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Losmapimod in FSHD1
NCT04004000Phase 2
Key facts
- Status
- Terminated
- Start
- Aug 2019
- Primary completion
- Oct 2024
- Countries
- 1
- Sponsor
- Fulcrum Therapeutics
- Modality
- Small Molecules
- Drugs
- losmapimod
- Conditions
- facioscapulohumeral muscular dystrophy type 1 (FSHD1)
01
Overview
Phase 2, open-label, single-arm trial of losmapimod in facioscapulohumeral muscular dystrophy type 1 (FSHD1), sponsored by Fulcrum Therapeutics. Primary endpoints: Treatment Emergent Adverse Events at 68 weeks, Clinically Significant Changes in Chemistry Parameters at 68 weeks and 2 more. Started Aug 2019; status: terminated.
02
Design
1 arm- Study design
- Non-randomizedOpen-labelSingle group
Treatment
FSHD1 subjects receiving losmapimod 15 mg BID (30 mg daily)
03
Who it enrols
Adults aged 18-65 with genetically confirmed FSHD1 (1-9 D4Z4 repeats), Clinical Severity Score 2-4 on Ricci's Scale, able to complete functional assessments, with MRI-eligible muscle for biopsy, excluding those with significant comorbidities, cardiac abnormalities, or recent investigational drug use
04
Endpoints
Primary
Treatment Emergent Adverse Events
Clinically Significant Changes in Chemistry Parameters
Clinically Significant Changes in Hematology Parameters
Clinically Significant Changes in Serum Coagulation Parameters
Secondary
Percent Change From Baseline in the Ratio of Phosphorylated HSP27 to Total HSP27 in Sorbitol-stimulated Whole Blood
Percent Change From Baseline in Ratio of Phosphorylated HSP27 to Total HSP27 in Muscle
Plasma Concentration of Losmapimod
Muscle Concentration of Losmapimod
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