Phase 1/2a study of ATX-01 in participants with DM1 (ArthemiR study)
Research type
Research Study
Full title
A Phase 1/2a Double-Blind, Placebo-controlled, Single- and Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Clinical Efficacy of Intravenous Administration of ATX-01 In Male and Female Participants aged 18 to 64 with Classic Myotonic Dystrophy Type 1 (DM1)
IRAS ID
1008471
Contact name
Judith Walker
Contact email
Sponsor organisation
Arthex Biotech, S.L.
Research summary
This study is being conducted to learn more about ATX-01, an experimental medication in participants with DM1. Also, the study will look at the effects of ATX-01 in the body, in particular in muscles. ATX-01 is expected to work by increasing the levels of the MBNL protein. This would mean that there is more MBNL protein available and more of the affected proteins could be produced correctly. The main objective of the study is to evaluate the safety and tolerability of ATX-01. In addition, the study will look at the effects of ATX-01 in the body, in particular in muscles. The study consists of 2 parts. The first part is the single ascending dose. This means that each participant will receive ATX-01 or placebo 1 time. Participants will be allocated to ATX-01 or placebo by chance. The first 4 participants will receive the lowest dose of ATX-01. An independent group of experts will review the data from these 4 participants and give a recommendation if the next dose level can go ahead. This will be repeated for Dose Level 2, Dose Level 3, and Dose Level 4 (8 participants in each Dose Level after Dose Level 1). After a participant is confirmed to be eligible, Part 1 will last approximately 13 weeks. The second part of the study is called the multiple ascending dose. In this part, each participant will receive ATX-01 or placebo 3 times. Part 2 will start at Dose Level 2 and will progress to Dose Level 3 and Dose Level 4 (8 participants at each Dose Level), with the independent group of experts reviewing data in between each dose level. Male and female participants aged 18 and 64 years with a diagnosis of DM1, who are able to walk, will be included. An optional long-term extension study is planned under a separate protocol for participants completing either part or both parts of the study. Participants will be monitored throughout the study, participation is voluntary and they will be required to sign the Informed Consent Form before any study procedures can start.
REC name
London - Fulham Research Ethics Committee
REC reference
24/LO/0191
Date of REC Opinion
8 May 2024
REC opinion
Further Information Favourable Opinion