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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
56
Solution for infusion
Solution for infusion
UCLA
Los Angeles, California, United States
RECRUITINGUniversity of Florida
Gainesville, Florida, United States
RECRUITINGUniversity of Iowa Health Care - Department of Neurology
Iowa City, Iowa, United States
RECRUITINGUniversity of Kansas Medical Center, Department of Neurology
Fairway, Kansas, United States
RECRUITINGVirginia Commonwealth University
Richmond, Virginia, United States
RECRUITINGCentre Intégré Universitaire de Santé et Services Sociaux du Saguenay-Lac-St-Jean
Chicoutimi, Quebec, Canada
RECRUITINGInstitute of Myology
Paris, France
RECRUITINGThe NeMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan
Milan, Italy
RECRUITINGFondazione Policlinico A. Gemelli- IRCCS
Rome, Italy
RECRUITINGRadboudumc
Nijmegen, Netherlands
RECRUITING...and 2 more locations
Incidence of adverse events
To evaluate the safety and tolerability of ATX-01 in adult participants with DM1
Time frame: Up to 120 days
Incidence of clinically significant changes in laboratory assessments, electrocardiograms (ECGs), vital signs, suicidal ideation and behavior
To further evaluate the safety and tolerability of ATX-01 in adult participants with DM1
Time frame: Up to 120 days
Maximum observed plasma concentration (Cmax) of ATX-01
Time frame: Up to 48 hours post-dose
Area under the plasma concentration-time curve (AUC) of ATX-01
Time frame: Up to 48 hours post-dose
Video hand opening time
To evaluate the efficacy of ATX-01 on myotonia in participants with DM1
Time frame: Change from baseline up to 120 days
Change from baseline in ankle dorsiflexion strength by quantitative myometry
To evaluate the effects of ATX-01 in participants with DM1 on ankle dorsiflexion strength
Time frame: Change from baseline up to 120 days
Change from baseline in Impact on Activities of Daily Living questionnaire item scores
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.
Time frame: Change from baseline up to 120 days
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