Studio Monocentrico in Doppio Cieco Randomizzato Dell'Effetto di Una Miscela di Flavonoidi ed Acidi Grassi Naturali in Pazienti Affetti da Distrofia Muscolare
Trial Snapshot
- Phase
- Not Applicable
- Status
- Completed
- Enrollment
- 29
- Locations
- 1
- Primary Endpoint
- Safety of the administered supplements
Study Overview
Brief Summary
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.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Eligibility Criteria
- Ages
- 9 Years to 70 Years (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •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.
Exclusion Criteria
- •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)
Arms & Interventions
Treated group
oral administration of flavonoids, DHA and EPA, once a day for 24 weeks.
Intervention: Flavonoids, DHA, EPA (Dietary Supplement)
Placebo group
oral administration of placebo compound, once a day for 24 weeks.
Intervention: placebo compound (Dietary Supplement)
Outcomes
Primary Outcomes
Safety of the administered supplements
Time Frame: month 0 and month +6
blood tests, to monitor any possible change before and after the study
Secondary Outcomes
- 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)