Related Experiment Video
Updated: Jul 10, 2025
Efficient and Scalable Production of Full-length Human Huntingtin Variants in Mammalian Cells using a Transient Expression System
Published on: December 10, 2021
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.
Insights
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.
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.
Abstract:
Huntington's Disease (HD) is a severely debilitating neurodegenerative disorder in which sufferers exhibit different combinations of movement disorders, dementia, and behavioral or psychiatric abnormalities. The disorder is a result of a trinucleotide repeat expansion mutation that is inherited in an autosomal dominant manner. While there is currently no treatment to alter the course of HD, there are medications that lessen abnormal movement and psychiatric symptoms. ClinicalTrials.gov was searched to identify drugs that are currently in or have completed phase III drug trials for the treatment of HD. The described phase III trials were further limited to interventional studies that were recruiting, active not recruiting, or completed. In addition, all studies must have posted an update within the past year. PubMed was used to gather further information on these interventional studies. Of the nine clinical trials that met these criteria, eight involved the following drugs: metformin, dextromethorphan/quinidine, deutetrabenazine, valbenazine, Cellavita HD, pridopidine, SAGE-718, and RO7234292 (RG6042). Of these drug treatments, four are already FDA approved. This systematic review provides a resource that summarizes the present therapies for treating this devastating condition that are currently in phase III clinical trials in the United States.
Related Concept Videos
01:28Preclinical Development: Overview
01:11Clinical Trials: Overview
01:26Drug Discovery: Overview
01:26Drug Administration and Therapy Phases: Overview
The pharmaceutical phase focuses on leveraging the physicochemical properties of the drug to design and manufacture an effective product. Variants include orally administered tablets or capsules, topical creams or ointments, and parenteral-delivery solutions or emulsions.
The pharmacokinetic phase...