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UK sites announced for myotonic dystrophy type 1 clinical trial

5 July 2024

Avidity Biosciences have announced they are starting to enrol participants into the Phase 3 HARBOR clinical trial for adults living with myotonic dystrophy type 1 (DM1).

The HARBOR clinical trial is a global, randomised, double-blind study, investigating how delpacibart etedesiran, previously known as AOC 1001 and also shortened to del-desiran, works for people aged 16 and over living with DM1. This trial was set up following positive results from the Phase 1/2 MARINA trial.

The trial is planned to open in 40 sites across the world, including the UK – for a detailed list of sites please see clinicaltrial.gov. In the UK, trials are planned in Oxford (Cancer and Haematology Centre) and Cardiff (University Hospital of Wales). For questions about the study, find the contact details here.

Myotonic dystrophy type 1 is a genetic muscle wasting condition that typically affects the skeletal muscles which help us move and breathe, heart muscles, and muscles, called smooth muscles, found within organs. A change in gene DMPK leads to the accumulation of DMPK in cells, which causes the condition.

Del-desiran is designed to reduce levels of DMPK accumulated in the skeletal, heart and smooth muscle of people living with DM1, targeting the root cause of the condition.

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