A study of ATL1102 in boys with DMD who cannot walk independently and need wheelchair assistance
Research type
Research Study
Full title
A multicentre, randomised, double-blind, placebo-controlled and open label extension study to assess the efficacy, safety, and pharmacokinetic profile of two dose levels of ATL1102 administered by subcutaneous injection in non-ambulatory participants with Duchenne Muscular Dystrophy
IRAS ID
1005179
Contact name
Nuket Desem
Contact email
Sponsor organisation
Antisense Therapeutics Limited
Eudract number
2022-003065-38
Research summary
This study of a new drug ATL1102, is for the treatment of Duchenne Muscular Dystrophy (DMD) in non-ambulant participants aged 10 to less than 18 years.
This study will test the effect of ATL1102 on upper limb muscle function in participants who cannot walk independently and require wheelchair assistance. The study will also measure the levels of ATL1102 in the blood, the effects on types of white blood cells and the safety of ATL1102.
ATL1102 works by decreasing inflammation that occurs in muscles of boys with DMD and it is hoped this slows down the weakening of the muscles. Currently, there are limited treatments that slow down this muscle weakening.The study consists of two phases: a double-blind phase followed by an open label extension (OLE) phase. In the double-blind phase ATL1102 will be administered once weekly for 25 weeks. There are 3 possible treatment groups: ATL1102 at two different concentrations and the placebo (injection that does not contain the active drug). The study is randomised, which means the medication participants receive is allocated randomly (like flipping a coin). Participants will have a 1 in 3 chance of receiving each of the study medications. Neither the participants nor their study doctor will know which study medication they will be taking. This is referred to as double-blind.
The OLE phase will take place after the blinded treatment period. Participants will continue on their current dose of ATL1102 for 24 weeks. Participants who received placebo during the blinded phase will receive the study drug at the placebo dose level to which they were randomised initially. All participants who complete the OLE will then enter the post-treatment follow-up period.
The Sponsor is Antisense Therapeutics Limited. This study will include approximately 45 participants from 12 centres in the United Kingdom, Turkey, Bulgaria and Australia.
REC name
South Central - Hampshire A Research Ethics Committee
REC reference
22/SC/0469
Date of REC Opinion
30 Jun 2023
REC opinion
Further Information Favourable Opinion