protocolSection identificationModule nctId: NCT06328725, orgStudyIdInfo id: EN001_POWER, briefTitle: Evaluate the Efficacy and Safety of EN001 in Patients With Duchenne Muscular Dystrophy, statusModule overallStatus: NOT_YET_RECRUITING, startDateStruct date: 2024-03, primaryCompletionDateStruct date: 2025-11, completionDateStruct date: 2025-11, studyFirstPostDateStruct date: 2024-03-25, lastUpdatePostDateStruct date: 2024-03-25, sponsorCollaboratorsModule leadSponsor name: ENCell, class: INDUSTRY, descriptionModule briefSummary: 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, conditionsModule conditions: Duchenne Muscular Dystrophy, designModule studyType: INTERVENTIONAL, phases: PHASE1, phases: PHASE2, designInfo allocation: RANDOMIZED, interventionModel: PARALLEL, interventionModelDescription: \<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)., primaryPurpose: TREATMENT, maskingInfo masking: DOUBLE, maskingDescription: 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, whoMasked: PARTICIPANT, whoMasked: INVESTIGATOR, enrollmentInfo count: 88, type: ESTIMATED, armsInterventionsModule interventions name: EN001, outcomesModule primaryOutcomes measure: <Phase 1> Adverse drug reactions related to dose limiting toxicity (DLT), primaryOutcomes measure: <Phase 1> Adverse drug reactions related to discontinuation of clinical trial drug administration, primaryOutcomes measure: <Phase 2> Change in time to stand test (TTSTAND), secondaryOutcomes measure: <Phase 1> Time to stand test (TTSTAND) change amount, secondaryOutcomes measure: <Phase 1> TTSTAND velocity (1/TTSTAND) change amount, secondaryOutcomes measure: <Phase 1> Time to run/walk 10 meters test (TTRW) change amount, secondaryOutcomes measure: <Phase 1> TTRW velocity (1/TTRW) change amount, secondaryOutcomes measure: <Phase 1> North Star Ambulatory Assessment (NSAA) change amount, secondaryOutcomes measure: <Phase 1> Time to climb 4 steps test (TTCLIMB) change amount, secondaryOutcomes measure: <Phase 1> TTCLIMB velocity (1/TTCLIMB) change amount, secondaryOutcomes measure: <Phase 1> 6-minute walk test (6MWT) change amount, secondaryOutcomes measure: <Phase 1> Changes amount in muscle strength by region, secondaryOutcomes measure: <Phase 1> Changes amount in parameters related to pulmonary function, secondaryOutcomes measure: <Phase 1> Changes amount in parameters related to cardiac function, secondaryOutcomes measure: <Phase 1> Change rate of creatine kinase (CK) at each visit after administration of investigational product compared to baseline (Visit 2), secondaryOutcomes measure: <Phase 2> Time to stand test (TTSTAND) change amount, secondaryOutcomes measure: <Phase 2> TTSTAND velocity (1/TTSTAND) change amount, secondaryOutcomes measure: <Phase 2> Time to run/walk 10 meters test (TTRW) change amount, secondaryOutcomes measure: <Phase 2> TTRW velocity (1/TTRW) change amount, secondaryOutcomes measure: <Phase 2> North Star Ambulatory Assessment (NSAA) change amount, secondaryOutcomes measure: <Phase 2> Time to climb 4 steps test (TTCLIMB) change, secondaryOutcomes measure: <Phase 2> TTCLIMB velocity (1/TTCLIMB) change amount, secondaryOutcomes measure: <Phase 2> 6-minute walk test (6MWT) change amount, secondaryOutcomes measure: <Phase 2> Changes amount in muscle strength by region, secondaryOutcomes measure: <Phase 2> Changes amount and rate of change in whole thigh muscle volume and index assessed by MRI, secondaryOutcomes measure: <Phase 2> Changes amount in parameters related to pulmonary function, secondaryOutcomes measure: <Phase 2> Changes amount in parameters related to cardiac function, secondaryOutcomes measure: <Phase 2> Change rate of creatine kinase (CK) at each visit after administration of investigational product compared to baseline (Visit 2), secondaryOutcomes measure: <Phase 2> Pediatric Outcomes Data Collection Instrument (PODCI) item score and total score change, secondaryOutcomes measure: <Phase 2> Pediatric Quality of Life inventory™ (PedsQL™) item scores and total score change, secondaryOutcomes measure: <Phase 1> Adverse Event, secondaryOutcomes measure: <Phase 1> Laboratory examination, secondaryOutcomes measure: <Phase 1> Vital sign, secondaryOutcomes measure: <Phase 2> Adverse Event, secondaryOutcomes measure: <Phase 2> Laboratory examination, secondaryOutcomes measure: <Phase 2> Vital sign, eligibilityModule sex: MALE, minimumAge: 6 Years, maximumAge: 11 Years, stdAges: CHILD, contactsLocationsModule locations facility: Samsung Medical Center, city: Seoul, country: Korea, Republic of, contacts name: Jeehun Lee, role: PRINCIPAL_INVESTIGATOR, geoPoint lat: 37.566, lon: 126.9784, locations facility: Seoul National University Hospital, city: Seoul, country: Korea, Republic of, contacts name: Jong-Hee Chae, role: PRINCIPAL_INVESTIGATOR, geoPoint lat: 37.566, lon: 126.9784, hasResults: False