search
Back to results

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

Primary Purpose

Muscular Dystrophies, Muscle Inflammation

Status
Completed
Phase
Not Applicable
Locations
Italy
Study Type
Interventional
Intervention
Flavonoids, DHA, EPA
placebo compound
Sponsored by
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Muscular Dystrophies focused on measuring dietary supplementation, flavonoids, omega 3

Eligibility Criteria

9 Years - 70 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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)

Sites / Locations

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Treated group

Placebo group

Arm Description

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

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

Outcomes

Primary Outcome Measures

Safety of the administered supplements
blood tests, to monitor any possible change before and after the study
Safety of the administered supplements
ECG, to monitor any possible change before and after the study
Safety of the administered supplements
neurological clinical assessment, to monitor any possible change before and after the study

Secondary Outcome Measures

Efficacy outcome - functional changes after the treatment
Biodex System 4 Dynamometer for quantitative measures (muscle strength and resistance)
Efficacy outcome - functional changes after the treatment
6 Minute Walking Test for quantitative measures (muscle strength and resistance)
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
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
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

Full Information

First Posted
October 11, 2017
Last Updated
October 23, 2017
Sponsor
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
search

1. Study Identification

Unique Protocol Identification Number
NCT03317171
Brief Title
Safety and Efficacy of a Flavonoids- and omega3s-based Compound for the Treatment of Muscular Dystrophies
Acronym
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
Study Type
Interventional

2. Study Status

Record Verification Date
October 2017
Overall Recruitment Status
Completed
Study Start Date
February 28, 2016 (Actual)
Primary Completion Date
December 31, 2016 (Actual)
Study Completion Date
December 31, 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

4. Oversight

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
Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Muscular Dystrophies, Muscle Inflammation
Keywords
dietary supplementation, flavonoids, omega 3

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
29 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Treated group
Arm Type
Experimental
Arm Description
oral administration of flavonoids, DHA and EPA, once a day for 24 weeks.
Arm Title
Placebo group
Arm Type
Placebo Comparator
Arm Description
oral administration of placebo compound, once a day for 24 weeks.
Intervention Type
Dietary Supplement
Intervention Name(s)
Flavonoids, DHA, EPA
Intervention Description
oral administration
Intervention Type
Dietary Supplement
Intervention Name(s)
placebo compound
Intervention Description
oral administration
Primary Outcome Measure Information:
Title
Safety of the administered supplements
Description
blood tests, to monitor any possible change before and after the study
Time Frame
month 0 and month +6
Title
Safety of the administered supplements
Description
ECG, to monitor any possible change before and after the study
Time Frame
month 0 and month +6
Title
Safety of the administered supplements
Description
neurological clinical assessment, to monitor any possible change before and after the study
Time Frame
month 0 and month +6
Secondary Outcome Measure Information:
Title
Efficacy outcome - functional changes after the treatment
Description
Biodex System 4 Dynamometer for quantitative measures (muscle strength and resistance)
Time Frame
month 0 and month +6
Title
Efficacy outcome - functional changes after the treatment
Description
6 Minute Walking Test for quantitative measures (muscle strength and resistance)
Time Frame
month 0 and month +6
Title
Efficacy outcome - functional changes after the treatment
Description
EK (Egen Klassifikation) scale to assess funtionality of patients with significative impairments. Min. score: 0 - max. score 30
Time Frame
month 0, month +2, month +4, month +6
Title
Efficacy outcome - functional changes after the treatment
Description
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
Time Frame
month 0, month +2, month +4, month +6
Title
Efficacy outcome - functional changes after the treatment
Description
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
Time Frame
month 0, month +2, month +4, month +6

10. Eligibility

Sex
All
Gender Based
Yes
Gender Eligibility Description
males, for patients affected by Duchenne muscular dystrophy
Minimum Age & Unit of Time
9 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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)
Facility Information:
Facility Name
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
City
MIlan
ZIP/Postal Code
20122
Country
Italy

12. IPD Sharing Statement

Citations:
PubMed Identifier
31396142
Citation
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.
Results Reference
derived

Learn more about this trial

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

We'll reach out to this number within 24 hrs