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HARBOR

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

A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy Type 1

Description

The study consists of a Screening Period of up to 6 weeks and 54-week Treatment Period. The anticipated duration is approximately 60 weeks.

Participants will be randomized to receive an intravenous infusion of either del-desiran or placebo at the clinical study site every 8 weeks for a total of 7 doses. The final dose will occur at Week 48, followed by a final assessment at Week 54.

After completion of Week 54 assessments, eligible participants will have the option to enroll into an open label extension (OLE) study, pending regulatory approval.

An Independent Data Monitoring Committee (IDMC) comprised of members independent and external to the Sponsor will review safety, tolerability, and efficacy (as needed) data of this study at regular intervals.

Details

Full study title A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1
Protocol number OCR46020
ClinicalTrials.gov ID NCT06411288
Phase Phase 3

Eligibility

Key Inclusion Criteria:

  • Clinical and genetic diagnosis (CTG repeat 鈮� 100) of DM1

  • Ability to walk independently (orthoses and ankle braces allowed) for at least 10 meters at screening

Key Exclusion Criteria:

  • Breastfeeding, pregnancy, or intent to become pregnant during the study

  • Unwilling or unable to comply with contraceptive requirements

  • Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study

  • Diabetes that is not adequately controlled

  • History of decompensated heart failure within 3 months of screening. Participants with preexisting pacemaker/ICD are not excluded.

  • Body Mass Index > 35 kg/m2 at Screening

  • Recently treated with an investigational drug or biological agent

  • Treatment with anti-myotonic medication within 5 half-lives or 14 days of baseline, whichever is longer, prior to baseline.

Note: Additional protocol defined Inclusion and Exclusion criteria apply

Lead researcher

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

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  1. Step
    1

    Contact the research team

    Call or email the research team listed within the specific clinical trial or study to let them know that you're interested. A member of the research team, such as the researcher or study coordinator, will be available to tell you more about the study and to answer any questions or concerns you may have.

    Primary contact

    Sankarasubramoney Subramony
  2. Step
    2

    Get screened to confirm eligibility

    You may be asked to take part in prescreening to make sure you are eligible for a study. The prescreening process ensures it is safe for you to participate. During the prescreening process, you will be asked some questions and you may also be asked to schedule tests or procedures to confirm your eligibility.

  3. Step
    3

    Provide your consent to participate

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  4. Step
    4

    Participate

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