2025-521012-18-00招募中3 期
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)
Avidity Biosciences Inc.20 个研究点 分布在 6 个国家目标入组 80 人开始时间: 2025年11月4日最近更新:
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 80
- 试验地点
- 20
- 主要终点
- Change from Baseline to Week 78 in 10MWRT velocity (m/sec)
研究概览
简要总结
To evaluate the efficacy of AOC 1020 on functional mobility
入排标准
- 年龄范围
- 18 years 至 65+ years（18-64 Years, 65+ Years）
- 接受健康志愿者
- 是
入选标准
- •Must have given written informed consent (signed and dated) and any authorizations required by local law and be willing and able to comply with all study requirements. When enrolling participants who are minors, it is necessary to also obtain consent from a legally designated representative and the participant will receive information in a way adapted to their age and mental maturity.
- •Male and females with FSHD confirmed by documented genetic diagnosis as defined below: - FSHD1 - FSHD2
- •18 to 70 years of age at time of informed consent
- •Able to walk independently (orthoses or ankle braces are allowed) for at least 10 meters.
排除标准
- •Females who are pregnant, breastfeeding, or planning to become pregnant during the study period or
- •Treatment with an oligonucleotide within 9 months of Screening
- •Blood or plasma donation within 16 weeks of Study Day 1
- •Recent history of or current drug or alcohol abuse in the opinion of the Investigator
- •History of multiple drug allergies or history of allergic reaction to any component of, or excipient in, the Study Drug
- •Presence or history of clinically significant illness, medical condition, or abnormal test result/finding that, in the opinion of the Investigator, could affect a participant’s safety or their ability to comply with study procedures and/or complete the study visit schedule. This may include, but is not limited to, a history of cardiovascular or central nervous system disease, neuromuscular diseases other than FSHD (e.g., myopathy, neuropathy, neuromuscular junction disorders), a cardiopulmonary condition, or a clinically significant mental disorder
- •Males or females not willing to comply with the contraceptive requirements
- •Screening laboratory results or any other clinically significant abnormalities in screening laboratory values that would render a participant unsuitable for inclusion.
- •BP > 140/90 mmHg at Screening
- •Anticipated survival less than 2 years
- •Evidence of current or chronic infection with hepatitis C, hepatitis B, or HIV (including chronic infection requiring ongoing treatment to maintain viral suppression)
- •Active infection requiring systemic antiviral or antimicrobial therapy that will not be completed prior to Study Day 1 or ongoing symptoms related to recent infection causing impairment to ADL in the opinion of the Investigator
- •Malignancy within 5 years, except for basal or squamous cell carcinoma, melanoma in situ of the skin, carcinoma in situ of the cervix, or other malignancies within 5 years that have been treated with curative intent and which are not expected to recur
- •Treatment with another investigational drug or biological agent within 1 month of Screening or 5 half-lives of the drug, whichever is longer; or participation or plan to participate in another interventional study with an investigational drug or device or other types of interventional studies (including those studying behavioral modification and/or physical therapy) while on study
结局指标
主要结局
Change from Baseline to Week 78 in 10MWRT velocity (m/sec)
Change from Baseline to Week 78 in 10MWRT velocity (m/sec)
次要结局
- 1. Change from Baseline to Week 78 in: o Timed-Up-and-Go (TUG) o NeuroQoL Upper Extremity Function o Quantitative Muscle Testing (QMT) total composite score (PPN)
- 2.1 Change from Baseline in: o 10MWRT velocity (m/sec) (excluding Week 78) o QMT total composite score (excluding Week 78) o TUG (excluding Week 78) o QMT of upper extremity composite score, lower extremity composite score, and individual muscle group scores (PPN) o Patient-reported Outcomes Measurement Information System instruments (PROMIS) (Physical Function) o PROMIS Fatigue
- 2.2 Change from Baseline in: o Worst Pain Numerical Rating Scale (NRS) o Patient Global Impression of Severity(PGI-S)/Patient Global Impression of Change (PGI-C) o Quality of Life in Neurological Disorders (NeuroQoL) Upper Extremity Function (excluding Week 78) o A DUX4-regulated circulating biomarker of of FSHD disease biology o Plasma CK
研究者
Avidity Biosciences Inc
Scientific
Avidity Biosciences Inc.
研究点 (20)
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