The double-blind, placebo-controlled phase I/II ACHIEVE trial is evaluating DYNE-101. This drug candidate for Steinert’s disease is being developed by the company Dyne Therapeutics. It is an antisense oligonucleotide coupled to an antibody fragment targeting the hTfR1 receptor (present in large quantities on the surface of muscle cells) administered by intravenous infusion.
The Institute of Myology was the first investigator centre opened in Europe. Five other centres are planned in Europe and New Zealand to recruit 64 patients.
Dr Guillaume Bassez is the principal investigator of the trial at the Institute. On the patient side, after an inclusion period to check that each candidate meets all the criteria required by the trial protocol, participants will receive DYNE-101 or placebo by intravenous infusion every four to eight weeks. Medical examinations and blood and muscle samples will be taken regularly. The trial is expected to end in 2026.
Further details on Clinicaltrials.gov : Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1 – ClinicalTrials.gov (visited on 23/02/23)
