. Circulation. 2011 Feb 8;123(5):515-23. doi: 10.1161/CIRCULATIONAHA.110.971986. Epub 2011 Jan 24.
PubMed 21262998de Greef JC, Frants RR, van der Maarel SM. Epigenetic mechanisms of facioscapulohumeral muscular dystrophy. Mutat Res. 2008 Dec 1;647(1-2):94-102. doi: 10.1016/j.mrfmmm.2008.07.011. Epub 2008 Aug 3.
PubMed 18723032Han 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 24828906Statland JM, Tawil R. Risk of functional impairment in Facioscapulohumeral muscular dystrophy. Muscle Nerve. 2014 Apr;49(4):520-7. doi: 10.1002/mus.23949. Epub 2014 Feb 10.
PubMed 23873337
Study at a glance
From the registry record, last updated by the sponsor on 2 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Pilot Study of Losmapimod to Evaluate the Safety, Tolerability, and Changes in Biomarker and Clinical Outcome Assessments in Subjects With Facioscapulohumeral Muscular Dystrophy 1 (FSHD1) With Extension
- ClinicalTrials.gov ID
- NCT04004000
- Acronym / sponsor ID
- FSHD / FIS-001-2019
- Conditions
- Facioscapulohumeral Muscular Dystrophy 1
- Keywords
- FSHD, FSHD1, Muscular Dystrophies, Facioscapulohumeral Muscular Disorders, Atrophic Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor Decision
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Fulcrum TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 23 Aug 2019 (actual)
- Primary completion
- 31 Oct 2024 (actual)
- Study completion
- 31 Oct 2024 (actual)
- First posted
- 1 Jul 2019 (actual)
- Results
- Posted 2 Dec 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This clinical trial is a study to evaluate the safety, tolerability, and changes in biomarker and clinical outcome assessments of Losmapimod for patients with Facioscapulohumeral Muscular Dystrophy 1 (FSHD1) with an open-label extension.
Detailed description
This study is a single-centre, open-label pilot study that will investigate the safety, tolerability, pharmacokinetics (PK), and target engagement during long-term dosing with losmapimod tablets in adult subjects with FSHD1. Subjects will be evaluated during an 8- week pre-treatment period (Visits 1 through 3) to establish pre-treatment baseline assessments. Subjects will then be treated with losmapimod for approximately 1 year (Visits 4 through 9) and assessed at relatively regular intervals for change from pre-treatment assessments. All subjects will undergo two muscle biopsies; one at baseline, pre-treatment (Visit 4, Week 8 ± 1 week) and the second on-treatment muscle biopsy approximately 4 or 8 weeks later (Visit 5, Week 14 ± 2 weeks). Up to 8 subjects will have an on-treatment biopsy at 4 weeks and up to 8 subjects will have the on-treatment biopsy at 8 weeks.
Only subjects who participated in and competed all study procedures in the OLS Study treatment period (Week 60) will be eligible to participate in the open-label extension study.
The extension of this study will enable continued investigation of the safety and tolerability of long-term dosing with losmapimod tablets in adult subjects with FSHD1. During the extension study, subjects will receive 15 mg of losmapimod by mouth twice daily for a total of 30 mg by mouth daily. All subjects will attend clinic visits approximately every 24 weeks and have a safety phone call 12 weeks between in-person clinic visits until 90 days after commercial drug is available post regulatory approval or until study termination.
The primary endpoint of the main study is to evaluate the safety and tolerability of long-term dosing of losmapimod tablets in subjects with FSHD1. Secondary endpoints include assessment of target engagement of losmapimod in blood and skeletal muscle and repeated dose pharmacokinetics in subjects with FSHD1 over long-term dosing.
The extension will continue investigation of efficacy with assessment of skeletal muscle by ultrasound as well as the safety, tolerability, pharmacokinetics (PK), and exploration of efficacy measures including whole body skeletal muscle MRI and selected clinical outcome assessments during long- term dosing with losmapimod tablets in adult subjects with FSHD1. Secondary endpoints include assessment of efficacy as evaluated by whole body skeletal muscle MRI parameters, safety and tolerability of long-term dosing, target engagement of losmapimod in blood and skeletal muscle and repeated dose pharmacokinetics in subjects with FSHD1 over long-term dosing.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- This is a single-centre, open-label pilot study with open-label extension
| Arm | Type | Receives |
|---|---|---|
| Treatment | Experimental | Drug: LosmapimodFSHD1 subjects with genetic confirmation will receive 15 mg of losmapimod twice daily given by mouth; for a total of 30 mg daily until 90 days after commercial drug is available post regulatory approval or study termination. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- FSHD1 subjects age 18-65 years.
- Subject will sign and date an informed consent form (ICF).
- Confirmed diagnosis of FSHD1 with 1 to 9 repeats via assessment of the size of the D4Z4 array on chromosome 4. Genetic confirmation must be obtained prior to the screening MRI and baseline muscle biopsy; genetic confirmation can come from previous testing if verified with appropriate documentation.
- Must be willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, scheduled needle muscle biopsies, and other study procedures.
- Both male and female subjects must be willing to practice an approved method of birth control.
Key exclusion criteria (22 listed)
- History of any illness or any clinical condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject. This may include, but is not limited to, history of relevant drug or food allergies; history of cardiovascular or central nervous system disease; history or presence of clinically significant pathology; clinically significant history of mental disease; and history of cancer, except for squamous cell skin cancer, basal cell skin cancer, and Stage 0 cervical carcinoma in situ (all 3 with no recurrence for the last 5 years).
- Subject has a known or clinically suspected infection with human immunodeficiency virus or hepatitis B or C viruses.
- Subject has current clinically significant liver (alanine aminotransferase > 2X upper limit of normal or total bilirubin >1.5 X upper limit of normal) or kidney (GFR < 30 mL/min/1.73m2) dysfunction.
- Subject screens positive for hepatitis B surface antigen, hepatitis C virus (HCV) antibody, or antibodies against human immunodeficiency viruses 1 and 2 (HIV 1/HIV 2 antibodies).
- Subject has any condition possibly affecting drug absorption (eg, gastrectomy, cholecystectomy, or other gastrointestinal tract surgery, except appendectomy).
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 |
|---|---|---|
| LosmapimodThe main study includes a treatment period of approximately one year. Subjects will receive 15 mg of losmapimod twice daily by mouth; for a total of 30 mg daily. The study drug should be taken with food and the date and time of each dose taken recorded in the subject diary. Only subjects who participated in and completed all procedures for the main study (Week 60) will be eligible to participate in the extension. For the extension, subjects will receive 15 mg of losmapimod by mouth twice daily for a total of 30 mg by mouth daily. Participation will continue until 90 days after commercial drug is available post regulatory approval or study termination. | Drug | Treatment |
Primary outcome
Number of Participants Reporting Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs
Time frame: 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.
Number of Participants With Clinically Significant Changes in Chemistry Parameters
Time frame: 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.
Number of Participants With Clinically Significant Changes in Hematology Parameters
Time frame: 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
Number of Participants With Clinically Significant Changes in Serum Coagulation Parameters
Time frame: 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.
4 secondary outcomes
Percent Change From Baseline in the Ratio of Phosphorylated HSP27 (pHSP27) /Total HSP27 in Sorbitol-stimulated Whole Blood
Time frame: Baseline and at Week 44
Percent Change From Baseline in Ratio of pHSP27/Total HSP27 in Muscle
Time frame: Baseline and at Week 8
Plasma Concentration of Losmapimod
Time frame: Post-dose at Baseline, Week 4, Week 8, Week 20, Week 32, Week 44, and Week 56
Muscle Concentration of Losmapimod
Time frame: Post dose at Baseline, Week 4, Week 8 and Week 44
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Dec 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 21262998Cheriyan J, Webb AJ, Sarov-Blat L, Elkhawad M, Wallace SM, Maki-Petaja KM, Collier DJ, Morgan J, Fang Z, Willette RN, Lepore JJ, Cockcroft JR, Sprecher DL, Wilkinson IB. Inhibition of p38 mitogen-activated protein kinase improves nitric oxide-mediated vasodilatation and reduces inflammation in hypercholesterolemia. Circulation. 2011 Feb 8;123(5):515-23. doi: 10.1161/CIRCULATIONAHA.110.971986. Epub 2011 Jan 24.
- Background: PubMed 18723032de Greef JC, Frants RR, van der Maarel SM. Epigenetic mechanisms of facioscapulohumeral muscular dystrophy. Mutat Res. 2008 Dec 1;647(1-2):94-102. doi: 10.1016/j.mrfmmm.2008.07.011. Epub 2008 Aug 3.
- 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 23873337Statland JM, Tawil R. Risk of functional impairment in Facioscapulohumeral muscular dystrophy. Muscle Nerve. 2014 Apr;49(4):520-7. doi: 10.1002/mus.23949. Epub 2014 Feb 10.
- Individual participant dataSharing planned: No
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Losmapimod in people with Facioscapulohumeral Muscular Dystrophy 1, run by Fulcrum Therapeutics at 1 site in Netherlands.
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 10 inclusion and 22 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 2 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Netherlands. 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. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 2 Dec 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?
Number of Participants Reporting Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs (time frame: Up to 68 Weeks). The study lists 4 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 NCT04004000, which also lists 5 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 2 Dec 2025; AzTrials last refreshed its copy on 8 Nov 2026.