Facioscapulohumeral muscular dystrophy Landouzy-Dejerine\u2019s disease
Conditions
Interventions
FTX-1821 will be provided in capsules of two different doses for oral
administration. Matching placebo.
Sponsors
Fulcrum Therapeutics
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: Part A: Eligible subjects must meet all the following inclusion criteria at screening: 1. Healthy male / female subjects, 18 to 65 years of age, inclusive at screening;;Part B: 1. Male or female subjects of 18 to 65 years of age, inclusive at screening with a diagnosis of FSHD1 verified by (prior) genetic testing 2. Clinical Severity Score between 1 and 4.5 (inclusive) on Ricci's scale (scale range is from 0 to 5).;Part C: 1. Male or female subjects of 18 to 65 years of age, inclusive at screening with a diagnosis of FSHD1 verified by (prior) genetic testing 2. Clinical Severity Score between 1 and 4.5 (inclusive) on Ricci's scale (scale range is from 0 to 5).
Exclusion criteria
Exclusion criteria: Part A and B: 1. For healthy volunteers: Use of any medication (prescription or over-the-counter (OTC) within 14 days of study drug administration, or use of herbal supplements, dietary supplements or multivitamins within 7 days of study drug administration or less than five half-lives (whichever is longer), prior to study drug administration, with the exception of contraceptives, hormonal replacement therapies, and paracetamol (up to 3g/day). Other exceptions will only be made if the rationale is clearly documented in writing by the investigator;;For FSHD patients: Medications may be allowed if there is no interference with the study endpoints (safety or efficacy). The rationale will be clearly documented in writing by the investigator.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Part A: To determine safety and tolerability of a single dose of two different doses of FTX-1821 in healthy subjects, based on the assessment of adverse events (AEs), severe AEs (SAEs), clinically significant laboratory test results, ECGs and vital signs. Part B and C: To determine safety and tolerability of a multiple dosing of two different doses of FTX-1821 in FSHD patients, based on the assessment of adverse events (AEs), severe AEs (SAEs), clinically significant laboratory test results, ECGs and vital signs. | — |
Secondary
| Measure | Time frame |
|---|---|
| Part A: Target engagement after a single dose of two different doses of FTX-1821 in healthy subjects after a single dose PK of FTX-1821 in healthy volunteers Exploratory analysis on serum will be done for discovery of FSHD biomarkers. Part B and C: Target engagement parameters derived from blood and muscle biopsy after multiple dosing of two different doses of FTX-1821 in FSHD patients. repeated dose PK of FTX-1821 in FSHD patients Exploratory analyses on muscle tissue will be done for discovery of FSHD biomarkers. | — |
Countries
Netherlands
Outcome results
None listed