Study of ATX-01 in Participants With DM1

Part of paid clinical trials in Los Angeles, California.

Sponsor
ARTHEx Biotech S.L.
Study ID
NCT06300307
Phase
PHASE1/PHASE2
Status
Recruiting
Apply to this trial

Conditions

  • Myotonic Dystrophy 1

Eligibility Criteria

Sex
ALL
Age
18 Years - 64 Years
Healthy Volunteers
Not accepted

Interventions

  • ATX-01 — DRUG
    Solution for infusion
  • Placebo — DRUG
    Solution for infusion

Study Details

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.

Key Dates

Start date
Oct 15, 2024
Status verified
Feb 2026
Primary completion
Jul 31, 2027
Completion
Jul 31, 2027

Study Design

Enrollment
56 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • 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
  • Placebo Comparator: Placebo
    Placebo to ATX-01

Primary Outcome Measure

Incidence of adverse events [ Time Frame: Up to 120 days ]

Central Contacts

Locations (5)

FacilityCityStateZIPSite coordinators
UCLALos AngelesCalifornia90095
Michael Yan
[email protected]
310-825-3264
Perry Shieh, MD, PhD (PRINCIPAL_INVESTIGATOR)
University of FloridaGainesvilleFlorida32610
Brandy Donahue-Quails
[email protected]
University of Iowa Health Care - Department of NeurologyIowa CityIowa52242
Heena Olalde
[email protected]
319-356-8326
Andrea Swenson (PRINCIPAL_INVESTIGATOR)
University of Kansas Medical Center, Department of NeurologyFairwayKansas66205
Cassidy Nelson
[email protected]
913-588-3653
Virginia Commonwealth UniversityRichmondVirginia23298
Jodie Howell
[email protected]
804-828-6110
Nicholas Johnson, MD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Los Angeles, CA

By research site
UCLA· Los Angeles, CAUniversity of Florida· Gainesville, FLUniversity of Iowa Health Care - Department of Neurology· Iowa City, IAUniversity of Kansas Medical Center, Department of Neurology· Fairway, KSVirginia Commonwealth University· Richmond, VA

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