NCT07798609尚未招募1 期
A Randomized, Double-Blind, Placebo-Controlled, Single and Multiple Ascending Dose Trial to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of SFL-0821 in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD)
Soufflé Therapeutics, Inc.7 个研究点 分布在 2 个国家目标入组 90 人开始时间: 2026年9月1日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 90
- 试验地点
- 7
- 主要终点
- Number of participants with treatment-emergent adverse events, serious adverse events, and any other dose-limiting toxicities as assessed by CTCAE v6.0.
研究概览
简要总结
The purpose of this study is to evaluate the safety, tolerability, and preliminary efficacy of SFL-0821 in adult patients with Facioscapulohumeral Muscular Dystrophy (FSHD)
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Genetically confirmed diagnosis of FSHD 1 or 2
- •FSHD Clinical Severity Score ≥1.5 to ≤4 at screening (5-point Ricci scale)
- •Ambulatory and able to walk 10 meters between ≥5 and ≤14 seconds (only assistive devices allowed are single cane or splints for ankle-foot orthoses)
- •Must have eligible lower extremity muscle for biopsy as determined from MRI by a central reader
排除标准
- •History of any illness or any clinical condition that, in the opinion of the Investigator, might confound the results of the study or pose an additional risk in administering investigational product to the participant.
- •History of biopsy of the same muscle within 30 days of the Screening biopsy or planning to undergo any non-study muscle biopsies over the duration of the study
- •Treatment with an oligonucleotide, gene therapy, or other experimental therapies for FSHD.
研究组 & 干预措施
SFL-0821
Experimental
SFL-0821 for Injection
干预措施: SFL-0821 for injection (Drug)
Placebo
Placebo Comparator
Placebo for Injection
干预措施: Placebo (Drug)
结局指标
主要结局
Number of participants with treatment-emergent adverse events, serious adverse events, and any other dose-limiting toxicities as assessed by CTCAE v6.0.
时间窗: Part A: Up to Week 24; Part B: Up to Week 48
次要结局
- Change from Baseline to Week 48 in 4-Step Stair Climb Test(Part B: Up to Week 48)
- Change from Baseline to Week 48 in Sit-to-Stand Test(Part B: Up to Week 48)
- Pharmacokinetics of SFL-0821: Maximum Observed Plasma Concentration (Cmax)(Part A and B: through 24 hours post dose (all cohorts))
- Pharmacokinetics of SFL-0821: Elimination half-life (T1/2 [h])(Part A and B: through 24 hours post dose (all cohorts))
- Pharmacokinetics of SFL-0821: Area under the concentration-time curve (AUC)(Part A and B: through 24 hours post dose (all cohorts))
- DUX4-regulated gene expression in skeletal muscle(Part A: Up to Week 24; Part B: Up to Week 48)
- Incidence of anti-drug antibodies (ADAs), and if positive, of neutralizing ADAs(Part A: Up to Week 24; Part B: Up to Week 48)
- Change from Baseline in quantitative skeletal muscle magnetic resonance imaging (MRI) at the cohort specified timepoints after dosing(Part B: Up to Week 48)
- Change from Baseline to Week 48 in 10-Meter Walk-Run-Test (10MWRT)(Part B: Up to Week 48)
- Change from Baseline to Week 48 in Timed Up-and-Go (TUG) Test(Part B: Up to Week 48)
研究者
研究点 (7)
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