A Phase 1 Randomized, Double-blind, Placebo-controlled, Single-dose and Multiple-dose Study Evaluating Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of FTX-1821 in Healthy Adult Volunteers and in Patients with Facioscapulohumeral Muscular Dystrophy (FSHD), including an open-label patient cohort.
- Conditions
- Facioscapulohumeral muscular dystrophyLandouzy-Dejerine\u2019s disease10028302
- Registration Number
- NL-OMON48094
- Lead Sponsor
- Fulcrum Therapeutics
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 30
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).
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.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Part A: To determine safety and tolerability of a single dose of two different<br /><br>doses of FTX-1821 in healthy subjects, based on the assessment of adverse<br /><br>events (AEs), severe AEs (SAEs), clinically significant laboratory test<br /><br>results, ECGs and vital signs. Part B and C: To determine safety and<br /><br>tolerability of a multiple dosing of two different doses of FTX-1821 in FSHD<br /><br>patients, based on the assessment of adverse events (AEs), severe AEs (SAEs),<br /><br>clinically significant laboratory test results, ECGs and vital signs.</p><br>
- Secondary Outcome Measures
Name Time Method <p>Part A: Target engagement after a single dose of two different doses of<br /><br>FTX-1821 in healthy subjects after a single dose PK of FTX-1821 in healthy<br /><br>volunteers Exploratory analysis on serum will be done for discovery of FSHD<br /><br>biomarkers. Part B and C: Target engagement parameters derived from blood and<br /><br>muscle biopsy after multiple dosing of two different doses of FTX-1821 in FSHD<br /><br>patients. repeated dose PK of FTX-1821 in FSHD patients Exploratory analyses on<br /><br>muscle tissue will be done for discovery of FSHD biomarkers.</p><br>