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

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

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.

Study Overview

• Status: Completed
• Conditions: Muscular Dystrophies; Muscle Inflammation
• Intervention / Treatment: Dietary supplement: Flavonoids, DHA, EPA; Dietary supplement: placebo compound

• Study Type: Interventional
• Enrollment (Actual): 29
• Phase: Not Applicable

Contacts and Locations

Study Locations

• Italy
  • MIlan, Italy, 20122
    • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 9 years to 70 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Treated group; oral administration of flavonoids, DHA and EPA, once a day for 24 weeks.	Dietary supplement: Flavonoids, DHA, EPA; oral administration
Placebo Comparator: Placebo group; oral administration of placebo compound, once a day for 24 weeks.	Dietary supplement: placebo compound; oral administration

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Efficacy outcome - functional changes after the treatment	Biodex System 4 Dynamometer for quantitative measures (muscle strength and resistance)	month 0 and month +6
Efficacy outcome - functional changes after the treatment	6 Minute Walking Test for quantitative measures (muscle strength and resistance)	month 0 and month +6
Efficacy outcome - functional changes after the treatment	EK (Egen Klassifikation) scale to assess funtionality of patients with significative impairments. Min. score: 0 - max. score 30	month 0, month +2, month +4, month +6
Efficacy outcome - functional changes after the treatment	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	month 0, month +2, month +4, month +6
Efficacy outcome - functional changes after the treatment	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	month 0, month +2, month +4, month +6

Collaborators and Investigators

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

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start (Actual): February 28, 2016
• Primary Completion (Actual): December 31, 2016
• Study Completion (Actual): December 31, 2016

Study Registration Dates

• First Submitted: October 11, 2017
• First Submitted That Met QC Criteria: October 17, 2017
• First Posted (Actual): October 23, 2017

Study Record Updates

• Last Update Posted (Actual): October 25, 2017
• Last Update Submitted That Met QC Criteria: October 23, 2017
• Last Verified: October 1, 2017

More Information

Terms related to this study

• Keywords: dietary supplementation; flavonoids; omega 3
• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Genetic Diseases, Inborn; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Muscular Disorders, Atrophic; Inflammation; Muscular Dystrophies; Myositis
• Other Study ID Numbers: 1768

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No