The Efficacy and Safety of Metoprolol as add-on Treatment to Standard of Care in Preventing Cardiomyopathy in Patients With DMD

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Brief Title

The Efficacy and Safety of Metoprolol as add-on Treatment to Standard of Care in Preventing Cardiomyopathy in Patients With DMD

Official Title

The Efficacy and Safety of Metoprolol as add-on Treatment to Standard of Care in Preventing Cardiomyopathy in Patients With Duchenne Muscular Dystrophy Aged 8-16 Years. A Randomized, Double-blind, Placebo-controlled Study

Brief 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.
    

Detailed 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 Phase

Phase 3

Study Type

Interventional


Primary Outcome

Change in left ventricular ejection fraction (LVEF %) by Teichholtz method (echocardiography), compared to baseline at Interim Analysis and Final Analysis.

Secondary Outcome

 DFS (the time to develop clinically evident heart failure)

Condition

Muscular Dystrophy, Duchenne

Intervention

Metoprolol Succinate

Study Arms / Comparison Groups

 Control Group
Description:  Matching placebo will be supplied by the sponsor in child-proof bottles containing dividable tablets with the following dosage of 25mg of IMP and 100mg of IMP. The drug will be administered orally at singular daily doses ranging from 0.75 to 4.5 mg/kg over a Double-Blind Treatment Period (DBTP) of up to 60 months or less dependently on the time of enrolment. The Treatment Period will begin with up to 12-weeks long Up-titration Phase, during which the dose will be gradually escalated. If patient presents with signs and symptoms of intolerance the dose may be temporarily or permanently downgraded at the discretion of the clinician. The up titration ends with reaching maximal tolerated dose level or at a dose corresponding to 4.5 mg/kg.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

150

Start Date

August 18, 2021

Completion Date

June 30, 2026

Primary Completion Date

June 30, 2023

Eligibility Criteria

        Inclusion Criteria:

          -  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)

          -  Stated willingness to comply with all study procedures and availability for the
             duration of the study

          -  Ability to take oral medication and be willing to adhere to the study intervention
             regimen

          -  Subject has confirmed diagnosis of DMD, as defined as clinical picture consistent with
             typical DMD and: i) Dystrophin immunofluorescence and/or immunoblot showing complete
             dystrophin deficiency, or ii) Identifiable mutation within the DMD gene
             (deletion/duplication of one or more exons), where reading frame can be predicted as
             'out-of-frame' or, iii) 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)

          -  Taking ACEi treatment at minimum required doses for at least 30 days

        Exclusion Criteria:

          -  Current or previous permanent use of any beta-blocker medication

          -  Treatment with another investigational drug or other intervention within 3 months
             prior to screening

          -  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

          -  Presence of pacemaker or ICD

          -  Clinical signs or symptoms of heart failure

          -  Left ventricular Ejection Fraction (LVEF) <57% (assessed by Teichholtz
             echocardiography)

          -  Inability to obtain adequate quality echocardiography images (necessary to monitor for
             primary endpoint and safety)

          -  Known allergic reactions to components of the IMPs
      

Gender

Male

Ages

8 Years - 17 Years

Accepts Healthy Volunteers

No

Contacts

, 58349 2899, [email protected]

Location Countries

Poland

Location Countries

Poland

Administrative Informations


NCT ID

NCT05066633

Organization ID

NBK 154/1/2020

Secondary IDs

2020-004901-29

Responsible Party

Sponsor

Study Sponsor

Medical University of Gdansk


Study Sponsor

, , 


Verification Date

September 2021