Clinical Trials

Inclusion Criteria:

- Patients must be between 18 and 65 years of age, inclusive.

- Genetically confirmed diagnosis of FSHD 1 or FSHD 2.

- Clinical severity score of 2 to 4 (Ricci Score; Range 0-5), at screening. Patients who
are wheelchair-dependent or dependent on walker or wheelchair for activities are not
permitted to enroll in the study.

- Baseline total relative surface area (RSA) (Q1-Q4) without weight in the dominant
upper extremities (UE) assessed by reachable workspace (RWS) ≥ 0.2 and ≤ 0.7.

- At least one short tau inversion recovery (STIR) positive muscle in the lower
extremities.

- No contraindications to MRI.

Exclusion Criteria:

- Previously diagnosed cancer that has not been in complete remission for at least 5
years. Localized carcinomas of the skin and carcinoma in situ of the cervix that have
been resected or ablated for cure are not exclusionary.

- Patients who are on drug(s) or supplements that may affect muscle function, as
determined by the Investigator: patients must be on a stable dose of that drug(s) or
supplement for at least 3 months prior to the first dose of study drug and remain on
that stable dose for the duration of the study.

- Known active opportunistic or life-threatening infections including HIV and hepatitis
B or C.

- Acute or chronic history of liver disease.

- Known severe renal impairment.

- History of cardiac dysrhythmias requiring anti-arrhythmia treatment(s); or history or
evidence of abnormal ECGs.

- Use of another investigational product within 30 days or 5 half-lives (whichever is
longer) or currently participating in a study of an investigational device.

Eligibility last updated 6/3/22. Questions regarding updates should be directed to the study team contact.