A Multi-center, Randomized, Double-blind, Placebo-controlled, Phase 1/2 Trial to Evaluate the Efficacy and Safety of EN001 in Patients With Duchenne Muscular Dystrophy

EN001_POWER

A Multi-center, Randomized, Double-blind, Placebo-controlled, Phase 1/2 Trial to Evaluate the Efficacy and Safety of EN001 in Patients with Duchenne Muscular Dystrophy

2024-03-01

2025-11-01

2025-11-01

Not yet recruiting

Early phase I

88

Inclusion Criteria: 1. Males aged between 6 and 11 years at the time of providing written consent. 2. Individuals exhibiting phenotypic signs of Duchenne Muscular Dystrophy (DMD), such as lower limb muscle weakness, a duck walk, or Gower's sign, and who are diagnosed with DMD following confirmation of a dystrophin gene mutation through genetic testing. 3. Participants who meet the Time to Stand Test (TTSTAND) criteria without the use of assistive devices or help from others during screening and baseline assessments: * Phase 1: Capable of completing the TTSTAND evaluation. * Phase 2: TTSTAND time of 10 seconds or less. 4. Participants with a 6-Minute Walk Test (6MWT) result of 75 meters or more at screening and baseline. 5. Individuals who meet the following laboratory test criteria at the time of screening and baseline: * Hemoglobin ≥10 g/dL * Platelet ≥50,000/μL * Serum albumin ≥2.5 g/dL * Gamma glutamyl transferase (γ-GT) and total bilirubin ≤ upper limit of normal (ULN) * Serum creatinine ≤ 1.5 x ULN 6. Participants who have been on a stable dose of glucocorticoids for at least 12 weeks prior to screening, with treatment maintained. Dosage adjustments for body weight changes are allowed. 7. Individuals who, along with their representatives when applicable, have voluntarily agreed in writing to participate in this clinical trial. Exclusion Criteria: 1. Individuals with confirmed comorbidities at the time of screening: * Left ventricular ejection fraction (LVEF) below 50%, as determined by echocardiography * Percent predicted forced vital capacity (FVC%) less than 35% * Positive for Hepatitis B surface antigen (HBsAg). However, individuals undergoing interferon or antiviral treatment can register * Positive for Hepatitis C virus antibody (HCV Ab). Registration is possible if the HCV ribonucleic acid (RNA) test result is negative * Positive for Human immunodeficiency virus (HIV) antibody * Comorbidities that are uncontrollable or require treatment that could affect the safety and efficacy evaluation of this clinical trial, based on the investigator's judgment 2. Individuals with confirmed treatment history at the time of screening: * Administration of cell therapy or gene therapy throughout life * Administer antisense oligonucleotide (e.g., exon skipping treatment) or stop- codon readthrough treatment (e.g., aminoglycoside, ataluren) within 24 weeks before screening. * Administration of the following medications within 12 weeks before screening: Idebenone, Resveratrol, Adenosine triphosphate * Administration of the following medications within 12 weeks before screening. However, registration is possible if the drug is being administered at a stable dose for at least 12 weeks before screening and the dose is expected to remain unchanged during the clinical trial period. Angiotensin-converting enzyme (ACE) inhibitor Angiotensin II receptor blocker (ARB) Beta-blocker Aldosterone antagonist Ivabradine Sacubitril Growth hormone Anabolic steroids * Major surgery within 12 weeks before screening or expected major surgery during the clinical trial period. * Use of other investigational products (or medical devices) within 4 weeks before screening. * Use of systemic immunosuppressants other than systemic glucocorticoids. 3. Individuals requiring mechanical ventilation during the day. 4. Persons with hypersensitivity to the components of the clinical investigational products. 5. Individuals unwilling to use appropriate contraception from the date of written consent to the termination visit: * Appropriate contraceptive methods are as follows, and use more than one method. * The use of hormonal contraceptives by the partner * Implantation of an intrauterine device or system in your partner * Sterilization or surgical procedures for you or your partner 6. Others who, in the investigator's discretion, are not willing or able to comply with the clinical trial procedures.

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2024-03-25