Studio Monocentrico in Doppio Cieco Randomizzato Dell'Effetto di Una Miscela di Flavonoidi ed Acidi Grassi Naturali in Pazienti Affetti da Distrofia Muscolare
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 29
- 试验地点
- 1
- 主要终点
- Safety of the administered supplements
研究概览
简要总结
The study aimed to assess the safety and, partially, the efficacy of dietary supplementation of a flavonoids-, DHA- and EPA-based natural supplement in non-ambulant DMD boys and in a cohort of LGMD and FSHD patients to compare its effect in MDs of different aetiology and to eventually highlight any differences in inflammatory involved pathways. To assess safety, patient's laboratory parameters were monitored and adverse events recorded, while efficacy was evaluated through performance scale questionnaire and strength measurement (6 minute walking test and Biodex System 4 Dynamometer parameter evaluation). This study was conceived as proof of principle for the safe use of flavonoids/omega3s-based compound as an adjuvant in the management of neuromuscular disorders; besides, its efficacy in alleviating symptoms linked to secondary effects of genetic mutation as inflammation, muscular pain and weakness was assessed.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 9 Years 至 70 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •documented genetic and histological diagnosis of DMD, FSHD and LGMD;
- •absence of severe cardiac and pulmonary disease;
- •age superior to 9 years for DMD, between 9 and 70 years for LGMD and between 20 and 70 years for FSHD;
- •glucocorticosteroid treatment for 6 or more months (stable dose and regimen for ≥3 months before screening) and for the duration of the study;
- •adhesion to inform consent by same patients or parents/tutors for minors.
排除标准
- •severe cardiac and pulmonary disease;
- •positive hepatitis B surface antigen (HBsAg) test, positive hepatitis C and HIV tests;
- •low kidney and liver functionality;
- •autoimmune disorders;
- •mental retardation (IQ via Wechsler Intelligence Scale < 70);
- •psychological-psychiatric disorders; adverse psychosocial conditions;
- •known allergies to some of compounds to be used in the trial;
- •pathologies occurring just before or during the trial (fever, metabolic disorders, drug abuse);
- •enrollment to other trials (steroids regime won't be considered in this list)
研究组 & 干预措施
Treated group
oral administration of flavonoids, DHA and EPA, once a day for 24 weeks.
干预措施: Flavonoids, DHA, EPA (Dietary Supplement)
Placebo group
oral administration of placebo compound, once a day for 24 weeks.
干预措施: placebo compound (Dietary Supplement)
结局指标
主要结局
Safety of the administered supplements
时间窗: month 0 and month +6
blood tests, to monitor any possible change before and after the study
次要结局
- Efficacy outcome - functional changes after the treatment(month 0, month +2, month +4, month +6)
- Efficacy outcome - functional changes after the treatment(month 0 and month +6)