Metoprolol Safety and Efficacy in the Prevention of Cardiomyopathy in Patients with Duchenne Muscular Dystrophy (DMD).

Phase 1

• Conditions: Duchenne muscular dystrophyCardiomyopathyTachycardia; MedDRA version: 20.0Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2020-004901-29-PL
• Lead Sponsor: Medical University of Gdansk

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-01-20
• Last Update Posted: 15 November 2021

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Male
• Target Recruitment: 144

Inclusion Criteria

In order to be eligible to participate in this study, an individual must meet all of the following criteria:
1. Subject's parent(s) or legal guardian(s) has (have) provided written informed consent, where applicable, prior to any study-related procedures; participants will be asked to give written or verbal assent
according to requirements (>16 years old)
2. Stated willingness to comply with all study procedures and availability for the duration of the study
3. Male, = 8 years and <17 years of age at time of enrolment in the study
4. Ability to take oral medication and be willing to adhere to the study intervention regimen
5. Subject has confirmed diagnosis of DMD, as defined as clinical picture consistent with typical DMD and:
• Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, or
• Identifiable mutation within the DMD gene (deletion/duplication of one or more exons), where reading frame can be predicted as 'out-offrame'
or
• Complete dystrophin gene sequencing showing an alteration (point mutation, duplication, other) that is expected to preclude production of the dystrophin protein (i.e., nonsense mutation, deletion/duplication
leading to a downstream stop codon)
6. Taking ACEi treatment at minimum required doses as outlined in the protocol for at least 30 days
Are the trial subjects under 18? yes
Number of subjects for this age range: 144
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

An individual who meets any of the following criteria will be excluded from participation in this study:
1. Current or previous permanent use of any beta-blocker medication
2. Treatment with another investigational drug or other intervention within 3 months prior to screening
3. Clinically significant bradycardia at rest or by Holter ECG, based on age and sex adjusted normal values, atrioventricular block higher than first degree at rest, or second degree Wenckebach at night, pauses longer than 2.5 seconds
4. Presence of pacemaker or ICD
5. Clinical signs or symptoms of heart failure
6. Left ventricular Ejection Fraction (LVEF) <57% (assessed by Teichholtz echocardiography)
7. Inability to obtain adequate quality echocardiography images (necessary to monitor for primary endpoint and safety)
8. Known allergic reactions to components of the IMPs

Study & Design

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