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OpenTrials
Completed

NCT Number: NCT03317171

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

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.

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Key information

Conditions

Muscular Dystrophies Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Muscle Inflammation Muscular Diseases Muscular Disorders, Atrophic Musculoskeletal Diseases Myositis Myositis, Inclusion Body Nervous System Diseases Neuromuscular Diseases

Age range

9 year–70 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, 20122, Italy

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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)

Treatment and study plan

Flavonoids, DHA, EPA

Dietary Supplement

oral administration

placebo compound

Dietary Supplement

oral administration

Primary outcomes

  1. Safety of the administered supplements

    Time frame: month 0 and month +6

    blood tests, to monitor any possible change before and after the study

  2. Safety of the administered supplements

    Time frame: month 0 and month +6

    ECG, to monitor any possible change before and after the study

  3. Safety of the administered supplements

    Time frame: month 0 and month +6

    neurological clinical assessment, to monitor any possible change before and after the study

Secondary outcomes

  1. Efficacy outcome - functional changes after the treatment

    Time frame: month 0 and month +6

    Biodex System 4 Dynamometer for quantitative measures (muscle strength and resistance)

  2. Efficacy outcome - functional changes after the treatment

    Time frame: month 0 and month +6

    6 Minute Walking Test for quantitative measures (muscle strength and resistance)

  3. Efficacy outcome - functional changes after the treatment

    Time frame: month 0, month +2, month +4, month +6

    EK (Egen Klassifikation) scale to assess funtionality of patients with significative impairments. Min. score: 0 - max. score 30

  4. Efficacy outcome - functional changes after the treatment

    Time frame: month 0, month +2, month +4, month +6

    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

  5. Efficacy outcome - functional changes after the treatment

    Time frame: month 0, month +2, month +4, month +6

    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

Sponsors and collaborators

Lead sponsor

Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

Other

Registry information

Official study title

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

Acronym: PRO1

Important dates

Study start
2016
Primary completion
2016
Study completion
2016
First posted
Oct 23, 2017
Registry last updated
Oct 25, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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