2023-506214-52-00招募中3 期
Pilot study of safety and efficacy of nicotinamide (vitamin B3) in OPA1 Dominant Optic Atrophy - NICOPA1-TOL
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 25
- 试验地点
- 1
- 主要终点
- The primary endpoint will be assessed by the number of patients with reported ophthalmological and/or neurological adverse events.
研究概览
简要总结
The main objective is to evaluate the tolerability of 3 grams per day of nicotinamide in DOA/DOA+ patients to ensure that this treatment does not induce ophthalmological or neurological toxic side effects.
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
- 否
入选标准
- •Major patients
- •Patients with DOA or DOA+ due to a heterozygous pathogenic variant in the OPA1 gene
- •Nicotinamide-naïve patients (> 3 months)
- •Patients able to take oral medication, and comply with specific study procedures
- •Patients affiliated with or benefiting from a social security scheme
- •Signature of voluntary, free and informed consent to participate in the study
排除标准
- •Asymptomatic patients (= healthy carriers of an OPA1 mutation who have not developed optic neuropathy)
- •Persons unable to give consent
- •Patients with other associated severe ophthalmological pathologies (advanced glaucoma, retinal pathology).
- •Patients treated with Idebenone
- •Patients with transaminase levels (ASAT and/or ALAT) twice the high normal value
- •Pregnant, breast-feeding or parturient women
- •Patients with a contraindication to nicotinamide
- •Person deprived of liberty by administrative or judicial decision
- •Patients under legal protection
- •Persons under compulsory psychiatric care
结局指标
主要结局
The primary endpoint will be assessed by the number of patients with reported ophthalmological and/or neurological adverse events.
The primary endpoint will be assessed by the number of patients with reported ophthalmological and/or neurological adverse events.
次要结局
- Number of patients with improved visual function at follow-up visits compared with baseline at inclusion.
- Increase in plasma and whole blood nicotinamide levels in all patients, comparing baseline at inclusion with two follow-up periods.
- Differences on the NEI-VFQ-25 scale between M0 and M6
- Clinical neurological improvement at follow-up visits compared with baseline at inclusion.
研究者
coordinating investigator
Scientific
Centre Hospitalier Universitaire D Angers
研究点 (1)
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