Safety and Efficacy of a Flavonoids- and omega3s-based Compound for the Treatment of Muscular Dystrophies (PRO1)

Institute of Hospitalization and Scientific Care (IRCCS)

Status

Completed

Conditions

Muscle Inflammation

Muscular Dystrophies

Treatments

Dietary Supplement: Flavonoids, DHA, EPA

Dietary Supplement: placebo compound

Study type

Interventional

Funder types

Other

Identifiers

NCT03317171

1768

Details and patient eligibility

About

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.

Enrollment

29 patients

Sex

All

Ages

9 to 70 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion 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)