โšกYour ready-to-use recruitment solution: IRB-compliant, SEO-optimized website that automatically routes qualified patient leads to your clinical study (14-day free trial, then $44/mo)

Study of ATX-01 in Participants With DM1

(ArthemiR)

Sponsored by ARTHEx Biotech S.L.

Quick Self-Assessment

See if you qualify and express your interest

Preparing your pre-screening questions...

Current Status

Recruiting

Auto-verified from ClinicalTrials.gov

Verified Feb 10, 2026

Phase

Clinical Trial

Sponsor

ARTHEx Biotech S.L.

Enrollment Target

56

Start Date

Oct 2024

๐Ÿ“Š
1 of 56 participants interested
2% interest

Express Your Interest Today

Interested in learning more? Complete the form below.

Secure & Confidential

Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Advance Medical Science

    Contribute to groundbreaking research

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel (varies by study)

  • Help Others

    Benefit future patients

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3In-person assessment if eligible
  4. 4Begin participation

About This Study

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.

Who Can Participate

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

โ“ Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility during the screening process.

Study Details

๐Ÿ“‹

Study Type

INTERVENTIONAL

๐Ÿ‘ฅ

Target Enrollment

56 participants

๐ŸŽ‚

Age Range

18 Years - 64 Years

โšง

Gender

ALL

๐Ÿข

Sponsor

ARTHEx Biotech S.L.

Study Locations

Choose your preferred location or select flexible during enrollment

RECRUITING

Los Angeles

California

Location available
View Los Angeles location page
RECRUITING

Gainesville

Florida

Location available
View Gainesville location page
RECRUITING

Iowa City

Iowa

Location available
View Iowa City location page
RECRUITING

Fairway

Kansas

Location available
View Fairway location page
RECRUITING

Richmond

Virginia

Location available
View Richmond location page

Frequently Asked Questions

Q:What is a clinical trial?

A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.

Q:Will I be compensated?

Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.

Q:Will I receive a placebo instead of treatment?

When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.

Q:Can I leave a trial if I change my mind?

Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.

Q:How long does a clinical trial last?

Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.

Still have questions? Our study coordinators are here to help.

Ready to Make a Difference?

Express your interest in this groundbreaking clinical trial

๐Ÿ”’ Secure ยท ๐Ÿฅ Expert Care

Find More Pulmonary Hypertension Trials by City

Browse all pulmonary hypertension clinical trials in these cities โ€” not just this study.

Looking for Myotonic Dystrophy Treatment in Gainesville?

Join others in Florida exploring innovative treatment options through clinical research

Myotonic Dystrophy Treatment Options in Gainesville, Florida

If you're searching for Myotonic Dystrophy treatment in Gainesville, participating in a clinical research study may provide access to innovative approaches under expert medical supervision. This study is actively recruiting participants in Gainesville, Fairway, Richmond and surrounding areas.

Clinical trials offer participants the opportunity to receive cutting-edge treatments while contributing to medical research that may help future patients with Myotonic Dystrophy. All study-related care is provided at no cost to participants.

Local Sites
3 locations in Florida
Now Enrolling
Up to 56 participants
Quick Start
Screening available now

Why Consider a Clinical Trial for Myotonic Dystrophy?

Potential Benefits

  • โœ“Access to new treatment approaches before public availability
  • โœ“Close monitoring by experienced medical professionals
  • โœ“Study-related care provided at no cost
  • โœ“Contribute to medical research for Myotonic Dystrophy

What to Expect

  • โ†’Initial screening to determine eligibility
  • โ†’Regular check-ups and monitoring visits
  • โ†’Possible compensation for time and travel
  • โ†’You can withdraw at any time

Frequently Asked Questions About This Myotonic Dystrophy Study

Important Clinical Trial Information

This information is provided for educational purposes and does not constitute medical advice. Clinical trial participation involves potential risks and benefits. Eligibility requirements apply and will be assessed during the screening process.

Study identifier: NCT06300307. For complete study details, visit ClinicalTrials.gov. Always consult with your healthcare provider before making decisions about your medical care or participating in clinical research.