Evaluate the Efficacy and Safety of EN001 in Patients With Duchenne Muscular Dystrophy

NCT06328725 | Not Yet Recruiting Phase 1

• 1 other identifier: EN001_POWER
• Study Type: interventional
• Target: 88
• Locations: 1 country
• Sites: 2

Brief Summary

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

Outcome Measures

Primary Outcomes (3)

• <Phase 1> Adverse drug reactions related to dose limiting toxicity (DLT)

  Present the frequency and percentage of dose-limiting toxicity (DLT) occurrence across dose cohorts, along with detailed information on the types of DLTs.

  Up to 14 weeks

• <Phase 1> Adverse drug reactions related to discontinuation of clinical trial drug administration

  Present the frequency and percentage of dose-limiting toxicity (DLT) occurrence across dose cohorts, along with detailed information on the types of DLTs.

  Up to 14 weeks

• <Phase 2> Change in time to stand test (TTSTAND)

  Present the changes in time to stand test (TTSTAND) at the 48-week time point compared to baseline (Visit 2). Provide the subject count, mean, standard deviation, median, minimum, and maximum for the change in each treatment group. Analyze the change as the dependent variable using a repeated measures mixed model (MMRM) with treatment group, visit (6, 12, 18, 24, 36, 48 weeks), and the interaction between treatment group and visit as factors. Include baseline TTSTAND values and age as fixed effects in the analysis.

  At 48 weeks compared to baseline (Visit 2)

Secondary Outcomes (33)

• <Phase 1> Time to stand test (TTSTAND) change amount

  At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)

• <Phase 1> TTSTAND velocity (1/TTSTAND) change amount

  At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)

• <Phase 1> Time to run/walk 10 meters test (TTRW) change amount

  At 6, 12, 18, 24, 36,, and 48 weeks compared to baseline (Visit 2)

• <Phase 1> TTRW velocity (1/TTRW) change amount

  At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)

• <Phase 1> North Star Ambulatory Assessment (NSAA) change amount

  At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)

Study Arms (4)

Phase 1 - Cohort 1

ACTIVE COMPARATOR

EN001 5.0x10\^5 cells/kg

Drug: EN001

Phase 1 - Cohort 2

ACTIVE COMPARATOR

EN001 2.5x10\^6 cells/kg

Drug: EN001

Phase 2 - Experimental Group

PLACEBO COMPARATOR

The recommended phase 2 dose (RP2D) of EN001

Drug: EN001

Phase 2 - Control Group

PLACEBO COMPARATOR

EN001 placebo

Drug: EN001

Interventions

EN001 DRUG

Phase 1 * Cohort 1: EN001 5.0x10\^5 cells/kg administered intravenously (IV) 3 times at 6 week intervals. * Cohort 2: EN001 2.5x10\^6 cells/kg administered intravenously (IV) 3 times at 6 week intervals. Phase 2 * Experimental Group: The recommended phase 2 dose (RP2D) of EN001 is administered intravenously (IV) three times at six-week intervals. * Control Group: EN001 placebo is administered intravenously (IV) three times at six-week intervals.

Also known as: EN001(allogenic early-passage mesenchymal stem cells derived from Wharton's jelly (WJ-MSCs) )

Phase 1 - Cohort 1; Phase 1 - Cohort 2; Phase 2 - Control Group; Phase 2 - Experimental Group

Eligibility Criteria

• Age: 6 Years - 11 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Males aged between 6 and 11 years at the time of providing written consent.
• 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.
• 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.
• Participants with a 6-Minute Walk Test (6MWT) result of 75 meters or more at screening and baseline.
• 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
• 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.
• Individuals who, along with their representatives when applicable, have voluntarily agreed in writing to participate in this clinical trial.

You may not qualify if:

• 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
• 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.

Sponsors & Collaborators

• ENCell: lead

Study Sites (2)

Samsung Medical Center

Seoul, South Korea

Location

Seoul National University Hospital

Seoul, South Korea

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

ENCell

CONTACT

82-2-6205-8054; encell@encellinc.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: The investigational drug for the clinical trial of the following cohort or group will be administered intravenously (IV) three times at 6-week intervals. \<Phase 1 Clinical Trial\> Cohort 1: EN001 5.0x10\^5 cells/kg / Cohort 2: EN001 2.5x10\^6 cells/kg \<Phase 2 Clinical Trial\> Experimental Group: Recommended Phase 2 Dose (RP2D) for EN001 / Control Group: Placebo for EN001
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: \<Phase 1 Clinical Trial\> A total of 6-12 participants (3-6 participants per cohort). \<Phase 2 Clinical Trial\> A total of 76 participants (a minimum of 30 participants per group, considering a dropout rate of 20%, resulting in 38 participants per group).

Study Record Dates

• First Submitted: February 19, 2024
• First Posted: March 25, 2024
• Study Start: March 1, 2024
• Primary Completion: November 1, 2025
• Study Completion: November 1, 2025
• Last Updated: March 25, 2024

Record last verified: 2024-03

Locations

KR (2)