EUCTR2019-000305-79-ES进行中（未招募）1 期
An Open-Label Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients with Facioscapulohumeral Muscular Dystrophy (FSHD) Previously Enrolled in Study A083-02 and in Patients with Charcot-Marie Tooth (CMT) Disease Types 1 and X Previously Enrolled in Study A083-03
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 150
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
入选标准
- •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
排除标准
- •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
研究者
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