Health TrialsbyHealthPanel

Study of ATX-01 in Participants With DM1

ConditionMyotonic Dystrophy 1
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18-64
SponsorARTHEx Biotech S.L.

About this trial

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

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial design

Design model

Parallel

Treatments tested in this trial

  • ATX-01

    Drug

    Solution for infusion

  • Placebo

    Drug

    Solution for infusion

Treatment groups

56 Participants
are divided into 2 treatment groups
Group A: ATX-01Experimental treatment 1 intervention
Group B: PlaceboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Incidence of adverse events

To evaluate the safety and tolerability of ATX-01 in adult participants with DM1

Time frame
Up to 120 days

Secondary outcomes

1

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

Time frame
Up to 120 days
2

Maximum observed plasma concentration (Cmax) of ATX-01

Time frame
Up to 48 hours post-dose
3

Area under the plasma concentration-time curve (AUC) of ATX-01

Time frame
Up to 48 hours post-dose
4

Video hand opening time

To evaluate the efficacy of ATX-01 on myotonia in participants with DM1

Time frame
Change from baseline up to 120 days

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

ARTHEx Biotech S.L.

Lead sponsor

Source ClinicalTrials.gov
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