rct
    2025
    High Quality

    Effect of Triheptanoin on Caudate Atrophy and Motor Scores in Patients With Early-Stage Huntington Disease: A Phase II Study

    Mochel F, Meneret A, Adanyeguh IM

    Published in Neurology

    Abstract

    Phase II randomized study of triheptanoin for early Huntington disease.

    Methodology

    Double-blind placebo-controlled six-month trial; a later exploratory comparison used an external placebo cohort.

    Key Findings

    The randomized primary endpoint showed no slowing of caudate atrophy over six months; later external-control findings were exploratory.

    Conclusions

    Triheptanoin did not slow caudate atrophy versus placebo over six months.

    Limitations

    Primary endpoint was negative; 12-month signal was post hoc with a nonrandomized external comparator; gastrointestinal withdrawals occurred.

    Related Health Concerns

    Huntington's Disease

    Study Details

    Year:2025
    Sample Size:100
    Duration:24 weeks
    Quality Score:7/10

    Access Study

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