A phase 1 trial design exploring cell replacement therapy in people with Huntington’s disease

Takeaway

  • This phase 1 trial is designed to assess the safety and feasibility of cell replacement therapy (CRT) for people with Huntington’s disease (HD), whilst also exploring methodological issues for informing future trial design.

Why this matters

    There are no therapeutics to cure HD; however, CRT may halt disease progression as it could potentially re-establish some degree of normal neural circuitry.