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

NCT06328725 · Status: NOT_YET_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 88

Last updated 2024-03-25

No results posted yet for this study

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

Interventions

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.

Sponsors & Collaborators

  • ENCell

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
PARALLEL

Eligibility

Min Age
6 Years
Max Age
11 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-03-31
Primary Completion
2025-11-30
Completion
2025-11-30

Countries

  • South Korea

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06328725 on ClinicalTrials.gov