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. 2024;13(1):1-14.
doi: 10.3233/JHD-240017.

Huntington's Disease Clinical Trials Corner: March 2024

Carlos Estevez-Fraga  1 , Sarah J Tabrizi  1 , Edward J Wild  1
Affiliations

Huntington's Disease Clinical Trials Corner: March 2024

Carlos Estevez-Fraga et al. J Huntingtons Dis. 2024.

Abstract

In this edition of the Huntington's Disease Clinical Trials Update, we expand on the ongoing program from VICO Therapeutics and on the recently terminated VIBRANT-HD clinical trials. We also discuss updates from uniQure's AMT-130 program and PTC therapeutics' trial of PTC518 and list all currently registered and ongoing clinical trials in Huntington's disease.

Trial registration: ClinicalTrials.gov NCT05822908 NCT05111249 NCT04120493 NCT05243017 NCT05358717.

Keywords: Huntington disease; clinical trials.

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Conflict of interest statement

CEF was an investigator in the LEGATO-HD (NCT02215616), IONIS HTTRx OLE (NCT0-3342053), GENERATION-HD1 (NCT03761849), Roche Natural History Study (NCT03664804), Roche GEN-EXTEND (NCT03842969), Roche GEN-PEAK (NCT04000594), uniQure AMT-130 (NCT05243017), Triplet Therapeutics SHIELD-HD (NCT04406636), VIBRANT-HD (NCT05111249), PIVOT HD (NCT05358717) trials.

SJT has undertaken consultancy services for Annexon, Alphasights, Alnylam Pharmaceuticals Inc., Atalanta Pharmaceuticals (SAB), F. Hoffmann-La Roche Ltd/Genentech, Guidepoint, Horama, Locanobio, LoQus23 Therapeutics Ltd (SAB), Novartis Pharma, PTC Therapeutics, Sanofi, Spark Therapeutics, Takeda Pharmaceuticals Ltd, Triplet Therapeutics (SAB), University College Irvine and Vertex Pharmaceuticals Incorporated. All honoraria for these consultancies were paid through the offices of UCL Consultants Ltd., a wholly owned subsidiary of University College London. SJT has a patent Application number 2105484.6 on the FAN1-MLH1 interaction and structural analogues licensed to Adrestia Therapeutics. SJT was an investigator on IONIS HTTRx (NCT02519036), IONIS HTTRx OLE (NCT03342053), GENERATION-HD1 (NCT03761849), Roche Natural History Study (NCT03664804), uniQure AMT-130 (NCT05-243017), SHIELD-HD (NCT04406636), PIVOT HD (NCT05358717) and Roche GEN-EXTEND (NCT03842969) trials.

EJW has undertaken consultancy/advisory board work with Hoffman La Roche Ltd, Triplet Therapeutics, Takeda, Vico Therapeutics, Voyager, Huntington Study Group, Teitur Trophics, EcoR1 Capital, PTC Therapeutics, Alnylam, Annexon Biosciences and Remix Therapeutics. He has participated in advisory boards for Hoffmann La Roche, Triplet therapeutics and PTC therapeutics. All honoraria for these consultancies were paid through the offices of UCL Consultants Ltd., a wholly owned subsidiary of University College London. He holds a stock option for Triplet Therapeutics in part compensation for advisory board membership. EJW was an investigator in the Amaryllis (NCT02197130), LEGATO-HD (NCT02215616), IONIS HTTRx (NCT02519036), IONIS HTTRx OLE (NCT03342053), GENERATION-HD1 (NCT03761849), Roche Natural History Study (NCT03664804), Roche GEN-EXTEND (NCT03842969), VIBRANT-HD (NCT05111249), PIVOT HD (NCT05358717), Roche GEN-PEAK trial (NCT04000594) and uniQure AMT-130 (NCT05243017).

The authors did not make use of confidential or privileged information: all materials included in this manuscript were collected from publicly available sources.

References

    1. Vico Therapeutics B. V. A Safety and Pharmacokinetics Trial of VO659 in SCA1, SCA3 and HD. 2023.https://clinicaltrials.gov/study/NCT05822908.
    1. Novartis Pharmaceuticals. A Dose Range Finding Study With Open-Label Extension to Evaluate the Safety of Oral LMI070/Branaplam in Early Manifest Huntington’s Disease (VIBRANT-HD). 2021.https://clinicaltrials.gov/ct2/show/NCT05111249.
    1. UniQure Biopharma B.V. Safety and Proof-of-Concept (POC) Study With AMT-130 in Adults With Early Manifest Huntington Disease. 2019.https://clinicaltrials.gov/study/NCT04120493.
    1. UniQure Biopharma B.V. Safety and Efficacy of AMT-130 in European Adults With Early Manifest Huntington’s Disease. 2022.https://clinicaltrials.gov/ct2/show/NCT05243017.
    1. PTC Therapeutics. A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington’s Disease (HD). 2022.https://clinicaltrials.gov/ct2/show/NCT05358717.

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