Pittsburgh, PANCT05004129Now EnrollingIRB Ready

Myotonic Dystrophy Clinical Trial in Pittsburgh, PA

Access cutting-edge myotonic dystrophy treatment through this clinical trial at a research site in Pittsburgh. Study-provided care at no cost to qualified participants.

Sponsored by AMO Pharma Limited

Quick Self-Assessment

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Expert Care in Pittsburgh

Access myotonic dystrophy specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related myotonic dystrophy treatment provided free

Apply for This Pittsburgh Location

Check if you qualify for this myotonic dystrophy clinical trial in Pittsburgh, PA

Secure & Confidential

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

Why Participate?

  • No-Cost Study Care

  • Local to Pittsburgh

    Convenient for PA residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Pittsburgh site if eligible
  4. 4Begin participation

About This Myotonic Dystrophy Study in Pittsburgh

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.

Sponsor: AMO Pharma Limited

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.

Frequently Asked Questions

Q:Is this study available in Pittsburgh?

Yes, this clinical trial (NCT05004129) has an active research site in Pittsburgh, PA that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

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

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

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

Myotonic Dystrophy Treatment Options in Pittsburgh, PA

If you're searching for myotonic dystrophy treatment options in Pittsburgh, PA, this clinical trial (NCT05004129) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Pittsburgh research site is actively enrolling participants for this clinical trial. You'll receive care from experienced myotonic dystrophy specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all myotonic dystrophy clinical trials near you to find additional studies recruiting in your area.

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Secure · Expert Care · Pittsburgh, PA