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This is a study to evaluate the safety and efficacy of Losmapimod in treating patients with Facioscapulohumeral Muscular Dystrophy (FSHD) over 48 weeks.
This study is a Phase 2, randomized, double-blind, placebo-controlled, parallel-group, multicenter study designed to evaluate the efficacy and safety of losmapimod in treating patients with Facioscapulohumeral Muscular Dystrophy (FSHD) over 48 weeks. Patients will participate in this study for approximately 53 weeks. This will include a 4-week screening period, a 48-week, placebo-controlled treatment period and a 7 day safety follow-up period. Patients must have a confirmed diagnosis of FSHD1 and genetic confirmation must be obtained prior to the screening MRI and baseline muscle biopsy. Patients will be randomized to receive 15 mg of losmapimod or placebo twice daily given as two 7.5 mg tablets per dose by mouth; for a total of 4 pills or 30 mg daily for 48 weeks. All patients will be asked to attend the study clinic for each scheduled visit.
The primary endpoint of the study is to evaluate the efficacy of losmapimod in inhibiting or reducing expression of DUX4, as measured by a subset of DUX4-regulated gene transcripts in skeletal muscle biopsies of FSHD patients. Secondary endpoints include evaluation of the safety and tolerability of losmapimod, the plasma concentrations of losmapimod, levels of losmapimod in skeletal muscle and losmapimod target engagement in blood and skeletal muscle in FSHD patients.
This study was conducted during the Coronavirus Disease-2019 (COVID-19) Pandemic. The pandemic restrictions limited some assessments in the FSHD1 population in the clinic, including collection of some data for Week 24.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treatment | Experimental | FSHD1 patients with genetic confirmation will receive Losmapimod 15 mg twice daily given as two 7.5 mg tablets per dose by mouth; for a total of 4 pills or 30 mg daily for 48 weeks. |
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| Placebo | Placebo Comparator | FSHD1 patients with genetic confirmation will receive a Placebo twice daily given as two 7.5 mg tablets per dose by mouth; for a total of 4 pills or 30 mg daily for 48 weeks. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Losmapimod oral tablet | Drug | Losmapimod will be administered with food when possible. |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Double Homeobox 4 (DUX4) Activity in Affected Skeletal Muscle | 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]. | Baseline and Week 16 to Week 36 |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Type of Adverse Events (AEs) to Losmapimod | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Reachable Work Space (RWS) Dominant and Non-dominant Total Relative Surface Area (RSA) With Weight Quintant (Q)1 to Q5 | The RWS was a 3-dimensional sensor-based system (using a single depth-ranging sensor) that could unobtrusively detect an individual's RWS and reflect an individual global upper extremity function, including shoulder and proximal arm. Participants were seated in front of a 3D camera and asked to perform a standardized upper extremity movement protocol under the supervision of a study clinical evaluator with and without weights and on both the right and left arms at indicated time points. The absolute total RWS surface envelope area as well as areas for each of the quadrants was calculated and provided in a blinded fashion, with no access to treatment assignment information. The RWS RSA represented the portion of the unit hemisphere that was covered by an individual's hand movement. 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. |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Michelle Mellion, MD | Fulcrum Therapeutics | Study Director |
| 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. | |
| 21262998 | Background |
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This study was a randomized, double-blind placebo-controlled treatment period for 48 weeks which evaluated the efficacy and safety of losmapimod.
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| ID | Title | Description |
|---|---|---|
| FG000 | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | Participants were randomized to receive losmapimod tablets 15 mg (2×7.5 mg tablets/dose BID) orally (PO) for 48 weeks |
| FG001 | Placebo |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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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 | Apr 10, 2020 | Jan 16, 2024 |
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This study is a randomized, double-blind, placebo-controlled, 48-week parallel-group study.
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This study will be performed in a double-blind fashion. The investigator, study staff, subjects, sponsor and monitor will remain blinded to the treatment until study closure.
| Placebo oral tablet |
| Drug |
Placebo will be administered with food when possible |
|
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| Up to Week 48 |
| Number of Participants With Severity of AEs to Losmapimod | 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. | Up to Week 48 |
| Number of Participants With Relationship of AEs to Losmapimod | 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) | Up to Week 48 |
| Number of Participants With Adverse Events of Special Interest (AESIs) | 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. | Up to Week 48 |
| Number of Participants Who Prematurely Discontinued Study Drug Due to a Treatment Emergent Adverse Event (TEAE) | 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. | Up to Week 48 |
| Change From Baseline in Muscle Fat Fraction (MFF) 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. | Baseline and at Week 12, Week 24 and Week 48 |
| Change From Baseline in Lean Muscle Volume (LMV) 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. | Baseline and at Week 12, Week 24 and Week 48 |
| Change From Baseline in Muscle Fat Infiltration (MFI) 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. | Baseline and at Week 12, Week 24 and Week 48 |
| Plasma Concentration After Administration of Losmapimod | Blood samples were collected at indicated time points for pharmacokinetic (PK) analysis 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 |
| Concentration of Losmapimod in Skeletal Muscle Biopsy | Skeletal muscle biopsy samples were collected at indicated time points for the assessment of concentration of losmapimod. | Baseline, Week 16 and Week 36 |
| Percent Change From Baseline in Phosphorylated Heat Shock Protein 27 (pHSP27)/Total Heat Shock Protein 27 (tHSP27) | Blood samples were collected for Pharmacodynamic (PD) analysis of pHSP27/tHSP27. Baseline was defined as the last non-missing evaluation on or before the day of first dose of study drug. Percent change from Baseline was defined as value of post Baseline minus Baseline value divided by Baseline value and multiplied by 100. | Baseline and at Day 1: 4 hours post dose, Week 16: pre dose, Week 16: 4 hours post dose, Week 36 pre dose, Week 36: 4 hours post dose |
| Baseline and Week 4, Week 12, Week 24, Week 36 and Week 48 |
| Change From Baseline in Classic Timed Up and Go (TUG) Average Completion Time | The TUG test was a simple test that was used to assess a person's mobility and required both static and dynamic balance. It measured the time that a person takes to rise from a chair, walk 3 meters, turn around, walk back to the chair, and sit down. The optimized TUG test was the classic TUG but added the component of getting up from a laying position on a bed-like table in the clinic at the start of the test and laying back down on his or her back at the end of the test. Participants were timed as they started from a seated or laying position, rise to a standing position, walked a total of 6 meters and then returned to either a seated or laying position. 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 | Baseline and at Week 4, Week 12, Week 24, Week 36 and Week 48 |
| Change From Baseline in Facioscapulohumeral Muscular Dystrophy (FSDH) TUG Average Completion Time | The TUG test was a simple test that was used to assess a person's mobility and required both static and dynamic balance. It measured the time that a person takes to rise from a chair, walk 3 meters, turn around, walk back to the chair, and sit down. The optimized TUG test was the classic TUG but added the component of getting up from a laying position on a bed-like table in the clinic at the start of the test and laying back down on his or her back at the end of the test. Participants were timed as they started from a seated or laying position, rise to a standing position, walked a total of 6 meters and then returned to either a seated or laying position. 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 | Baseline and at Week 4, Week 12, Week 24, Week 36 and Week 48 |
| Change From Baseline in All Muscles Total Average Strength as Assessed by Hand-held Dynamometry | Quantitative isometric dynamometry (hand-held dynamometer) was used to assess the skeletal muscles strength of study participants in both the upper and lower limbs bilaterally. The MicroFET2 hand-held dynamometer was used to measure strength in the bilateral shoulders, elbow flexors and extensors, and ankle dorsiflexors. The Jamar Plus Digital Hand Dynamometer was used to measure bilateral grip strengths. 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. | Baseline and at Week 4, Week 12, Week 24, Week 36 and Week 48 |
| Change From Baseline in Motor Function Measure (MFM) Domain 1 Score | The MFM scale assessed the severity of the motor deficit as determined by an experienced physical therapist. The MFM domain 1 was a validated evaluator administered functional measure for neuromuscular disorders, with 13 items related to standing and transfers. Generic Values for each domain were: 0 = cannot perform the task, 1 = initiated the task, 2 - performs the movement incompletely, or completely but imperfectly, 3 = performs the task fully and 'normally'. Total score was the sum of all the domains for D1 divided by the maximum score possible and multiplied by 100. Min=0, Max = 100. Lower numbers on the scale represented a worse outcome. 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. | Baseline and at Week 48 |
| Change From Baseline in FSHD Health Index (HI) | The FSHD-HI was a 15-domain questionnaire designed and based on participant interviews to measure total FSHD health-related quality-of-life, including both motor impairment and the social and emotional impact of FSHD. 116 questions were combined into a total score, the score is transformed onto a percentage scale, with score ranging from 0 (no disability) to 100 (maximal disability); lower scores represented decreasing disability. Baseline was defined as last non-missing evaluation on or before the day of first dose of study drug. Change from Baseline was calculated as Baseline minus post-dose value. | Baseline and at Week 48 |
| Number of Participants With Improved and Not Improved Response to Patients' Global Impression of Change (PGIC) | The PGI-C was a one-time assessment to measure the participant's impression of the how their illness had changed over time. It is a single question that assessed on a scale of 1-7 if there has been an improvement, decline or no change in clinical status. The score ranged from: Responses of 1= Very much improved, 2= Much improved, and 3= Minimally improved which were considered as improved and responses of 4 = No change, 5 = Minimally worse, 6= Much worse, and 7=Very much worse were considered as not improved. Higher scores indicated worse symptoms. | At Week 48 |
| Los Angeles |
| California |
| 90095 |
| 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 |
| 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 | Columbus | Ohio | 43221 | United States |
| University of Utah | Salt Lake City | Utah | 84132 | United States |
| Virginia Commonwealth University | Richmond | Virginia | 23298 | United States |
| University of Washinton Medical Center | Seattle | Washington | 98195 | United States |
| Ottawa Hospital Research Institute | Ottawa | Ontario | K1Y 4E9 | Canada |
| Montreal Neurological Institute and Hospital | Montreal | Quebec | H3A 2B4 | Canada |
| CHU de NICE- CHU pasteur2 | Nice | 06001 | France |
| Hospital de la Sta Creu i St Pau | Barcelona | 08041 | Spain |
| Hospital UiP La Fe | Valencia | 46026 | Spain |
| Cheriyan 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. |
| 19728363 | Background | de Greef JC, Lemmers RJ, van Engelen BG, Sacconi S, Venance SL, Frants RR, Tawil R, van der Maarel SM. Common epigenetic changes of D4Z4 in contraction-dependent and contraction-independent FSHD. Hum Mutat. 2009 Oct;30(10):1449-59. doi: 10.1002/humu.21091. |
| 24828906 | Background | 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. |
| 28171552 | Background | Jagannathan S, Shadle SC, Resnick R, Snider L, Tawil RN, van der Maarel SM, Bradley RK, Tapscott SJ. Model systems of DUX4 expression recapitulate the transcriptional profile of FSHD cells. Hum Mol Genet. 2016 Oct 15;25(20):4419-4431. doi: 10.1093/hmg/ddw271. |
| 23873337 | Background | Statland 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. |
| 30312408 | Background | Wang LH, Friedman SD, Shaw D, Snider L, Wong CJ, Budech CB, Poliachik SL, Gove NE, Lewis LM, Campbell AE, Lemmers RJFL, Maarel SM, Tapscott SJ, Tawil RN. MRI-informed muscle biopsies correlate MRI with pathology and DUX4 target gene expression in FSHD. Hum Mol Genet. 2019 Feb 1;28(3):476-486. doi: 10.1093/hmg/ddy364. |
| 38631764 | Derived | Tawil R, Wagner KR, Hamel JI, Leung DG, Statland JM, Wang LH, Genge A, Sacconi S, Lochmuller H, Reyes-Leiva D, Diaz-Manera J, Alonso-Perez J, Muelas N, Vilchez JJ, Pestronk A, Gibson S, Goyal NA, Hayward LJ, Johnson N, LoRusso S, Freimer M, Shieh PB, Subramony SH, van Engelen B, Kools J, Leinhard OD, Widholm P, Morabito C, Moxham CM, Cadavid D, Mellion ML, Odueyungbo A, Tracewell WG, Accorsi A, Ronco L, Gould RJ, Shoskes J, Rojas LA, Jiang JG. Safety and efficacy of losmapimod in facioscapulohumeral muscular dystrophy (ReDUX4): a randomised, double-blind, placebo-controlled phase 2b trial. Lancet Neurol. 2024 May;23(5):477-486. doi: 10.1016/S1474-4422(24)00073-5. |
| 35039091 | Derived | Jagannathan S, de Greef JC, Hayward LJ, Yokomori K, Gabellini D, Mul K, Sacconi S, Arjomand J, Kinoshita J, Harper SQ. Meeting report: the 2021 FSHD International Research Congress. Skelet Muscle. 2022 Jan 17;12(1):1. doi: 10.1186/s13395-022-00287-8. |
Participants were randomized to receive matching placebo tablets PO BID for 48 weeks.
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| NOT COMPLETED |
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Full Analysis Set: included all participants who were randomly assigned to receive double-blind study drug in the placebo-controlled treatment period.
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| ID | Title | Description |
|---|---|---|
| BG000 | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | Participants were randomized to receive losmapimod tablets 15 mg (2×7.5 mg tablets/dose BID) orally (PO) for 48 weeks |
| BG001 | Placebo | Participants were randomized to receive matching placebo tablets PO BID 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 | ||||||||||
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| 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 | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Primary | Change From Baseline in Double Homeobox 4 (DUX4) Activity in Affected Skeletal Muscle | 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]. | Full Analysis Set. Only those participants with data available at the specified data points were analyzed. | Posted | Least Squares Mean | Standard Error | Delta threshold cycle (Ct) | Baseline and Week 16 to Week 36 |
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| Secondary | Number of Participants With Type of Adverse Events (AEs) to Losmapimod | 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. | Safety Analysis Set: included all participants who received any study drug. | Posted | Count of Participants | Participants | Up to Week 48 |
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| Secondary | Number of Participants With Severity of AEs to Losmapimod | 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. | Safety Analysis Set | Posted | Count of Participants | Participants | Up to Week 48 |
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| Secondary | Number of Participants With Relationship of AEs to Losmapimod | 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) | Safety Analysis Set | Posted | Count of Participants | Participants | Up to Week 48 |
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| Secondary | Number of Participants With Adverse Events of Special Interest (AESIs) | 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. | Safety Analysis Set | Posted | Count of Participants | Participants | Up to Week 48 |
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| Secondary | Number of Participants Who Prematurely Discontinued Study Drug Due to a Treatment Emergent Adverse Event (TEAE) | 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. | Safety Analysis Set | Posted | Count of Participants | Participants | Up to Week 48 |
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| Secondary | Change From Baseline in Muscle Fat Fraction (MFF) 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. | Full Analysis Set. Only those participants with data available at the specified data points were analyzed. | Posted | Mean | Standard Deviation | Percentage point | Baseline and at Week 12, Week 24 and Week 48 |
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| Secondary | Change From Baseline in Lean Muscle Volume (LMV) 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. | Full Analysis Set. Only those participants with data available at the specified data points were analyzed. | Posted | Mean | Standard Deviation | Liter | Baseline and at Week 12, Week 24 and Week 48 |
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| Secondary | Change From Baseline in Muscle Fat Infiltration (MFI) 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. | Full Analysis Set. Only those participants with data available at the specified data points were analyzed. | Posted | Mean | Standard Deviation | Percentage point | Baseline and at Week 12, Week 24 and Week 48 |
|
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| Secondary | Plasma Concentration After Administration of Losmapimod | Blood samples were collected at indicated time points for pharmacokinetic (PK) analysis of losmapimod. | 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. | Posted | Median | Full Range | Nanogram per milliliter | 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 |
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| Secondary | Concentration of Losmapimod in Skeletal Muscle Biopsy | Skeletal muscle biopsy samples were collected at indicated time points for the assessment of concentration of losmapimod. | Pharmacokinetics Analysis Set. Only those participants with data available at the specified data points were analyzed. | Posted | Median | Full Range | Nanogram per gram | Baseline, Week 16 and Week 36 |
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| Secondary | Percent Change From Baseline in Phosphorylated Heat Shock Protein 27 (pHSP27)/Total Heat Shock Protein 27 (tHSP27) | Blood samples were collected for Pharmacodynamic (PD) analysis of pHSP27/tHSP27. Baseline was defined as the last non-missing evaluation on or before the day of first dose of study drug. Percent change from Baseline was defined as value of post Baseline minus Baseline value divided by Baseline value and multiplied by 100. | Pharmacodynamics Analysis Set: included all participants who received at least 1 dose of losmapimod and have evaluable PD data for losmapimod. Only those participants with data available at the specified data points were analyzed. | Posted | Mean | Standard Deviation | Percent change | Baseline and at Day 1: 4 hours post dose, Week 16: pre dose, Week 16: 4 hours post dose, Week 36 pre dose, Week 36: 4 hours post dose |
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| Other Pre-specified | Change From Baseline in Reachable Work Space (RWS) Dominant and Non-dominant Total Relative Surface Area (RSA) With Weight Quintant (Q)1 to Q5 | The RWS was a 3-dimensional sensor-based system (using a single depth-ranging sensor) that could unobtrusively detect an individual's RWS and reflect an individual global upper extremity function, including shoulder and proximal arm. Participants were seated in front of a 3D camera and asked to perform a standardized upper extremity movement protocol under the supervision of a study clinical evaluator with and without weights and on both the right and left arms at indicated time points. The absolute total RWS surface envelope area as well as areas for each of the quadrants was calculated and provided in a blinded fashion, with no access to treatment assignment information. The RWS RSA represented the portion of the unit hemisphere that was covered by an individual's hand movement. 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. | Full Analysis Set. Only those participants with data available at the specified data points were analyzed. | Posted | Mean | Standard Deviation | Unitless | Baseline and Week 4, Week 12, Week 24, Week 36 and Week 48 |
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| Other Pre-specified | Change From Baseline in Classic Timed Up and Go (TUG) Average Completion Time | The TUG test was a simple test that was used to assess a person's mobility and required both static and dynamic balance. It measured the time that a person takes to rise from a chair, walk 3 meters, turn around, walk back to the chair, and sit down. The optimized TUG test was the classic TUG but added the component of getting up from a laying position on a bed-like table in the clinic at the start of the test and laying back down on his or her back at the end of the test. Participants were timed as they started from a seated or laying position, rise to a standing position, walked a total of 6 meters and then returned to either a seated or laying position. 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 | Full Analysis Set. Only those participants with data available at the specified data points were analyzed. | Posted | Mean | Standard Deviation | Seconds | Baseline and at Week 4, Week 12, Week 24, Week 36 and Week 48 |
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| Other Pre-specified | Change From Baseline in Facioscapulohumeral Muscular Dystrophy (FSDH) TUG Average Completion Time | The TUG test was a simple test that was used to assess a person's mobility and required both static and dynamic balance. It measured the time that a person takes to rise from a chair, walk 3 meters, turn around, walk back to the chair, and sit down. The optimized TUG test was the classic TUG but added the component of getting up from a laying position on a bed-like table in the clinic at the start of the test and laying back down on his or her back at the end of the test. Participants were timed as they started from a seated or laying position, rise to a standing position, walked a total of 6 meters and then returned to either a seated or laying position. 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 | Full Analysis Set. Only those participants with data available at the specified data points were analyzed. | Posted | Mean | Standard Deviation | Seconds | Baseline and at Week 4, Week 12, Week 24, Week 36 and Week 48 |
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| Other Pre-specified | Change From Baseline in All Muscles Total Average Strength as Assessed by Hand-held Dynamometry | Quantitative isometric dynamometry (hand-held dynamometer) was used to assess the skeletal muscles strength of study participants in both the upper and lower limbs bilaterally. The MicroFET2 hand-held dynamometer was used to measure strength in the bilateral shoulders, elbow flexors and extensors, and ankle dorsiflexors. The Jamar Plus Digital Hand Dynamometer was used to measure bilateral grip strengths. 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. | Full Analysis Set. Only those participants with data available at the specified data points were analyzed. | Posted | Mean | Standard Deviation | Kilograms | Baseline and at Week 4, Week 12, Week 24, Week 36 and Week 48 |
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| Other Pre-specified | Change From Baseline in Motor Function Measure (MFM) Domain 1 Score | The MFM scale assessed the severity of the motor deficit as determined by an experienced physical therapist. The MFM domain 1 was a validated evaluator administered functional measure for neuromuscular disorders, with 13 items related to standing and transfers. Generic Values for each domain were: 0 = cannot perform the task, 1 = initiated the task, 2 - performs the movement incompletely, or completely but imperfectly, 3 = performs the task fully and 'normally'. Total score was the sum of all the domains for D1 divided by the maximum score possible and multiplied by 100. Min=0, Max = 100. Lower numbers on the scale represented a worse outcome. 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. | Full Analysis Set. Only those participants with data available at the specified data points were analyzed. | Posted | Mean | Standard Deviation | Scores on a scale | Baseline and at Week 48 |
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| Other Pre-specified | Change From Baseline in FSHD Health Index (HI) | The FSHD-HI was a 15-domain questionnaire designed and based on participant interviews to measure total FSHD health-related quality-of-life, including both motor impairment and the social and emotional impact of FSHD. 116 questions were combined into a total score, the score is transformed onto a percentage scale, with score ranging from 0 (no disability) to 100 (maximal disability); lower scores represented decreasing disability. Baseline was defined as last non-missing evaluation on or before the day of first dose of study drug. Change from Baseline was calculated as Baseline minus post-dose value. | Full Analysis Set. Only those participants with data available at the specified data points were analyzed. | Posted | Mean | Standard Deviation | Scores on a scale | Baseline and at Week 48 |
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| Other Pre-specified | Number of Participants With Improved and Not Improved Response to Patients' Global Impression of Change (PGIC) | The PGI-C was a one-time assessment to measure the participant's impression of the how their illness had changed over time. It is a single question that assessed on a scale of 1-7 if there has been an improvement, decline or no change in clinical status. The score ranged from: Responses of 1= Very much improved, 2= Much improved, and 3= Minimally improved which were considered as improved and responses of 4 = No change, 5 = Minimally worse, 6= Much worse, and 7=Very much worse were considered as not improved. Higher scores indicated worse symptoms. | Full Analysis Set. | Posted | Count of Participants | Participants | At Week 48 |
|
|
Up to Week 48
Safety population which included all participants who received any study drug.
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 | Losmapimod 15 Milligrams (mg) Twice Daily (BID) | Participants were randomized to receive losmapimod tablets 15 mg (2×7.5 mg tablets/dose BID) orally (PO) for 48 weeks | 0 | 40 | 2 | 40 | 29 | 40 |
| EG001 | Placebo | Participants were randomized to receive matching placebo tablets PO BID for 48 weeks. | 0 | 40 | 0 | 40 | 23 | 40 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Postoperative wound infection | Infections and infestations | MedDRA 22.0 | Non-systematic Assessment |
| |
| Alcohol poisoning | Injury, poisoning and procedural complications | MedDRA 22.0 | Non-systematic Assessment |
| |
| Suicide attempt | Psychiatric disorders | MedDRA 22.0 | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Alcohol poisoning | Injury, poisoning and procedural complications | MedDRA 22.0 | Non-systematic Assessment |
| |
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA 22.0 | Non-systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 22.0 | Non-systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 22.0 | Non-systematic Assessment |
| |
| Foot fracture | Injury, poisoning and procedural complications | MedDRA 22.0 | Non-systematic Assessment |
| |
| Head injury | Injury, poisoning and procedural complications | MedDRA 22.0 | Non-systematic Assessment |
| |
| Incision site rash | Injury, poisoning and procedural complications | MedDRA 22.0 | Non-systematic Assessment |
| |
| Limb injury | Injury, poisoning and procedural complications | MedDRA 22.0 | Non-systematic Assessment |
| |
| Meniscus injury | Injury, poisoning and procedural complications | MedDRA 22.0 | Non-systematic Assessment |
| |
| Muscle strain | Injury, poisoning and procedural complications | MedDRA 22.0 | Non-systematic Assessment |
| |
| Post procedural contusion | Injury, poisoning and procedural complications | MedDRA 22.0 | Non-systematic Assessment |
| |
| Post procedural haematoma | Injury, poisoning and procedural complications | MedDRA 22.0 | Non-systematic Assessment |
| |
| Post procedural swelling | Injury, poisoning and procedural complications | MedDRA 22.0 | Non-systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA 22.0 | Non-systematic Assessment |
| |
| Seroma | Injury, poisoning and procedural complications | MedDRA 22.0 | Non-systematic Assessment |
| |
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA 22.0 | Non-systematic Assessment |
| |
| Skin laceration | Injury, poisoning and procedural complications | MedDRA 22.0 | Non-systematic Assessment |
| |
| Stress fracture | Injury, poisoning and procedural complications | MedDRA 22.0 | Non-systematic Assessment |
| |
| Abdominal discomfort | Gastrointestinal disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Bowel movement irregularity | Gastrointestinal disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Dental necrosis | Gastrointestinal disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Haemorrhoids | Gastrointestinal disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Stomatitis | Gastrointestinal disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Exostosis | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Haematoma muscle | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Joint swelling | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Mobility decreased | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Musculoskeletal stiffness | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Pain in jaw | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Tendon disorder | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA 22.0 | Non-systematic Assessment |
| |
| Folliculitis | Infections and infestations | MedDRA 22.0 | Non-systematic Assessment |
| |
| Gastrointestinal bacterial overgrowth | Infections and infestations | MedDRA 22.0 | Non-systematic Assessment |
| |
| Hordeolum | Infections and infestations | MedDRA 22.0 | Non-systematic Assessment |
| |
| Kidney infection | Infections and infestations | MedDRA 22.0 | Non-systematic Assessment |
| |
| Localised infection | Infections and infestations | MedDRA 22.0 | Non-systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 22.0 | Non-systematic Assessment |
| |
| Postoperative wound infection | Infections and infestations | MedDRA 22.0 | Non-systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 22.0 | Non-systematic Assessment |
| |
| Amnesia | Nervous system disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Balance disorder | Nervous system disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Postural tremor | Nervous system disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Angioedema | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Dermatitis acneiform | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Night sweats | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Psoriasis | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Skin depigmentation | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Yellow skin | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Initial insomnia | Psychiatric disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Stress | Psychiatric disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Suicide attempt | Psychiatric disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Chills | General disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Pain | General disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 22.0 | Non-systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA 22.0 | Non-systematic Assessment |
| |
| Blood iron decreased | Investigations | MedDRA 22.0 | Non-systematic Assessment |
| |
| Glycosylated haemoglobin increased | Investigations | MedDRA 22.0 | Non-systematic Assessment |
| |
| Dry eye | Eye disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Erythema of eyelid | Eye disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Tinnitus | Ear and labyrinth disorders | MedDRA 22.0 | Non-systematic Assessment |
| |
| Food allergy | Immune system disorders | MedDRA 22.0 | 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 | May 28, 2020 | Jan 16, 2024 | 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
| 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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|---|---|
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| Participants |
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| Participants |
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| Participants |
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| Participants |
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| Participants |
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Participants were randomized to receive matching placebo tablets PO BID for 48 weeks. |
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| Units |
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| Participants |
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