Study of ATX-01 in Participants with DM1
- Registration Number
- NCT06300307
- Lead Sponsor
- ARTHEx Biotech S.L.
- Brief Summary
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, participants will receive one dose of ATX-01 or placebo. In the multiple-ascending dose part, participants will receive three doses of ATX-01 or placebo.
ATX-01 is a novel anti-miR (synthetic single stranded oligonucleotide) that inhibits a microRNA called miR-23b.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 56
- 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 as canes, walkers, or orthoses, except for ankle-foot orthoses
- Presence for >3 seconds of grip myotonia as confirmed by a central reader
Key
- Participants with congenital DM1
- Medical Research Council Muscle Scale score of less than 4 on ankle dorsiflexion or significant tibialis anterior atrophy that prevents a muscle biopsy
- Use of mexiletine or other agent for myotonia within 21 days or 5 half-lives, whichever is longer, prior to screening
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description ATX-01 ATX-01 ATX-01 is a formulation of the anti-microRNA 23b (anti-miR-23b), known as X82108, a novel type of antisense oligonucleotide Placebo Placebo Placebo to ATX-01
- Primary Outcome Measures
Name Time Method Incidence of adverse events Up to 120 days To evaluate the safety and tolerability of ATX-01 in adult participants with DM1
- Secondary Outcome Measures
Name Time Method Maximum observed plasma concentration (Cmax) of ATX-01 Up to 48 hours post-dose Area under the plasma concentration-time curve (AUC) of ATX-01 Up to 48 hours post-dose Video hand opening time Change from baseline up to 120 days To evaluate the efficacy of ATX-01 on myotonia in participants with DM1
Change from baseline in ankle dorsiflexion strength by quantitative myometry Change from baseline up to 120 days To evaluate the effects of ATX-01 in participants with DM1 on ankle dorsiflexion strength
Change from baseline in Impact on Activities of Daily Living questionnaire item scores Change from baseline up to 120 days The Impact on Activities of Daily Living questionnaire is a 7-item patient-reported outcome designed to evaluate the impact of ATX-01 on activities of daily living in participants with DM1.
Incidence of clinically significant changes in laboratory assessments, electrocardiograms (ECGs), vital signs, suicidal ideation and behavior Up to 120 days To further evaluate the safety and tolerability of ATX-01 in adult participants with DM1
Trial Locations
- Locations (9)
University of Florida
🇺🇸Gainesville, Florida, United States
University of Kansas Medical Center, Department of Neurology
🇺🇸Fairway, Kansas, United States
Virginia Commonwealth University
🇺🇸Richmond, Virginia, United States
Centre Intégré Universitaire de Santé et Services Sociaux du Saguenay-Lac-St-Jean
🇨🇦Chicoutimi, Quebec, Canada
Institute of Myology
🇫🇷Paris, France
The NeMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan
🇮🇹Milan, Italy
Fondazione Policlinico A. Gemelli- IRCCS
🇮🇹Rome, Italy
Hospital Universitario Donostia
🇪🇸Donostia, Spain
St. George's University Hospital
🇬🇧London, United Kingdom