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

NCT03317171 | Completed

• 1 other identifier: 1768
• Study Type: interventional
• Target: 29
• Locations: 1 country
• Sites: 1

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.

Conditions

Muscular Dystrophies; Muscle Inflammation

Keywords

dietary supplementation; flavonoids; omega 3

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (5)

• Efficacy outcome - functional changes after the treatment

  month 0 and month +6

• Efficacy outcome - functional changes after the treatment

  month 0 and month +6

• Efficacy outcome - functional changes after the treatment

  month 0, month +2, month +4, month +6

• Efficacy outcome - functional changes after the treatment

  month 0, month +2, month +4, month +6

• Efficacy outcome - functional changes after the treatment

  month 0, month +2, month +4, month +6

Study Arms (2)

Treated group

EXPERIMENTAL

oral administration of flavonoids, DHA and EPA, once a day for 24 weeks.

Dietary Supplement: Flavonoids, DHA, EPA

Placebo group

PLACEBO COMPARATOR

oral administration of placebo compound, once a day for 24 weeks.

Dietary Supplement: placebo compound

Interventions

Flavonoids, DHA, EPA DIETARY_SUPPLEMENT

oral administration

Treated group

placebo compound DIETARY_SUPPLEMENT

oral administration

Placebo group

Eligibility Criteria

• Age: 9 Years - 70 Years
• Gender Eligibility Details: males, for patients affected by Duchenne muscular dystrophy
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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)

Sponsors & Collaborators

• Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico: lead

Study Sites (1)

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, 20122, Italy

Location

Related Publications (1)

• 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.

  PMID: 31396142 DERIVED

MeSH Terms

Conditions

Muscular Dystrophies; Myositis, Inclusion Body

Interventions

Flavonoids

Condition Hierarchy (Ancestors)

Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Myositis

Intervention Hierarchy (Ancestors)

Chromones; Benzopyrans; Pyrans; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 11, 2017
• First Posted: October 23, 2017
• Study Start: February 28, 2016
• Primary Completion: December 31, 2016
• Study Completion: December 31, 2016
• Last Updated: October 25, 2017

Record last verified: 2017-10

Locations

IT (1)