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

Study Overview
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Brief Summary
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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.

Official Title
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Studio Monocentrico in Doppio Cieco Randomizzato Dell'Effetto di Una Miscela di Flavonoidi ed Acidi Grassi Naturali in Pazienti Affetti da Distrofia Muscolare

Conditions
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Muscular Dystrophies, Muscle Inflammation

Intervention / Treatment
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* Dietary Supplement: Flavonoids, DHA, EPA
* Dietary Supplement: placebo compound


Participation Criteria
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Eligibility Criteria
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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)

Ages Eligible for Study
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Minimum Age: 9 Years
Maximum Age: 70 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Treated group<br>oral administration of flavonoids, DHA and EPA, once a day for 24 weeks. | Dietary Supplement: Flavonoids, DHA, EPA<br>* oral administration<br>|
| Placebo Comparator: Placebo group<br>oral administration of placebo compound, once a day for 24 weeks. | Dietary Supplement: placebo compound<br>* oral administration<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety of the administered supplements | blood tests, to monitor any possible change before and after the study | month 0 and month +6 | 
| Safety of the administered supplements | ECG, to monitor any possible change before and after the study | month 0 and month +6 | 
| Safety of the administered supplements | neurological clinical assessment, to monitor any possible change before and after the study | month 0 and month +6 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Efficacy outcome - functional changes after the treatment | Biodex System 4 Dynamometer for quantitative measures (muscle strength and resistance) | month 0 and month +6 | 
| Efficacy outcome - functional changes after the treatment | 6 Minute Walking Test for quantitative measures (muscle strength and resistance) | month 0 and month +6 | 
| Efficacy outcome - functional changes after the treatment | EK (Egen Klassifikation) scale to assess funtionality of patients with significative impairments. Min. score: 0 - max. score 30 | month 0, month +2, month +4, month +6 | 
| Efficacy outcome - functional changes after the treatment | 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 | month 0, month +2, month +4, month +6 | 
| Efficacy outcome - functional changes after the treatment | 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 | month 0, month +2, month +4, month +6 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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dietary supplementation, flavonoids, omega 3