Trial to evaluate the efficacy of the drug metformin on the muscles in patients with myotonic distrophy type 1

EFFICACY OF METFORMIN ON MOTILITY AND STRENGTH IN MYOTONIC DYSTROPHY TYPE 1. A randomized, double blind, placebo-controlled, multicenter clinical trial. - MetMyd

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2018-000692-32-IT

Enrollment

194

Registered

2020-01-14

Start date

2019-07-17

Completion date

Unknown

Last updated

2025-01-27

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Myotonic Distrophy type 1, confirmed by genetic testing, with a CTG expansion size >100 MedDRA version: 20.0 Level: PT Classification code 10068871 Term: Myotonic dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0 Level: LLT Classification code 10013987 Term: Dystrophia myotonica System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Trade Name: METFORMINA - 500 30 COMPRESSE 500 MG Product Name: Metformina Pharmaceutical Form: Film-coated tablet INN or Proposed INN: Metformina Current Sponsor code: N/A Concentration unit: mg milli

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Inclusion criteria are as stated below: Principal inclusion criteria - Male or female patients, aged between 18 and 64 years - A diagnosis of DM1, confirmed by genetic testing, with a CTG expansion size >100 - MIRS score 3 or 4 - Ambulatory, able to perform the 6 Minute Walk Test (6MWT) - Able to provide written informed consent - For women of child-bearing potential, blood screening for beta-HCG before randomization and use of one effective method of birth control during the conduct of the study Are the trial subjects under 18? no Number of subjects for this age range: 1 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 194 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: Exclusion criteria are as stated below: Principal exclusion criteria - Any medical contraindications to metformin including: known hypersensitivity to Metformin Hydrochloride or any of the other ingredients; Renal disease or renal dysfunction (serum creatinine levels =1.5 mg/dL [males], =1.4 mg/dL [females] or abnormal creatinine clearance, 12 months - Women who are pregnant or breast-feeding - Chronic administration of any drugs that may interfere with the actions of Metformin. - Participation in another experimental therapeutic protocol within 6 months prior to baseline and during the study period (participation in natural history study is allowed) - Any other condition that, in the opinion of the investigator, may compromise the safety or compliance of the patient or would preclude the patient from successful completion of the study or would impair interpretation of results - Any neurological disease with motor impairment or other neuromuscular disease than DM1

Design outcomes

Primary

Measure	Time frame
Main Objective: To compare metformin hydrochloride against a placebo in the treatment of DM1. The intervention is focused on the effect of the experimental intervention on motility performances. This parameter is marker of the most involved motor capabilities in DM1 patients and will be measured with sensitive and validated scales. It is expected that the trial and outcome work will lead to new clinical guidelines for DM1 treatment.;Secondary Objective: Secondary objectives of the study are categorized as: 1) Dexterity and motility; 2) Muscle quantitative testing of upper and lower limbs; 3) Fatigue; 4) Quality of life. ;Primary end point(s): The primary outcome measure will be the 6 minute walk test (6MWT) with BORG Scale assessment (0 to 10 rating of perceived exertion score) measured at the end of the 24 months intervention period. The 6MWT is a widely used walking test: the distance walked in 6 minutes is measured and a greater distance indicates a better performance. The 6MWT has been validated for DM1 patients and is a sensitive measure that shows deterioration of motility in a relative short term within the natural history of the disease;Timepoint(s) of evaluation of this end point: After 24 months of treatment	—

Measure

Time frame

Main Objective: To compare metformin hydrochloride against a placebo in the treatment of DM1. The intervention is focused on the effect of the experimental intervention on motility performances. This parameter is marker of the most involved motor capabilities in DM1 patients and will be measured with sensitive and validated scales. It is expected that the trial and outcome work will lead to new clinical guidelines for DM1 treatment.;Secondary Objective: Secondary objectives of the study are categorized as: 1) Dexterity and motility; 2) Muscle quantitative testing of upper and lower limbs; 3) Fatigue; 4) Quality of life. ;Primary end point(s): The primary outcome measure will be the 6 minute walk test (6MWT) with BORG Scale assessment (0 to 10 rating of perceived exertion score) measured at the end of the 24 months intervention period. The 6MWT is a widely used walking test: the distance walked in 6 minutes is measured and a greater distance indicates a better performance. The 6MWT has been validated for DM1 patients and is a sensitive measure that shows deterioration of motility in a relative short term within the natural history of the disease;Timepoint(s) of evaluation of this end point: After 24 months of treatment

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Secondary

Measure	Time frame
Secondary end point(s): 1. Dexterity and motility (measurement time 20 min); 2. Muscle strength (20 min); 3. Fatigue (10 min); 4. Quality of life (10 min); 4. Analysis of alternative splicing regulated by metformin; 5. Biochemical analysis to quantify markers of oxidative stress in order to assess their sensitivity to metformin treatment.;Timepoint(s) of evaluation of this end point: After 24 months of treatment	—

Measure

Time frame

Secondary end point(s): 1. Dexterity and motility (measurement time 20 min); 2. Muscle strength (20 min); 3. Fatigue (10 min); 4. Quality of life (10 min); 4. Analysis of alternative splicing regulated by metformin; 5. Biochemical analysis to quantify markers of oxidative stress in order to assess their sensitivity to metformin treatment.;Timepoint(s) of evaluation of this end point: After 24 months of treatment

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Countries

Italy

Contacts

Public ContactDipartimento di Neuroscienze

Fondazione Policlinico Tor Vergata

massa@uniroma2.it0620903014

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026