NCT05338099

Determine the Safety and Dose of EN001 in Patients With Duchenne Muscular Dystrophy(DMD)

Open-label, Dose-escalation, Phase 1 Clinical Trial to Determine the Safety and Dose of EN001 in Patients With Duchenne Muscular Dystrophy(DMD)

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 7 (actual)
• Sponsor: ENCell · Industry
• Sex: Male
• Age: 2 Years – 18 Years
• Healthy volunteers: Not accepted

Summary

Open-label, Dose-escalation, Phase 1 Clinical Trial to Determine the Safety and Dose of EN001 in Patients with Duchenne Muscular Dystrophy(DMD)

Detailed description

It is the first in human (FIH), 3+3 design clinical trial to evaluate the safety and tolerability and determine the maximum tolerated dose (MTD) of EN001 (allogeneic umbilical cord-derived mesenchymal stem cells) in the treatment of Duchenne Muscular Dystrophy (DMD).

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	EN001	EN001 intravenously (IV) in the treatment of Duchenne Muscular Dystrophy (DMD) Dosage for each group is as follows. Dose group A (Low dose): 5.0x10\^5 cells/kg
DRUG	EN001	EN001 intravenously (IV) in the treatment of Duchenne Muscular Dystrophy (DMD) Dosage for each group is as follows. Dose group B (High dose): 2.5x10\^6 cells/kg

Timeline

• Start date: 2022-01-18
• Primary completion: 2022-12-28
• Completion: 2022-12-28
• First posted: 2022-04-20
• Last updated: 2024-08-30

Locations

1 site across 1 country: South Korea