Clinical trial NCT03317171: Safety and Efficacy of a Flavonoids- and omega3s-based Compound for the Treatment of Muscular Dystrophies
Official title: 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: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Muscular Dystrophies
Muscle Inflammation
Keywords: dietary supplementation
flavonoids
omega 3
Lead sponsor: Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Sponsor class: OTHER
First posted: 2017-10-23
Enrollment: 29
Sex: ALL
Minimum age (years): 9
Maximum age (years): 70
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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)

Primary outcomes:
measure: Safety of the administered supplements
timeFrame: month 0 and month +6
measure: Safety of the administered supplements
timeFrame: month 0 and month +6
measure: Safety of the administered supplements
timeFrame: month 0 and month +6
Secondary outcomes:
measure: Efficacy outcome - functional changes after the treatment
timeFrame: month 0 and month +6
measure: Efficacy outcome - functional changes after the treatment
timeFrame: month 0 and month +6
measure: Efficacy outcome - functional changes after the treatment
timeFrame: month 0, month +2, month +4, month +6
measure: Efficacy outcome - functional changes after the treatment
timeFrame: month 0, month +2, month +4, month +6
measure: Efficacy outcome - functional changes after the treatment
timeFrame: month 0, month +2, month +4, month +6
Other outcomes:
Locations:
city: MIlan
country: Italy
facility: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Study officials:
References:
citation: Sitzia C, Meregalli M, Belicchi M, Farini A, Arosio M, Bestetti D, Villa C, Valenti L, Brambilla P, Torrente Y. Preliminary Evidences of Safety and Efficacy of Flavonoids- and Omega 3-Based Compound for Muscular Dystrophies Treatment: A Randomized Double-Blind Placebo Controlled Pilot Clinical Trial. Front Neurol. 2019 Jul 23;10:755. doi: 10.3389/fneur.2019.00755. eCollection 2019.