Clinical trial NCT06300307: Study of ATX-01 in Participants With DM1
Official title: A Phase 1/2a Double-Blind, Placebo-controlled, Single- and Multiple Ascending Dose Study to Assess the Safety, Tolerability, PK, PD and Efficacy of IV Administration of ATX-01 In Male and Female Participants Aged 18 to 64 With Classic DM1
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: Myotonic Dystrophy 1
Lead sponsor: ARTHEx Biotech S.L.
Sponsor class: INDUSTRY
First posted: 2024-03-08
Enrollment: 56
Sex: ALL
Minimum age (years): 18
Maximum age (years): 64
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


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 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

Primary outcomes:
measure: Incidence of adverse events
timeFrame: Up to 120 days
Secondary outcomes:
measure: Incidence of clinically significant changes in laboratory assessments, electrocardiograms (ECGs), vital signs, suicidal ideation and behavior
timeFrame: Up to 120 days
measure: Maximum observed plasma concentration (Cmax) of ATX-01
timeFrame: Up to 48 hours post-dose
measure: Area under the plasma concentration-time curve (AUC) of ATX-01
timeFrame: Up to 48 hours post-dose
measure: Video hand opening time
timeFrame: Change from baseline up to 120 days
measure: Change from baseline in ankle dorsiflexion strength by quantitative myometry
timeFrame: Change from baseline up to 120 days
measure: Change from baseline in Impact on Activities of Daily Living questionnaire item scores
timeFrame: Change from baseline up to 120 days
Other outcomes:
Locations:
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Fairway
country: United States
facility: University of Kansas Medical Center, Department of Neurology
state: Kansas
city: Richmond
country: United States
facility: Virginia Commonwealth University
state: Virginia
city: Chicoutimi
country: Canada
facility: Centre Intégré Universitaire de Santé et Services Sociaux du Saguenay-Lac-St-Jean
state: Quebec
city: Paris
country: France
facility: Institute of Myology
city: Milan
country: Italy
facility: The NeMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan
city: Rome
country: Italy
facility: Fondazione Policlinico A. Gemelli- IRCCS
city: Donostia
country: Spain
facility: Hospital Universitario Donostia
city: London
country: United Kingdom
facility: St. George's University Hospital
Study officials:
References: