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. - Evaluation of the safety and tolerability of FTX-1821 for FSHD

Fulcrum Therapeutics0 个研究点目标入组 30 人开始时间: 待定最近更新: 2024年4月15日

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•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.

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