A Study of the Safety, Tolerability and Effectiveness of Eleclazine on Shortening the QT Interval in Adults with Long QT-3 Syndrome

Overview

  • Study type

    Interventional
  • Study phase

    III
  • Study IDs

  • Describes the nature of a clinical study. Types include:

    • Observational study — observes people and measures outcomes without affecting results.
    • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
    • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.
  • During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.

  • Site IRB
    • Rochester, Minnesota: 14-007259
    NCT ID: NCT02300558
    Sponsor Protocol Number: GS-US-372-1234

About this study

The purpose of this study is to evaluate the safety, tolerability and effectiveness of eleclazine for shortening  the corrected QT interval in adults with type 3 long QT syndrome.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. If you need assistance understanding the eligibility criteria, please contact the study team.

See eligibility criteria

Inclusion Criteria:

  • Individuals with an established diagnosis of LQT3 (by genotype testing) made at least 6 months prior to screening
  • Mean (of triplicate) QTc interval ≥ 480 msec (or ≥ 460 msec, for individuals who are currently taking ranolazine or Class I antiarrhythmic drugs such as mexiletine) at 4 or more time points, determined by standard 12-lead ECG, at screening

Exclusion Criteria:

  • Known mutations associated with type 1 long QT syndrome (LQT1) or type 2 long QT syndrome (LQT2)
  • Known or suspected history of seizures or epilepsy
  • History of heart failure defined as New York Heart Association (NYHA) Class IV and/or known left ventricular ejection fraction ≤ 45%
  • Body mass index (BMI) ≥ 36 kg/m^2 at screening
  • Severe renal impairment at screening (defined as an estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73m^2, using the 4 Variable Modification of Diet in Renal Disease equation, as determined by the study center)
  • Abnormal liver function tests at screening, defined as alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 2 x upper limit of normal (ULN), or total bilirubin > 1.5 x ULN
  • An aborted cardiac arrest, implantable cardioverter-defibrillator (ICD) implantation, syncopal episode, or appropriate ICD therapy within 3 months prior to screening
  • Any other condition or circumstance that in the opinion of the investigator would preclude compliance with the study protocol

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

Mayo Clinic Location Status

Rochester, Minn.

Mayo Clinic principal investigator

Michael Ackerman, M.D., Ph.D.

Closed for enrollment

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CLS-20204971

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