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Huntington's Disease Drug Development: A Phase 3 Pipeline Analysis.

Hannah J Van de Roovaart1, Nguyen Nguyen1, Timothy D Veenstra1

  • 1School of Pharmacy, Cedarville University, Cedarville, OH 45314, USA.

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Summary
This summary is machine-generated.

This review summarizes Huntington's Disease (HD) treatments in phase III trials, identifying eight key drugs, including four FDA-approved options, to manage symptoms of this neurodegenerative disorder.

Keywords:
Huntington’s diseaseclinical trialmetforminphase IIItreatmentvalbenazine

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Area of Science:

  • Neuroscience
  • Genetics
  • Pharmacology

Background:

  • Huntington's Disease (HD) is a severe neurodegenerative disorder characterized by movement, cognitive, and psychiatric symptoms.
  • HD is caused by an autosomal dominant trinucleotide repeat expansion mutation.
  • Currently, no cure exists for HD, but treatments can alleviate symptoms.

Purpose of the Study:

  • To identify and summarize drugs in phase III clinical trials for Huntington's Disease (HD).
  • To provide a resource on current interventional therapies for HD in the United States.

Main Methods:

  • Systematic review of ClinicalTrials.gov for phase III interventional HD trials.
  • Inclusion criteria: recruiting, active not recruiting, or completed studies with updates in the past year.
  • PubMed was used for supplementary information on identified trials.

Main Results:

  • Nine clinical trials met the search criteria.
  • Eight trials focused on specific drugs: metformin, dextromethorphan/quinidine, deutetrabenazine, valbenazine, Cellavita HD, pridopidine, SAGE-718, and RO7234292 (RG6042).
  • Four of the investigated drug treatments are already FDA-approved.

Conclusions:

  • This review consolidates information on promising HD therapies currently in late-stage clinical development.
  • Highlights the availability of FDA-approved treatments among those in phase III trials.
  • Offers a valuable resource for understanding the current landscape of Huntington's Disease therapeutic interventions.