Study of ATX-01 in Participants with DM1

Purpose

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.

Condition

  • Myotonic Dystrophy 1

Eligibility

Eligible Ages
Between 18 Years and 64 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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

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

Study Design

Phase
Phase 1/Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Triple (Participant, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
ATX-01 		ATX-01 is a formulation of the anti-microRNA 23b (anti-miR-23b), known as X82108, a novel type of antisense oligonucleotide
  • Drug: ATX-01
    Solution for infusion
Placebo Comparator
Placebo 		Placebo to ATX-01
  • Drug: Placebo
    Solution for infusion

Recruiting Locations

University of Kansas Medical Center, Department of Neurology
Fairway, Kansas 66205
Contact:
Cassidy Nelson
913-588-3653
cnelson15@kumc.edu

More Details

Status
Recruiting
Sponsor
ARTHEx Biotech S.L.

Study Contact

Project Manager
+34676229821
clinical@arthexbiotech.com