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

Phase 1

• 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; Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

• Registration Number: EUCTR2018-000692-32-IT
• Lead Sponsor: DIP. MEDICINA DEI SISTEMI UNIVERSITà DEGLI STUDI DI ROMA TOR VERGATA

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-01-14
• Last Update Posted: 1 February 2020

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Not specified
• Target Recruitment: 194

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 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, < 60 ml/min); Any acute condition which can lead to renal dysfunction (severe infection or injury, dehydration, shock); Heart
complications such as heart failure (even if the condition is under control) or recent heart attack; Respiratory failure (patients with nocturnal non-invasive ventilation CPAP or BiPAP could be included); Liver disease; Excessive alcohol intake within 12 months from enrollment; Acute or chronic metabolic acidosis; Diabetic complications such as diabetic coma and ketoacidosis
-Symptomatic insulin-requiring diabetes or type 2 diabetes requiring oral anti-diabetic agents
-Any history of malignancy except for cases of remission > 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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified