St Louis, MONCT05481879Now EnrollingIRB Ready

Myotonic Dystrophy Type 1 (DM1) Clinical Trial in St Louis, MO

Access cutting-edge myotonic dystrophy type 1 (dm1) treatment through this clinical trial at a research site in St Louis. Study-provided care at no cost to qualified participants.

Sponsored by Dyne Therapeutics

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Expert Care in St Louis

Access myotonic dystrophy type 1 (dm1) specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related myotonic dystrophy type 1 (dm1) treatment provided free

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Why Participate?

  • No-Cost Study Care

  • Local to St Louis

    Convenient for MO 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 St Louis site if eligible
  4. 4Begin participation

About This Myotonic Dystrophy Type 1 (DM1) Study in St Louis

The primary purpose of the study is to evaluate the safety and tolerability of multiple intravenous (IV) doses of DYNE-101 administered to participants with Myotonic Dystrophy Type 1 (DM1). The study consists of 4 periods: A Screening Period (up to 8 weeks), a Placebo-Controlled Period (24 weeks), a Treatment Period (24 weeks) and a Long-Term Extension (LTE) Period (168 weeks) in both multiple-ascending dose (MAD) and dose expansion cohorts.

Sponsor: Dyne Therapeutics

Who Can Participate

Inclusion Criteria

Diagnosis of DM1 with trinucleotide repeat size \>100.
Age of onset of DM1 muscle symptoms ≥12 years.
Clinically apparent myotonia equivalent to hand opening time of at least 2 seconds in the opinion of the Investigator.
Hand grip strength and ankle dorsiflexion strength.
Able to complete 10-MWRT, stair ascend/descend (MAD cohorts only), and 5×STS at screening without the use of assistive devices such as canes, walkers, or orthoses.

Exclusion Criteria

History of major surgical procedure within 12 weeks prior to the start of investigative product administration or an expectation of a major surgical procedure (eg, implantation of cardiac defibrillator) during the study.
History of anaphylaxis.
Medical condition other than DM1 that would significantly impact ambulation or participation in functional assessments.
Treatment with medications that can improve myotonia within a period of 5 half-lives of the medication prior to performing screening assessments.
Electrocardiogram (ECG) with the corrected QT interval by Fridericia's Formula (QTcF) ≥450 milliseconds (ms) in men and QTcF ≥460 ms in women, PR ≥240 ms, left bundle-branch block, or a conduction defect, which is clinically significant in the opinion of the Investigator.
Percent predicted forced vital capacity (FVC) \<50%.
History of tibialis anterior biopsy within 3 months of Day 1 or planning to undergo tibialis anterior biopsies during study period for reasons unrelated to the study.
Participant has a history of suicide attempt, suicidal behavior, or has any suicidal ideation within 6 months prior to Screening that meets criteria at a level of 4 or 5 of the Columbia Suicide Severity Rating Scale (C-SSRS) or who, in the opinion of the Investigator, is at significant risk to commit suicide.
Use of glucagon-like peptide 1 (GLP-1) agonist medications including semaglutide, dulaglutide, liraglutide, exenatide, or tirzepatide within a period of 5 half-lives of the medication prior to performing screening assessments.
Significant weight loss during study participation may impact weight-based dosing, performance on muscle function assessments, and pharmacodynamic (PD) biomarkers. Note: Other inclusion and exclusion criteria may apply.

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 St Louis?

Yes, this clinical trial (NCT05481879) has an active research site in St Louis, MO 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 Type 1 (DM1) Treatment Options in St Louis, MO

If you're searching for myotonic dystrophy type 1 (dm1) treatment options in St Louis, MO, this clinical trial (NCT05481879) 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 St Louis research site is actively enrolling participants for this clinical trial. You'll receive care from experienced myotonic dystrophy type 1 (dm1) 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 type 1 (dm1) clinical trials near you to find additional studies recruiting in your area.

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