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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
- Source :
- Frontiers in Neurology, Vol 10 (2019), Frontiers in Neurology
- Publication Year :
- 2019
- Publisher :
- Frontiers Media S.A., 2019.
-
Abstract
- Background: Nutritional compounds can exert both anti-inflammatory and anti-oxidant effects. Since these events exacerbate the pathophysiology of muscular dystrophies, we investigated nutraceutical supplementation as an adjuvant therapy in dystrophic patients, to low costs and easy route of administration. Moreover, this treatment could represent an alternative therapeutic strategy for dystrophic patients who do not respond to corticosteroid treatment. Objective: A 24 weeks randomized double-blind placebo-controlled clinical study was aimed at evaluating the safety and efficacy of daily oral administration of flavonoids- and omega3-based natural supplement (FLAVOMEGA) in patients affected by muscular dystrophy with recognized muscle inflammation. Design: We screened 60 patients diagnosed for Duchenne (DMD), Facioscapulohumeral (FSHD), and Limb Girdle Muscular Dystrophy (LGMD). Using a computer-generated random allocation sequence, we stratified patients in a 2:1:1 ratio (DMD:FSHD:LGMD) to one of two treatment groups: continuous FLAVOMEGA, continuous placebo. Of 29 patients included, only 24 completed the study: 15 were given FLAVOMEGA, 14 placebo. Results: FLAVOMEGA was well tolerated with no reported adverse events. Significant treatment differences in the change from baseline in 6 min walk distance (6MWD; secondary efficacy endpoint) (P = 0.033) and in isokinetic knee extension (P = 0.039) (primary efficacy endpoint) were observed in LGMD and FSHD subjects. Serum CK levels (secondary efficacy endpoint) decreased in all FLAVOMEGA treated groups with significant difference in DMD subjects (P = 0.039). Conclusions: Although the small number of patients and the wide range of disease severity among patients reduced statistical significance, we obtained an optimal profile of safety and tolerability for the compound, showing valuable data of efficacy in primary and secondary endpoints. Trial registration number: NCT03317171 Retrospectively registered 25/10/2017.
- Subjects :
- 0301 basic medicine
musculoskeletal diseases
Duchenne muscular dystrophy
safety
medicine.medical_specialty
BIO/12 - BIOCHIMICA CLINICA E BIOLOGIA MOLECOLARE CLINICA
Placebo
lcsh:RC346-429
03 medical and health sciences
0302 clinical medicine
Internal medicine
medicine
Adjuvant therapy
Muscular dystrophy
tolerability
Adverse effect
strength recovery
lcsh:Neurology. Diseases of the nervous system
business.industry
medicine.disease
Clinical Trial
Clinical trial
030104 developmental biology
Tolerability
Neurology
Neurology (clinical)
nutraceutical supplementation
business
030217 neurology & neurosurgery
Limb-girdle muscular dystrophy
Subjects
Details
- Language :
- English
- ISSN :
- 16642295
- Volume :
- 10
- Database :
- OpenAIRE
- Journal :
- Frontiers in Neurology
- Accession number :
- edsair.doi.dedup.....2849423cccd15b0349c50b60d006924d