Is NCT05004129 recruiting?

Yes, NCT05004129 is currently recruiting. This myotonic dystrophy study is enrolling participants at 10 locations.

What is the status of NCT05004129?

NCT05004129 is currently recruiting. This is a Clinical Trial trial for myotonic dystrophy sponsored by AMO Pharma Limited. Status verified May 28, 2025 via ClinicalTrials.gov.

Where is NCT05004129 enrolling participants?

NCT05004129 has 10 active sites including Little Rock, Arkansas; Los Angeles, California; Palo Alto, California and 7 more locations.

Who is eligible for NCT05004129?

This myotonic dystrophy trial is targeting 76 participants. Detailed eligibility criteria including age requirements and medical history are listed on this page.

Who can participate in this Myotonic Dystrophy study?

Eligibility varies by study, but generally includes adults diagnosed with Myotonic Dystrophy who meet specific medical criteria. Complete the interest form above to learn if you may qualify.

Is the study available in Little Rock, Arkansas?

Yes! This study has active research sites in Little Rock, Los Angeles, Palo Alto. When you express interest, you'll be connected with the site nearest to you.

What are the benefits of joining a clinical trial for Myotonic Dystrophy?

Participants often receive: access to new treatments before they're widely available, close monitoring by medical professionals, study-related care at no cost, and the opportunity to contribute to medical research.

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NCT05004129 RECRUITINGIRB Ready

Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy

(REACH CDM X)

Express Your Interest Today

Interested in learning more? Complete the form below.

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

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

About This Study

This is an open-label phase 2/3 study for individuals with Congenital Myotonic Dystrophy (Congenital DM1) who participated in the preceding AMO-02-MD-2-003 study or individuals with either Congenital or Childhood Onset DM1 who are treatment naïve.

Who Can Participate

Inclusion Criteria

Subjects who do not enter this study directly from completing the AMO-02-MD-2-003 study (i.e. subjects who did not complete AMO-02-MD-2-003, subjects who completed AMO-02-MD-2-003 but did not directly rollover or subjects who are re-entering AMO-02-MD-2-004), will not be considered eligible for the study without meeting all of the criteria below: 1. Subjects under study must be individuals with a diagnosis of Congenital or Childhood Onset DM1. 2. Diagnosis must be genetically confirmed 3. Subjects must be male or female aged ≥6 years to ≤45 years at Screening 4. Subjects must have a Clinical Global Impression - Severity (CGI-S) score of 3 or greater at Screening (V-1) 5. Written, voluntary informed consent must be obtained before any study related procedures are conducted. Where a parent or legally authorized representative (LAR) provides consent, there must also be assent from the subject (as required by local regulations) 6. Subject's caregiver must be willing a

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

Study Details

📋

Study Type

INTERVENTIONAL

👥

Target Enrollment

76 participants

🎂

Age Range

6 Years - 45 Years

⚧

Gender

ALL

🏢

Sponsor

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

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Looking for Myotonic Dystrophy Treatment in Little Rock?

Join others in Arkansas exploring innovative treatment options through clinical research

Myotonic Dystrophy Treatment Options in Little Rock, Arkansas

If you're searching for Myotonic Dystrophy treatment in Little Rock, participating in a clinical research study may provide access to innovative approaches under expert medical supervision. This study is actively recruiting participants in Little Rock, Los Angeles, Palo Alto 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 Arkansas

Now Enrolling

Up to 76 participants

Quick Start

Screening available now

Why Consider a Clinical Trial for Myotonic Dystrophy?

Potential Benefits

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

What to Expect

→Initial screening to determine eligibility
→Regular check-ups and monitoring visits
→Possible compensation for time and travel
→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: NCT05004129. 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.