Muscular DystrophiesPRO1NCT03317171
Safety and Efficacy of a Flavonoids- and omega3s-based Compound for the Treatment of Muscular Dystrophies
A study testing Flavonoids, DHA, EPA in people with Muscular Dystrophies and Muscle Inflammation, run by Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico at 1 site in Italy.
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… Read more about this study
- Who is it for?
- Ages 9 to 70 years
- Conditions the study is for
- Muscular Dystrophies, Muscle Inflammation
- What is being tested?
- Flavonoids, DHA, EPA
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 29 participants
- Where
- 1 siteItaly
- Study dates
- 28 Feb 2016to 31 Dec 2016
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 9 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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;
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- 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)
Trial locations and contacts
1 site in 1 country.
Italy1 site
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, 20122
About this study
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
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
- Being tested
- Flavonoids, DHA, EPA, placebo compound
- Keywords
- dietary supplementation, flavonoids, omega 3
- Medical subject headings
- Muscular Dystrophies, Myositis, Inclusion Body
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Flavonoids, DHA, EPADietary supplement
oral administration
placebo compoundDietary supplement
oral administration
| Study group | Role | What they receive |
|---|---|---|
| Treated group | Experimental | oral administration of flavonoids, DHA and EPA, once a day for 24 weeks. Receives: Dietary Supplement: Flavonoids, DHA, EPA |
| Placebo group | Placebo comparator | oral administration of placebo compound, once a day for 24 weeks. Receives: Dietary Supplement: placebo compound |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of the administered supplements
blood tests, to monitor any possible change before and after the study
Measured: month 0 and month +6
Safety of the administered supplements
ECG, to monitor any possible change before and after the study
Measured: month 0 and month +6
Safety of the administered supplements
neurological clinical assessment, to monitor any possible change before and after the study
Measured: month 0 and month +6
Secondary outcomes (5)
Efficacy outcome - functional changes after the treatment
Biodex System 4 Dynamometer for quantitative measures (muscle strength and resistance)
Measured: month 0 and month +6
Efficacy outcome - functional changes after the treatment
6 Minute Walking Test for quantitative measures (muscle strength and resistance)
Measured: 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
Measured: 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
Measured: 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
Measured: month 0, month +2, month +4, month +6
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Oct 2017
- Study start28 Feb 2016
- Primary completion31 Dec 2016
- Study completion31 Dec 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Muscular DystrophiesStudies of the same condition, open and under way.
- More trials run by Fondazione IRCCS Ca' Granda, Ospedale Maggiore PoliclinicoThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 29 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03317171
- Sponsor's protocol ID
- 1768
- Acronym
- PRO1
- Status last verified
- Oct 2017
- First submitted
- 11 Oct 2017
- Last updated
- 25 Oct 2017
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
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.
PubMed 31396142
Study at a glance
From the registry record, last updated by the sponsor on 25 Oct 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03317171
- Acronym / sponsor ID
- PRO1 / 1768
- Conditions
- Muscular Dystrophies, Muscle Inflammation
- Keywords
- dietary supplementation, flavonoids, omega 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 29 participants (actual)
- Lead sponsor
- Fondazione IRCCS Ca' Granda, Ospedale Maggiore PoliclinicoUniversity, hospital or other
- Study start
- 28 Feb 2016 (actual)
- Primary completion
- 31 Dec 2016 (actual)
- Study completion
- 31 Dec 2016 (actual)
- First posted
- 23 Oct 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Treated group | Experimental | Dietary Supplement: Flavonoids, DHA, EPAoral administration of flavonoids, DHA and EPA, once a day for 24 weeks. |
| Placebo group | Placebo comparator | Dietary Supplement: placebo compoundoral administration of placebo compound, once a day for 24 weeks. |
Who may qualify
- Ages
- 9 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (9 listed)
- 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);
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Flavonoids, DHA, EPAoral administration | Dietary supplement | Treated group |
| placebo compoundoral administration | Dietary supplement | Placebo group |
Primary outcome
Safety of the administered supplements
Time frame: month 0 and month +6
blood tests, to monitor any possible change before and after the study
Safety of the administered supplements
Time frame: month 0 and month +6
ECG, to monitor any possible change before and after the study
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
5 secondary outcomes
Efficacy outcome - functional changes after the treatment
Time frame: month 0 and month +6
Efficacy outcome - functional changes after the treatment
Time frame: month 0 and month +6
Efficacy outcome - functional changes after the treatment
Time frame: month 0, month +2, month +4, month +6
Efficacy outcome - functional changes after the treatment
Time frame: month 0, month +2, month +4, month +6
Efficacy outcome - functional changes after the treatment
Time frame: month 0, month +2, month +4, month +6
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Oct 2017.
- Derived from this study: PubMed 31396142Sitzia 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.
Related trials for Muscular Dystrophies
Active, not recruiting
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis
RecruitingEarly Phase 1
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
Duchenne Muscular Dystrophy (DMD)
RecruitingPhase 2
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
RecruitingObservational study
Preclinical Assessment of an Engineered HMGB1 Protein as a Novel Companion Therapy for Muscular Dystrophies
LAMA2-MD (Merosin Deficient Congenital Muscular Dystrophy, MDC1A)
RecruitingObservational study
Natural History Study of Children With LAMA2-related Dystrophies
Merosin Deficient Congenital Muscular Dystrophy
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Flavonoids, DHA, EPA in people with Muscular Dystrophies and Muscle Inflammation, run by Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico at 1 site in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 25 Oct 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety of the administered supplements (time frame: month 0 and month +6). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03317171, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Oct 2017.