2025-521628-31-00招募中3 期
A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects with Friedreich’s Ataxia
Larimar Therapeutics Inc.1 个研究点 分布在 1 个国家目标入组 18 人开始时间: 2026年5月31日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 18
- 试验地点
- 1
- 主要终点
- PRIMARY: • Difference between participant groups (see Study Design Section) on the Modified Friedreich’s Ataxia Rating Scale (mFARS) total score at study Week 72 compared with baseline
研究概览
简要总结
PRIMARY: • To learn if nomlabofusp improves movement ability in study participants (adults and children with FA) compared with placebo
研究设计
- 分配方式
- Randomized
- 主要目的
- Treatment
- 盲法
- Double (Carer, Investigator, Subject)
入排标准
- 年龄范围
- 0 years 至 64 years（0-17 Years, 18-64 Years）
- 接受健康志愿者
- 否
入选标准
- •Individuals (children and adults) aged 12 to 40, male or female, with a diagnosis of FA
- •Individuals capable of giving informed consent and completing physical function tests (see Objectives)
- •Individuals able to administer subcutaneous injections at home (with the help of a caregiver if needed)
- •Individuals using contraception, if able to conceive
排除标准
- •Previous treatment with nomlabofusp
- •Allergy to nomlabofusp ingredients, cetirizine, or famotidine
- •Pregnancy during the study
- •Severe diabetes, liver disease, or heart disease
- •Health conditions that could pose an increased risk to the participant or interfere with the understanding of the study results
- •Drug or alcohol use disorder
研究组 & 干预措施
Nomlabofusp
Test
干预措施: Nomlabofusp (Drug)
Placebo to Nomlabofusp
Placebo
干预措施: Placebo to Nomlabofusp (Drug)
结局指标
主要结局
PRIMARY: • Difference between participant groups (see Study Design Section) on the Modified Friedreich’s Ataxia Rating Scale (mFARS) total score at study Week 72 compared with baseline
PRIMARY: • Difference between participant groups (see Study Design Section) on the Modified Friedreich’s Ataxia Rating Scale (mFARS) total score at study Week 72 compared with baseline
次要结局
- SECONDARY: • Difference between participant groups in Clinical Global Impression of Severity (CGI-S) score at study Week 72 compared with baseline
- SECONDARY: • Differences between participant groups in scores on physical function tests (Upright Stability Score (USS) Subscale E of the mFARS, 9-hole peg test, and 25-foot walk test) at study Week 72 compared with baseline
- SECONDARY: • Differences between participant groups in scores on 1) the Friedreich’s Ataxia Rating Scale – Activities of Daily Living and 2) the fatigue numeric rating scale at study Week 72 compared with baseline
- SECONDARY: • Differences between participant groups in left ventricular mass index, measured on echocardiogram, at study Week 72 compared with baseline
- SECONDARY: • Differences between participant groups in changes over time in frataxin amount measured in the skin and in the inside of the cheek
- SAFETY: • Incidence of side effects and serious side effects happening after receiving nomlabofusp, monitored for the entire study duration
- IMMUNOGENICITY: • Incidence of antidrug antibodies (proteins that cause the immune system to fight the drug) throughout the study
研究者
Larimar Therapeutics
Scientific
Larimar Therapeutics Inc.
研究点 (1)
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