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Review
. 2025 May;14(2):191-206.
doi: 10.1177/18796397251337000. Epub 2025 Apr 30.

Huntington's disease clinical trials update: March 2025

Mena Farag  1 , Sarah J Tabrizi  1 , Edward J Wild  1
Affiliations
Review

Huntington's disease clinical trials update: March 2025

Mena Farag et al. J Huntingtons Dis. 2025 May.

Abstract

In this edition of the Huntington's Disease Clinical Trials Update, we expand on the ongoing phase I clinical trial of ALN-HTT02 from Alynlam Pharmaceuticals. We also report on the SAGE-718 (also known as dalzanemdor) program from Sage Therapeutics, with results of the phase II DIMENSION study and the recent termination of the open-label phase III PURVIEW study. Additionally, we discuss recent developments in the regulatory pathway for AMT-130, following discussions between uniQure and the U.S. Food and Drug Administration regarding key aspects of accelerated approval. Finally, we provide a comprehensive listing of all currently registered and ongoing clinical trials in Huntington's disease.

Keywords: Huntington's disease; clinical trials.

Plain language summary

In this edition we expand on the ongoing phase I clinical trial of ALN-HTT02 from Alynlam Pharmaceuticals. We also report on the SAGE-718 (also known as dalzanemdor) program from Sage Therapeutics, with results of the phase II DIMENSION study and the recent termination of the open-label phase III PURVIEW study. Additionally, we discuss recent developments in the regulatory pathway for AMT-130, following discussions between uniQure and the U.S. Food and Drug Administration regarding key aspects of accelerated approval. Finally, we provide a comprehensive listing of all currently registered and ongoing clinical trials in Huntington's disease.

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References

    1. Rodrigues FB, Wild EJ. Clinical Trials Corner: September 2017. J Huntingtons Dis 2017; 6: 255–263. - PMC - PubMed
    1. Rodrigues FB, Wild EJ. Huntington’s Disease Clinical Trials Corner: February 2018. J Huntingtons Dis 2018; 7: 89–98. - PMC - PubMed
    1. Rodrigues FB, Wild EJ. Huntington’s Disease Clinical Trials Corner: August 2018. J Huntingtons Dis 2018; 7: 279–286. - PMC - PubMed
    1. Rodrigues FB, Quinn L, Wild EJ. Huntington’s Disease Clinical Trials Corner: January 2019. J Huntingtons Dis 2019; 8: 115–125. - PubMed
    1. Rodrigues FB, Ferreira JJ, Wild EJ. Huntington’s Disease Clinical Trials Corner: June 2019. J Huntingtons Dis 2019; 8: 363–371. - PMC - PubMed

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