NCT07435129招募中2 期
A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter, 52-Week Study Evaluating the Efficacy and Safety of Apitegromab in Participants With Facioscapulohumeral Muscular Dystrophy (FORGE)
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 60
- 试验地点
- 1
- 主要终点
- Assess the efficacy of apitegromab compared with placebo in participants with FSHD
研究概览
简要总结
A randomized Phase 2 study to evaluate the efficacy and safety of apitegromab as a monotherapy in participant with FSHD
详细描述
This Phase 2, randomized, double-blind, placebo-controlled, multicenter study is designed to evaluate the efficacy, safety, and tolerability of apitegromab in participants with facioscapulohumeral muscular dystrophy (FSHD)
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
- Triple (Participant, Care Provider, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 60 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female participants, 18 to 60 years of age at the time of informed consent.
- •Genetic diagnosis of FSHD Type 1 or FSHD Type 2, confirmed with the appropriate documentation from an accredited laboratory
- •Clinical severity score of 1.5 to 3.0 (Ricci score; range 0 to 5), inclusive, at screening
- •Baseline 10-meter walk/run test time ≤5 seconds
排除标准
- •Prior history of a hypersensitivity reaction to a mAb or recombinant protein bearing an Fc domain (eg, a soluble receptor-Fc fusion protein), apitegromab, or excipients of apitegromab
- •Treatment with other investigational drugs in a clinical trial within 3 months or 5 half-lives, whichever is longer, before screening
- •Previous treatment with apitegromab, or with other anti-myostatin therapies, including activin receptor antagonists
- •Current or prior use of anabolic steroids, growth hormones, glucagon-like peptide-1 receptor agonist or other substances with known effects on muscle.
- •Use of therapies with potentially significant muscle effects (eg, androgens, insulin-like growth factor, growth hormone, systemic beta-agonist, botulinum toxin, or muscle relaxants or muscle-enhancing supplements) or potentially significant neuromuscular effects (eg, acetylcholinesterase inhibitors) within 60 days before screening
- •Use of systemic or corticosteroids within 60 days prior to screening. Inhaled or topical steroids are allowed.
- •Pregnant or breastfeeding.
- •Contraindications for MRI that may include, but are not limited to, certain implanted electronic devices, cochlear implants, metallic foreign bodies, vascular clips, and metallic implants; or claustrophobia, contrast agent allergies, inability to lie still, or external medical devices that may not be removed.
- •History of alcoholism, or illicit drug use (drugs that are illegal and have not been prescribed).
- •Taking medications that impede coagulation or platelet aggregation or has a history or active coagulopathy disorder.
- •Any acute or comorbid condition interfering with the well-being of the participant within 7 days prior to screening, including active systemic infection, the need for acute treatment, or inpatient observation due to any reason
研究组 & 干预措施
Apitegromab
Experimental
10 mg/kg IV
干预措施: Apitegromab (Drug)
Placebo
Placebo Comparator
Placebo IV
干预措施: Placebo (Drug)
结局指标
主要结局
Assess the efficacy of apitegromab compared with placebo in participants with FSHD
时间窗: 52 Weeks
Percent change from baseline in total lean muscle volume (LMV) as measured by full body magnetic resonance imaging (MRI) at week 52
次要结局
- Further assess the efficacy of apitegromab compared with placebo in participants with FSHD(24 Weeks)
- Further assess the efficacy of apitegromab compared with placebo in participants with FSHD(24 Weeks and 52 Weeks)
- Evaluate the pharmacokinetics of apitegromab in participants with FSHD(52 Weeks)
- Evaluate the pharmacodynamics of apitegromab in participants with FSHD(52 Weeks)
- Evaluate the safety and tolerability of apitegromab in participants with FSHD(52 Weeks)
- Evaluate the safety and tolerability of apitegromab in FSHD participants(52 Weeks)
研究者
研究点 (1)
Loading locations...
相似试验
尚未招募
3 期
A clinical trial involving a new drug combination for treatment of advanced lung cancer with KRAS G12C mutationCTRI/2026/01/100675Mirati Therapeutics Inc, a Bristol Myers Squibb Company630
尚未招募
3 期
APG-157 in Locally Advanced Head and Neck Squamous Cell CarcinomaHead and Neck (HNSCC)OropharyngealOral CavityOral Cavity CarcinomaOropharynx Squamous Cell CarcinomaOral Cavity Squamous Cell CarcinomaHead and Neck CancerNCT07667296Aveta Biomics, Inc.826
招募中
1 期
Bacteriophages for Adults With Cystic Fibrosis and Chronic Achromobacter Lung InfectionNCT07275905Ghady Haidar12
招募中
3 期
A Study to Assess Safety, Tolerability and Efficacy of Garetosmab versus Placebo Administered Intravenously (IV) in Adult Participants With Fibrodysplasia Ossificans Progressiva (FOP) (OPTIMA)Fibrodysplasia Ossificans Progressiva2023-508350-26-00Regeneron Pharmaceuticals Inc.24
招募中
2 期
A Chronic Pain Master Protocol (CPMP): A Study of LY4065967 in Participants With Chronic Low Back PainNCT07641504Eli Lilly and Company150