NCT05938023

A Study of ATL1102 or Placebo in Participants With Non-ambulatory Duchenne Muscular Dystrophy

A Multicentre, Randomised, Double-blind, Placebo-controlled and Open Label Extension Study to Assess the Efficacy, Safety, and Pharmacokinetic Profile of of ATL1102 in Non-ambulatory Participants With Duchenne Muscular Dystrophy

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 48 (actual)
• Sponsor: Percheron Therapeutics · Industry
• Sex: Male
• Age: 10 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

This Phase IIb study is a two part, multicenter study to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of ATL1102 in non-ambulant boys with Duchenne Muscular Dystrophy aged 10 to \<18 years old. The study includes a randomised, double-blind, placebo-controlled treatment period (Part A), followed by an open labelled treatment period (Part B).

Detailed description

This Phase IIb study is a two part, multicenter study to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of ATL1102 and will enroll 45 non-ambulant boys with Duchenne Muscular Dystrophy (DMD) aged 10 to \<18 years old. During the 24 week randomised, double-blind, placebo-controlled treatment period (Part A) participants will be enrolled and randomised to receive either ATL1102 25mg, ATL1102 50mg or matched placebo in a 1:1:1 ratio given as a weekly subcutaneous injection. Participants will then continue to the 24 week Open Labelled Treatment Period (Part B) and continue to receive ATL1102 25mg or ATL1102 50mg for a further 24 weeks. Participants on placebo in Part A will transition to ATL1102. The study will consist of a 4 week screening period, 24 week randomised, double-blind, placebo-controlled treatment period (Part A), 24 week open label treatment period (Part B) and 16 week follow up period.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	ATL1102 25mg	Dose and scheduled as specified in the Arm description
DRUG	ATL1102 50mg	Dose and scheduled as specified in the Arm description
DRUG	Placebo	Dose and scheduled as specified in the Arm description

Timeline

• Start date: 2023-05-18
• Primary completion: 2024-11-19
• Completion: 2025-01-15
• First posted: 2023-07-10
• Last updated: 2025-02-03

Locations

13 sites across 5 countries: Australia, Bulgaria, Serbia, Turkey (Türkiye), United Kingdom