Clinical Trials Logo

Clinical Trial Summary

The study includes 150 patients with DMD diagnosis confirmed by genetic testing, 8-16 years old (≥8 and <17) at the study entry with a follow-up of up to 5 years. Random enrollment of a patient to one of two groups (intervention or control) takes place after pre-screening and screening stage starts the first phase of the trial. To be eligible for participation in the study, patients must receive standard of care cardiac therapy, which is an Angiotensin-converting-enzyme inhibitor (ACEi) for at least one-month prior to enrollment. A major part of the trial is equal for all patients - who will be receiving indistinguishable investigational medicinal products (IMPs), the drug metoprolol succinate or placebo. As a part of the clinical trial, diagnostic examinations evaluating progression of the disease, will be performed periodically. In addition, all patients will be monitored at home. Heart rate, blood pressure and patients' personal well-being will be controlled using telemedicine technologies. Additional visits in the research center will be provided if any adverse events occur. This model will be continued for 30 months from the enrollment of a first patient. After this period the first drug efficiency analysis will be performed. After that, the intervention may be continued or in case of negative impact of the intervention on patients' health and well-being, terminated with further patients monitoring.

Clinical Trial Description

Patients will be evaluated for inclusion during a Screening period of up to one year (possible to extend for another year if warranted). Written informed consent from the patient and/or parent/legal guardian and assent, if applicable, to participate in the study must be obtained prior to beginning any study-related procedures. Once eligibility is confirmed, patients will undergo Screening Assessments. Two groups will receive either metoprolol succinate (Treatment 1 - Intervention) at a dose depending on the weight category as described below or matching placebo (Treatment 2 - Control) to maintain the study blinding, one per day for up to 60 months. All patients (both arms) will receive ACEi (e.g. perindopril, enarenal, lisinopril) or, if indicated, ARB (e.g. losartan) as a standard of care at an appropriate therapeutic dose (see below) through all the study duration. A total of approximately 150 subjects will be randomized into the study. Subjects will be assessed for safety and tolerability, clinical efficacy, at scheduled visits throughout the study. Adverse events, including SAEs, and concomitant medications will be recorded throughout the study in eCRF record. Access credentials for ePROP will be dispensed to the participant or his legal guardian when appropriate at the Enrolment Visit to record vital signs (HR and NIBP) and peak-flow-meter readings, AEs, changes to concomitant medications taken during the study, and any missed or incomplete doses of study medication. Site study staff will contact the parent(s)/ legal guardian(s) by telephone at quarterly to ensure that the tablet dispensing is proceeding according to protocol and to address any questions the parent(s)/guardian(s) may have. All subjects will periodically return to the clinical site for assessments according to the Visit Plan. The study is comprised of a Pre-screening, Screening-Enrolment-Randomization Double-Blind Treatment Period further subdivided into Phases. Subjects will be enrolled into this study at the time written informed consent is given and randomized to treatment only after completion of all Pre-treatment Screening and Enrolment assessments. Study drug dosing will occur at home on all days starting from Enrolment Visit until the Final Visit. In the event that relevant clinical or laboratory parameters remain abnormal at the time of discharge from the study, the subject will be followed medically, as clinically indicated. Any subject who prematurely discontinues the study should return to the study centre for scheduled assessments at the time of early withdrawal, whenever possible assuming the subject has not withdrawn consent. ;

Study Design

Related Conditions & MeSH terms

NCT number NCT05066633
Study type Interventional
Source Medical University of Gdansk
Contact Joanna Kwiatkowska, MD, PhD
Phone 58349 2899
Email [email protected]
Status Recruiting
Phase Phase 3
Start date August 18, 2021
Completion date June 30, 2026

See also
  Status Clinical Trial Phase
Terminated NCT01865084 - A Study of Tadalafil for Duchenne Muscular Dystrophy Phase 3
Completed NCT00243789 - Study of Daily Pentoxifylline as a Rescue Treatment in Duchenne Muscular Dystrophy Phase 1/Phase 2
Completed NCT00033189 - An Open-label Pilot Study of Coenzyme Q10 in Steroid-Treated Duchenne Muscular Dystrophy Phase 2
Completed NCT03703882 - Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy Phase 3
Enrolling by invitation NCT04626674 - A Gene Delivery Study to Evaluate the Safety of and Expression From SRP-9001 in Duchenne Muscular Dystrophy (DMD) Phase 1
Completed NCT02286947 - Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy Phase 2
Completed NCT03406780 - A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy Phase 2
Completed NCT01826487 - Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Phase 3
Completed NCT02710591 - Rimeporide in Patients With Duchenne Muscular Dystrophy Phase 1
Completed NCT01826422 - Effect of EPA and DHA in the Inflammation and Metabolic Disorders in DMD/DMB Patients N/A
Completed NCT00102453 - Pentoxifylline in Duchenne Muscular Dystrophy Phase 1/Phase 2
Terminated NCT02090959 - An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy Phase 3
Completed NCT03789734 - Safety Study of BLS-M22 in Healthy Volunteers Phase 1
Completed NCT00016653 - Creatine and Glutamine in Steroid-Naive Duchenne Muscular Dystrophy Phase 2/Phase 3
Completed NCT03127241 - User-centred Assistive System for Arm Functions in Neuromuscular Subjects N/A
Completed NCT03490214 - Non-invasive Imaging of Muscle Structure in Duchenne Muscular Dystrophy Using Multispectral Optoacoustic Tomography N/A
Active, not recruiting NCT03179631 - Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy Phase 3
Terminated NCT03400852 - A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy Phase 2
Completed NCT01027884 - Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD) Phase 3
Active, not recruiting NCT03992430 - A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Duchenne Muscular Dystrophy (DMD) Patients (MIS51ON) Phase 3