Study of ATX-01 in Participants With DM1
Recruiting
Phase 1
Phase 2
Interventional Study
Myotonic Dystrophy 1
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 18 – 64
- Sex
- Any
- Trial phase
- Phase 1/2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 56 (estimated)
- Sponsor
- ARTHEx Biotech S.L. · Industry
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About This Trial
The goal of this clinical trial is to test ATX-01 in participants with myotonic dystrophy type 1 (DM1). The main question it aims to answer is if ATX-01 is safe and well tolerated. The trial will compare the safety and tolerability of ATX-01 and a matching placebo.
There will be a single-ascending dose part of the trial and a multiple-ascending dose part. In the single-ascending dose, participant…
Trial Locations
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Eligibility Criteria
Key Inclusion Criteria:
* Participants with a documented clinical diagnosis of DM1 (CTG expansion of \>150 repeats in DMPK gene measured in peripheral blood mononuclear cells)
* Ambulatory, defined as able to complete a 10-meter walk/run test at screening without the use of assistive devices such a…
Contacts