Myotonic Dystrophy 1
Conditions
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.
Interventions
DRUGATX-01
Solution for infusion
DRUGPlacebo
Solution for infusion
Sponsors
ARTHEx Biotech S.L.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 64 Years
Healthy volunteers
No
Inclusion 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 as canes, walkers, or orthoses, except for ankle-foot orthoses * Presence for \>3 seconds of grip myotonia as confirmed by a central reader Key
Exclusion criteria
* 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of adverse events | Up to 120 days | To evaluate the safety and tolerability of ATX-01 in adult participants with DM1 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 |
| 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. |
Countries
Canada, France, Italy, Netherlands, Spain, United Kingdom, United States
Contacts
CONTACTProject Manager
Outcome results
None listed