An extension study to the A083-02 and A083-03 studies that evaluate the long-term effect of the ACE-083 investigational product in patients with Facioscapulohumeral Muscular Dystrophy (FSHD) previously participated in study A083-02 and also in patients with Charcot-Marie Tooth (CMT) disease type 1 and X who previously participated in study A083-03.
- Conditions
- Facioscapulohumeral Muscular DystrophyMedDRA version: 20.0 Level: PT Classification code 10064087 Term: Facioscapulohumeral muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
- Registration Number
- EUCTR2019-000305-79-ES
- Lead Sponsor
- Acceleron Pharma Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Not Recruiting
- Sex
- Not specified
- Target Recruitment
- 150
1. 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
2. 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 a 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.
3. Ability to adhere to the study visit schedule/procedures and to understand and comply with protocol requirements
4. Signed written informed consent
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 150
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
1. 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
2. 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
3. Type 1 or type 2 diabetes mellitus
4. 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
5. Renal impairment (serum creatinine = 2 times the upper limit of normal [ULN])
6. Aspartate transaminase (AST) and/or alanine transaminase (ALT) = 3 times ULN
7. 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)
8. Severe deformity or ankle fixation that would sufficiently limit passive range of motion to affect functional assessments (TA subjects only)
9. Major surgery within 4 weeks prior to Study Day 1
10. 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
11. 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
12. 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)
13. 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)
14. 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
15. Any condition that would prevent the use of MRI or compromise the ability to obtain a clear and interpretable image of the treated muscles (e.g., knee/hip replacement metallic implants)
16. Known active substance abuse, including alcohol
17. History of sensitivity to protein pharmaceuticals
18. Female that is pregnant or lactating/breast-feeding
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method