2025-524343-13-00招募中2 期
Pilot study of the efficacy of nicotinamide (vitamin B3) in Leber's hereditary optic neuropathy - NICOLHON
干预措施
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 13
- 试验地点
- 2
- 主要终点
- The primary endpoint will be the change in corrected distance visual acuity measured eye by eye using the ETDRS (Early Treatment Diabetic Retinopathy Study) scale over the entire follow-up period (inclusion, 3, 6, 9, and 12 months).
研究概览
简要总结
The primary objective is to evaluate the efficacy of administering 2 grams per day of nicotinamide for one year in patients who have developed NOHL due to the m.11778G>A or m.3460G>A mutation within the last 18 months
入排标准
- 年龄范围
- 0 years 至 65+ years（0-17 Years, 65+ Years, 18-64 Years）
- 接受健康志愿者
- 否
入选标准
- •Patients aged 16 years and older
- •Patients with NOHL due to a m.11778G>A or m.3460G>A mutation in mitochondrial DNA
- •Patients who are naïve (> 3 months) to nicotinamide treatment
- •Patients able to take oral medication and comply with specific study procedures
- •Patients affiliated with or beneficiaries of a social security system
- •Patients who have signed an informed consent form or parental consent form (or guardianship holders) for minors
排除标准
- •Asymptomatic patients (= healthy carriers of a m.11778G>A or m.3460G>A mutation in mitochondrial DNA but who have not developed optic neuropathy)
- •Persons deprived of their liberty by administrative or judicial decision
- •Patients subject to legal protection measures
- •Persons undergoing compulsory psychiatric care
- •Individuals unable to express consent
- •Patients already included in an interventional study modifying the management of NOHL
- •Patients with symptomatic or asymptomatic LOHN caused by another mitochondrial DNA mutation or a nuclear DNA mutation
- •Patients with NOHL for more than 18 months
- •Patients taking idebenone or who have stopped treatment less than 3 months ago
- •Patients with another severe associated ophthalmological condition (advanced glaucoma, retinal disease, etc.)
- •Patients treated with gene therapy
- •Patients with transaminase (AST and/or ALT) levels twice the upper normal limit
- •Pregnant women, breastfeeding women, or women in labor
- •Patients with a contraindication to nicotinamide, an allergy or intolerance to lactose or galactose
研究组 & 干预措施
NICOBION 500 mg, comprimé pelliculé
Test
干预措施: NICOBION 500 mg, comprimé pelliculé (Drug)
结局指标
主要结局
The primary endpoint will be the change in corrected distance visual acuity measured eye by eye using the ETDRS (Early Treatment Diabetic Retinopathy Study) scale over the entire follow-up period (inclusion, 3, 6, 9, and 12 months).
The primary endpoint will be the change in corrected distance visual acuity measured eye by eye using the ETDRS (Early Treatment Diabetic Retinopathy Study) scale over the entire follow-up period (inclusion, 3, 6, 9, and 12 months).
次要结局
- corrected distance visual acuity measured eye by eye on a Monoyer scale
- corrected near visual acuity measured eye by eye on a Parinaud scale
- Optical coherence tomography (OCT) changes in the macula
- corrected distance visual acuity measured eye by eye at 12 months using the ETDRS scale, taking the nadir (lowest visual acuity reached a few weeks after the onset of NOHL) as the reference value
- campimetric deficits based on the average and corrected average deviation measured in STAT 30 on an automated visual field
- the appearance of the Goldman-type manual visual field
- changes in the thickness of the retinal nerve fiber layer (RNFL) and the thickness of the retinal ganglion cell complex (GCC) measured by OCT (Optical Coherence Tomography)
- Patients' quality of life assessed using the NEI VFQ 25 questionnaire
- The biological efficacy of the treatment assessed by the evolution of nicotinamide in patients' blood at 3 and 12 months
- Hepatic toxicity assessed by transaminase levels
研究者
Coordinating investigator
Scientific
Centre Hospitalier Universitaire D'Angers
研究点 (2)
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