Study of ATX-01 in Participants With DM1

Myotonic Dystrophy 1 Clinical Trial

— ArthemiR  

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06300307
• Other study ID #: CT-ATX-01-DM1-1.1
• Status: Not yet recruiting
• Phase: Phase 1/Phase 2
• Start date: May 2024
• Est. completion date: December 2025

Study information

• Verified date: March 2024
• Source: ARTHEx Biotech S.L.
• Contact: Project Manager
• Phone: +34676229821
• Email: clinical[@]arthexbiotech.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 56
• Est. completion date: December 2025
• Est. primary completion date: December 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 64 Years

Eligibility

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 within 21 days or 5 half-lives, whichever is longer, prior to screening

Related Conditions & MeSH terms

• Myotonic Dystrophy
• Myotonic Dystrophy 1

Intervention

Drug:
• ATX-01
Solution for infusion
• Placebo
Solution for infusion

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
ARTHEx Biotech S.L.

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of adverse events	To evaluate the safety and tolerability of ATX-01 in adult participants with DM1	Up to 120 days
Secondary	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	Up to 120 days
Secondary	Maximum observed plasma concentration (Cmax) of ATX-01		Up to 48 hours post-dose
Secondary	Area under the plasma concentration-time curve (AUC) of ATX-01		Up to 48 hours post-dose
Secondary	Video hand opening time	To evaluate the efficacy of ATX-01 on myotonia in participants with DM1	Change from baseline up to 120 days
Secondary	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	Change from baseline up to 120 days
Secondary	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.	Change from baseline up to 120 days