A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 200
- 试验地点
- 54
- 主要终点
- Change from Baseline to Week 78 in Quantitative Muscle Testing (QMT) total composite score (PPN)
研究概览
简要总结
A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)
详细描述
The study duration is approximately 22 months, consisting of a screening period of up to 6 weeks, a 78-week active treatment period, and a 12-week follow-up period (18 weeks from the last dose).
Participants will be randomized to receive an intravenous infusion of either delbrax or placebo at the clinical study site every 6 weeks for a total of 13 doses. The final dose will occur at Week 72, followed by a final assessment at Week 78.
After completion of the Week 78 visit, eligible participants will have the option to enroll in an open-label extension (OLE) study, pending regulatory approval. Participants who decline participation in the OLE will be followed for 12 weeks for safety.
An Independent Data Monitoring Committee (IDMC) comprising members independent and external to the Sponsor will review safety, tolerability, and efficacy (as needed) data of this study at regular intervals.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 16 Years 至 70 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical and genetic diagnosis of FSHD1 or FSHD2
- •Ability to walk independently at pre-specified walking speed (orthoses and ankle braces allowed) for at least 10 meters at screening
- •Adequate muscle strength based on QMT composite score
排除标准
- •Breastfeeding, pregnancy, or intent to become pregnant during the study
- •Unwilling or unable to comply with contraceptive requirements
- •Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study
- •Blood Pressure > 140/90 mmHg at Screening
- •Treatment with another investigational drug or biological agent within 1 month of Screening or 5 half-lives of the drug, whichever is longer
- •Treatment with an oligonucleotide within 9 months of Screening
结局指标
主要结局
Change from Baseline to Week 78 in Quantitative Muscle Testing (QMT) total composite score (PPN)
时间窗: Baseline through Week 78
Assessment of muscle strength using standardized quantitative measurements
Quantitative Muscle Testing (QMT) composite score
时间窗: Baseline through Week 78
次要结局
- Change from Baseline to Week 78 in Timed Up-and-Go (TUG)(Baseline through Week 78)
- Change from Baseline to Week 78 in 10MWRT(Baseline through Week 78)
- Change from Baseline to Week 78 in Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function Item Bank v2.0-Physical Function-Short Form 20a Questionnaire(Baseline through Week 78)
- Change from Baseline to Week 78 in PROMIS Fatigue(Baseline through Week 78)
- Change from Baseline to Week 78 in Worst Pain Numeric Rating Scale (NRS)(Baseline through Week 78)
- Change from Baseline to Week 78 in Patient Global Impression of Severity/Change (PGI-S/PGI-C)(Baseline through Week 78)
- Change from Baseline to Week 78 in Quality of Life in Neurological Disorders (NeuroQoL) Upper Extremity Function(Baseline through Week 78)
- Change from Baseline to Week 78 in NeuroQoL Sleep Disturbance(Baseline through Week 78)
- Change from Baseline to Week 78 in DUX4-regulated plasma KHDC1L(Baseline through Week 78)
- Change from Baseline to Week 78 in Serum CK(Baseline through Week 78)
- 10-Meter Walk/Run Test (10MWRT)(Baseline through Week 78)
- Timed Up-and-Go (TUG)(Baseline through Week 78)
- Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function Item Bank v2.0-Physical Function-Short Form 20a Questionnaire(Baseline through Week 78)
- Worst Pain Numeric Rating Scale (NRS)(Baseline through Week 78)
- Patient Global Impression of Severity/Change (PGI-S/PGI-C)(Baseline through Week 78)
- DUX-4 Regulated Circulating Biomarker of FSHD Disease Biology(Baseline through Week 78)
- Circulating Creatine Kinase (CK)(Baseline through Week 78)