Viewing Study NCT06328725



Ignite Creation Date: 2024-05-06 @ 8:18 PM
Last Modification Date: 2024-10-26 @ 3:24 PM
Study NCT ID: NCT06328725
Status: NOT_YET_RECRUITING
Last Update Posted: 2024-03-25
First Post: 2024-02-19

Brief Title: Evaluate the Efficacy and Safety of EN001 in Patients With Duchenne Muscular Dystrophy
Sponsor: ENCell
Organization: ENCell

Study Overview

Official Title: A Multi-center Randomized Double-blind Placebo-controlled Phase 12 Trial to Evaluate the Efficacy and Safety of EN001 in Patients With Duchenne Muscular Dystrophy
Status: NOT_YET_RECRUITING
Status Verified Date: 2024-03
Last Known Status: None
Delayed Posting: No
If Stopped, Why?: Not Stopped
Has Expanded Access: False
If Expanded Access, NCT#: N/A
Has Expanded Access, NCT# Status: N/A
Acronym: None
Brief Summary: A Multi-center Randomized Double-blind Placebo-controlled Phase 12 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 33 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 33 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 25 x 106 cellskg 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 11 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

Study Oversight

Has Oversight DMC: None
Is a FDA Regulated Drug?: False
Is a FDA Regulated Device?: False
Is an Unapproved Device?: None
Is a PPSD?: None
Is a US Export?: None
Is an FDA AA801 Violation?: None