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

Trial ID: NCT06328725

Interventional - Phase 1/2 - Unknown status

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

Conditions

• Duchenne Muscular Dystrophy

Interventions

• EN001

Eligibility

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.

Sponsor

ENCell

Source: ClinicalTrials.gov

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