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DYNE-201 ACHIEVE

  • Status
    Accepting Candidates
  • Age
    18 Years - 65 Years
  • Sexes
    All
  • Healthy Volunteers
    No
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Objective

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.

Details

Full study title A Randomized, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-101 Administered to Participants with Myotonic Dystrophy Type 1. Short title: DYNE101-DM1-201 ACHIEVE.
Protocol number OCR49449
ClinicalTrials.gov ID NCT05481879
Phase Phase 1/Phase 2

Eligibility

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)

Lead researcher

  • Sub Subramony, MD
    Neuromuscular Medicine Specialist
    Languages: Hindi, Malayalam, Tamil

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  1. Step
    1

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  2. Step
    2

    Get screened to confirm eligibility

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  3. Step
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