NCT05590468进行中（未招募）2 期
Randomized, Double-blinded, Placebo-controlled Study Evaluating the Efficacy of Nicotinamide Riboside (NR) - a Vitamin B3 Derivative - for Treatment of Mitochondrial Myopathy Disorder
Ralitza Gavrilova1 个研究点 分布在 1 个国家目标入组 34 人开始时间: 2023年5月26日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 34
- 试验地点
- 1
- 主要终点
- Changes in 6-Minute Walk Test Performance
研究概览
简要总结
The purpose of this study is to determine the effects of Nicotinamide Riboside (NR) supplement in adult-onset symptoms of mitochondrial myopathy.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Biochemically and/or genetically confirmed or confirmed primary mitochondrial myopathy based on published diagnostic criteria.
- •Biochemically confirmed mitochondrial disorder would mean that the patient meets clinical criteria and has either biopsy or biochemical testing that supports the diagnosis.
- •Confirmed mitochondrial disorder means that the patient meets published clinical criteria for the diagnosis and has also had confirmatory genetic testing for the disorder type.
- •Agreed to avoid vitamin supplementation or nutritional products with vitamin B3 forms 14 days prior to the enrollment and during the study in order to not exceed 200 mg/day of vitamin B3 derivatives intake.
- •Female of childbearing potential agreed to use effective contraception throughout the study.
- •Written, informed consent to participate in the study.
排除标准
- •Unwilling to comply with the follow-up schedule.
- •Inability or refusal to give informed consent by the patient or a legally authorized representative.
- •Known pregnancy or breastfeeding.
- •Concurrent participation in another investigational drug study or within washout period of treatment.
- •Presence of other medical symptoms or condition, which may interfere with interpretation of outcome measures as determined by the study PI.
- •Clinical / Laboratory Exclusion Criteria:
- •Estimated Glomerular Filtration Rate (eGFR) < 30 mL/min.
- •Patients in permanent Renal Replacement Therapy.
- •Serum alkaline phosphatase 50% above normal limit.
- •Serum aspartate transaminase 50% above normal limit.
- •Serum Thyroxine (T4) 50% above or below normal limit.
- •Serum Thyroid Stimulating Hormone (TSH) 50% above or below normal limit.
- •Severe anemia with Hb < 7g/dL.
- •Severe leukocytosis with WBC > 15,000/mm^3.
研究组 & 干预措施
Nicotinamide Riboside treatment group
Experimental
Subjects will receive vitamin B3 derivative Nicotinamide Riboside (NR) daily for 12 months
干预措施: Nicotinamide Riboside (Dietary Supplement)
Placebo Group
Placebo Comparator
Subjects will receive a placebo daily for 12 months
干预措施: Placebo (Drug)
结局指标
主要结局
Changes in 6-Minute Walk Test Performance
时间窗: Baseline, 6 months, 12 months
The 6-minute walk test (6MWT) quantifies the distance a person can walk in six minutes. Measured in meters.
次要结局
- Change in isometric strength of trunk flexors(Baseline, 6 months, 12 months)
- Change in isometric strength of trunk extensor muscle(Baseline, 6 months, 12 months)
- Change in skeletal muscle function(Baseline, 6 months, 12 months)
- Change in skeletal muscle strength(Baseline, 6 months, 12 months)
- Change in maximal oxygen uptake (VO2 max)(Baseline, 6 months, 12 months)
研究者
Ralitza Gavrilova
Regulatory Sponsor
Mayo Clinic
研究点 (1)
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