NCT06328725

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

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

• Status: Unknown
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 88 (estimated)
• Sponsor: ENCell · Industry
• Sex: Male
• Age: 6 Years – 11 Years
• Healthy volunteers: Not accepted

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

Detailed description

This clinical trial is a multi-center study conducted in two phases: Phase 1 and Phase 2. Phase 1 follows a 3+3 dose-escalation design to assess the safety, efficacy, and tolerability of EN001, an investigational product. Phase 2 evaluates the efficacy and safety of EN001 at the recommended phase 2 dose (RP2D), as determined in Phase 1, compared to a placebo. Phase 1 is designed using the traditional 3+3 dose-escalation method to determine the maximum tolerated dose (MTD) and establish the RP2D. Dose escalation continues until the MTD is identified, which must be within the maximum planned dose (MPD) of 2.5 x 10\^6 cells/kg (Cohort 2) or lower. The MTD is defined as the highest dose at which the incidence rate of dose-limiting toxicity (DLT) is less than 33%. To determine the MTD, 3-6 subjects are enrolled in each dose cohort. They receive EN001 every 6 weeks for 3 cycles, with DLTs evaluated up to the 2-week time point (Visit 7). The Safety Review Committee (SRC) consists of the coordinating Investigator, the responsible trial monitor for subjects enrolled in cohorts requiring safety review, and the sponsor. These members participate as committee members. At the conclusion of each cohort-defined as the endpoint of the DLT assessment for the last subject in that cohort-they comprehensively review the safety data for EN001. The committee makes decisions related to dose adjustments, whether to increase or decrease the dose, and ultimately determines the RP2D. Phase 2 clinical trials are randomized, double-blind, placebo-controlled clinical trials. In phase 2, eligible subjects will be randomly assigned to the test group (recommended phase 2 dose (RP2D) of EN001) or the control group (placebo of EN001) in a 1:1 ratio. Efficacy and safety will be evaluated up to 48 weeks after EN001 administration compared to placebo. In addition, test subjects participating in phase 1 and phase 2 will be followed up for safety and effectiveness for 5 years from the time of EN001 administration according to the long-term follow-up protocol.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	EN001	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.

Timeline

• Start date: 2024-03-01
• Primary completion: 2025-11-01
• Completion: 2025-11-01
• First posted: 2024-03-25
• Last updated: 2024-03-25

Locations

2 sites across 1 country: South Korea