Intervention: N-{4-Hydroxy-3,5-bis[(piperidin-1-yl)methyl]phenyl}acetamide
Trial phase: Phase 2
SMILES: CC(=O)Nc1cc(CN2CCCCC2)c(O)c(CN2CCCCC2)c1
Sex: All
Age groups: Adult | Older Adult
Healthy volunteers: No
Participants at risk: 6
Group description: ACE-083 Administered into the TA muscle q8w for up to 24 months (12 doses) for FSHD patients

ACE-083: Recombinant fusion protein
Eligibility criteria:
Key Inclusion Criteria:

Completion of treatment with study drug per protocol and completion of the end of treatment (ET) visit in Study A083-02 or Study A083-03.
Females of childbearing potential (defined as sexually mature women who have not undergone hysterectomy or bilateral oophorectomy or are not naturally postmenopausal ≥ 24 consecutive months) must have negative urine pregnancy test prior to enrollment and use highly effective birth control methods (abstinence, oral contraceptives, barrier method with spermicide, or surgical sterilization) during study participation and for 8 weeks following the last dose of ACE-083. Hormonal birth control use must be stable for at least 14 days prior to Day 1. Males must agree to use a condom during any sexual contact with females of childbearing potential while participating in the study and for 8 weeks following the last dose of ACE-083, even if they have undergone a vasectomy. Subjects must be counseled about contraception prior to the first dose of ACE-083 and every three months thereafter during the study.
Ability to adhere to the study visit schedule/procedures and to understand and comply with protocol requirements
Signed written informed consent

Key Exclusion Criteria:

Current/active malignancy (e.g., remission less than 5 years' duration), with the exception of fully excised or treated basal cell carcinoma, cervical carcinoma in-situ, or ≤ 2 squamous cell carcinomas of the skin
Co-morbidities, including symptomatic cardiopulmonary disease, significant orthopedic or neuropathic pain, or other conditions that, in the opinion of the investigator, would limit a subject's ability to complete strength and/or functional assessments
Type 1 or type 2 diabetes mellitus
Thyroid disorder unless condition is stable with no change in treatment for at least 4 weeks before the first dose and no expected change for duration of study
Renal impairment (serum creatinine ≥ 2 times the upper limit of normal [ULN])
Aspartate transaminase (AST) and/or alanine transaminase (ALT) ≥ 3 times ULN
Increased risk of bleeding (i.e., due to hemophilia, platelet disorders, or use of any anticoagulation/platelet modifying therapies up to 2 weeks prior to Study Day 1 and for duration of study; single agent low dose aspirin [≤ 100 mg daily] is permitted)
Severe deformity or ankle fixation that would sufficiently limit passive range of motion to affect functional assessments (TA patients only)
Major surgery within 4 weeks prior to Study Day 1
Chronic pharmacologic doses of systemic corticosteroids (≥ 2 weeks) within 4 weeks before Study Day 1 and for duration of study; intra-articular/topical/inhaled/intranasal physiologic doses of systemic corticosteroids are permitted
Androgens, growth hormone, insulin or oral hormone replacement therapy within 6 months before Study Day 1 and for duration of study; topical physiologic androgen replacement is permitted. Chronic insulin therapy is permitted for diabetic FSHD patients. Oral HRT is permitted if started at least 3 months prior to receiving study drug
Any change in medications potentially affecting muscle strength or function within 4 weeks of Study Day 1 and for duration of study (e.g., creatinine, CoQ10, systemic beta-adrenergic agonists)
Previous exposure to any other investigational agent (not including ACE-083) potentially affecting muscle volume, muscle strength, or muscle or nerve function within 5 half-lives of last dose plus an additional 8-week washout period (or 12 weeks prior to Study Day 1 if half-life is unknown)
Significant change in physical activity or exercise (e.g., significant increase or decrease in intensity or frequency) within 8 weeks before Study Day 1 or inability to maintain the baseline level of physical activity throughout the study
Any condition that would prevent MRI scanning or compromise the ability to obtain a clear and interpretable scan of the treated muscles (e.g., knee/hip replacement metallic implants)
Known active substance abuse, including alcohol
History of sensitivity to protein pharmaceuticals
Female that is pregnant or lactating/breast-feeding