Randomized, Multicenter, Double-Blind, Placebo-Controlled, Efficacy, Safety, and Pharmacokinetic Study of ACTIMMUNE® (Interferon γ-1b) in Children and Young Adults With Friedreich's Ataxia
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Amgen
- 入组人数
- 92
- 试验地点
- 4
- 主要终点
- Change From Baseline to Week 26 in the Friedreich's Ataxia Rating Scale (FARS)-mNeuro Score
研究概览
简要总结
The purpose of this phase 3 randomized, multi-center, double-blind, placebo-controlled study is to evaluate the efficacy and safety of ACTIMMUNE® (interferon-γ 1b) in the treatment of Friedreich's Ataxia (FA) and to evaluate the pharmacokinetic (PK) characteristics of ACTIMMUNE® in FA patients.
详细描述
Study with completed results acquired from Horizon in 2024.
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
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 10 Years 至 25 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Written informed consent and child assent, if applicable.
- •FA confirmed by genetic testing with two expanded guanine-adenine-adenine (GAA) repeats.
- •FA functional stage of >1 to <5 and ability to walk 25 feet with or without an assistive device.
- •Male or female subject between the ages of 10 and 25 years, inclusive.
- •If female, the subject is not pregnant or lactating or intending to become pregnant during the study, or within 30 days after the last dose of study drug. Female subjects of child-bearing potential must have a negative serum pregnancy test result at Screening, a negative urine pregnancy test result at Baseline, and agree to use a reliable method of contraception throughout the study and for 30 days after the last dose of study drug.
排除标准
- •Any unstable illness that in the investigator's opinion precludes participation in the study.
- •Use of any investigational product within 30 days prior to randomization.
- •A history of substance abuse.
- •Presence of clinically significant cardiac disease (as determined by the investigator based on electrocardiogram [ECG] and echocardiogram results at Screening). Specifically, an ejection fraction of <40% or a prolonged QT interval (>50% of cycle duration) will result in exclusion. If the investigator notes any other clinically significant abnormalities on the ECG or echocardiogram, the subject may be eligible if they are provided clearance from a cardiologist.
- •History of hypersensitivity to interferon (IFN)-ɣ or E. coli-derived products.
- •Presence of moderate or severe renal disease (estimated creatinine clearance <50 mL/min) or hepatic disease (aspartate aminotransferase [AST] or alanine aminotransferase [ALT] >2x the upper limit of normal) as evidenced by laboratory results at Screening.
- •Clinically significant abnormal white blood cell count, hemoglobin, or platelet count as evidenced by laboratory test results at Screening.
研究组 & 干预措施
Interferon γ-1b
Approximately 45 participants will receive subcutaneous (SC) doses of ACTIMMUNE® 3 times a week (TIW) for a total of 26 weeks.
干预措施: Interferon γ-1b (Drug)
Placebo
Approximately 45 participants will receive SC doses of placebo TIW for a total of 26 weeks.
干预措施: Placebo (Drug)
结局指标
主要结局
Change From Baseline to Week 26 in the Friedreich's Ataxia Rating Scale (FARS)-mNeuro Score
时间窗: Baseline, Week 26
The FARS assessment includes neurological signs that specifically reflect neural substrates affected in FA. Based on a neurological examination, bulbar, upper limb, lower limb, peripheral nerve, and upright stability/gait functions were assessed. The FARS-mNeuro score excludes the peripheral nervous system subscale score and the facial and tongue atrophy and fasciculations from the bulbar subscale score. Scores range from 0 (normal) to 93 (most impairment). A negative change from baseline is an improvement.
次要结局
- Change From Baseline to Week 26 in Total Friedreich Ataxia Rating Scale Score (FARStot)(Baseline, Week 26)
- Change From Baseline to Week 26 in Activities of Daily Living (ADL) Score(Baseline, Week 26)
- Change From Baseline at Week 26 in Timed 25-Foot Walk (T25FW)(Baseline, Week 26)
- Number of FARS-mNeuro Responders and Non-Responders at Week 26(Week 26)