Safety and Efficacy of a Flavonoids- and omega3s-based Compound for the Treatment of Muscular Dystrophies

Overview

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.

Full Title of Study: “Studio Monocentrico in Doppio Cieco Randomizzato Dell’Effetto di Una Miscela di Flavonoidi ed Acidi Grassi Naturali in Pazienti Affetti da Distrofia Muscolare”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Randomized
    • Intervention Model: Parallel Assignment
    • Primary Purpose: Treatment
    • Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
  • Study Primary Completion Date: December 31, 2016

Interventions

  • Dietary Supplement: Flavonoids, DHA, EPA
    • oral administration
  • Dietary Supplement: placebo compound
    • oral administration

Arms, Groups and Cohorts

  • Experimental: Treated group
    • oral administration of flavonoids, DHA and EPA, once a day for 24 weeks.
  • Placebo Comparator: Placebo group
    • oral administration of placebo compound, once a day for 24 weeks.

Clinical Trial Outcome Measures

Primary Measures

  • Safety of the administered supplements
    • Time Frame: month 0 and month +6
    • blood tests, to monitor any possible change before and after the study
  • Safety of the administered supplements
    • Time Frame: month 0 and month +6
    • ECG, to monitor any possible change before and after the study
  • Safety of the administered supplements
    • Time Frame: month 0 and month +6
    • neurological clinical assessment, to monitor any possible change before and after the study

Secondary Measures

  • Efficacy outcome – functional changes after the treatment
    • Time Frame: month 0 and month +6
    • Biodex System 4 Dynamometer for quantitative measures (muscle strength and resistance)
  • Efficacy outcome – functional changes after the treatment
    • Time Frame: month 0 and month +6
    • 6 Minute Walking Test for quantitative measures (muscle strength and resistance)
  • Efficacy outcome – functional changes after the treatment
    • Time Frame: month 0, month +2, month +4, month +6
    • EK (Egen Klassifikation) scale to assess funtionality of patients with significative impairments. Min. score: 0 – max. score 30
  • Efficacy outcome – functional changes after the treatment
    • Time Frame: month 0, month +2, month +4, month +6
    • ACTIVLIM (Activity Limitation) scale. The scale measures activity limitations for patients with upper and/or lower limb impairments and has been validated in patients affected by neuromuscular disorders. Min. score: 0 – max. score 36
  • Efficacy outcome – functional changes after the treatment
    • Time Frame: month 0, month +2, month +4, month +6
    • ABILHAND (manual ability for adults with upper limb impairments) scale. The scale measures manual ability for adults with upper limb impairments.Min. score: 0 – max. score 36

Participating in This Clinical Trial

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)

Gender Eligibility: All

males, for patients affected by Duchenne muscular dystrophy

Minimum Age: 9 Years

Maximum Age: 70 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Fondazione IRCCS Ca’ Granda, Ospedale Maggiore Policlinico
  • Provider of Information About this Clinical Study
    • Sponsor

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