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

Muscular Dystrophies Clinical Trial

— PRO1  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03317171
• Other study ID #: 1768
• Status: Completed
• Phase: N/A
• First received: October 11, 2017
• Last updated: October 23, 2017
• Start date: February 28, 2016
• Est. completion date: December 31, 2016

Study information

• Verified date: October 2017
• Source: Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 29
• Est. completion date: December 31, 2016
• Est. primary completion date: December 31, 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: 9 Years to 70 Years

Eligibility

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)

Related Conditions & MeSH terms

• Inflammation
• Muscle Inflammation
• Muscular Dystrophies
• Myositis

Intervention

Dietary Supplement:
• Flavonoids, DHA, EPA
oral administration
• placebo compound
oral administration

Locations

Country	Name	City	State
Italy	Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico	MIlan

Sponsors (1)

Lead Sponsor	Collaborator
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

Country where clinical trial is conducted

Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety of the administered supplements	blood tests, to monitor any possible change before and after the study	month 0 and month +6
Primary	Safety of the administered supplements	ECG, to monitor any possible change before and after the study	month 0 and month +6
Primary	Safety of the administered supplements	neurological clinical assessment, to monitor any possible change before and after the study	month 0 and month +6
Secondary	Efficacy outcome - functional changes after the treatment	Biodex System 4 Dynamometer for quantitative measures (muscle strength and resistance)	month 0 and month +6
Secondary	Efficacy outcome - functional changes after the treatment	6 Minute Walking Test for quantitative measures (muscle strength and resistance)	month 0 and month +6
Secondary	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
Secondary	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
Secondary	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