In this edition of the Huntington's Disease Clinical Trials Update, we expand on the ongoing extension study of PTC518 from PTC Therapeutics, including 12-month interim results from the parent study. We also discuss 24-month interim results from uniQure's AMT-130 program and 28-week follow-up results from Wave Life Sciences’ SELECT-HD clinical trial of WVE-003. Additionally, we provide a comprehensive listing of all currently registered and ongoing clinical trials in Huntington's disease.
PTC Therapeutics. An extension study to evaluate the long-term safety and efficacy of PTC518 in participants with Huntington’s disease, https://clinicaltrials.gov/study/NCT06254482 (2024, accessed 14 August 2024).
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PTC Therapeutics. A study to evaluate the safety and efficacy of PTC518 in participants with Huntington’s disease, https://clinicaltrials.gov/study/NCT05358717 (2024, accessed 14 August 2024).
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PTC Therapeutics. Interim PIVOT-HD results demonstrate evidence of favorable CNS biomarker and clinical effects at Month 12 in Huntington’s disease patients, https://ir.ptcbio.com/node/16811/pdf (2024, accessed 14 August 2024).
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UniQure Biopharma B.V. Safety and Proof-of-Concept (POC) study with AMT-130 in adults with early manifest Huntington’s disease.
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UniQure Biopharma B.V. Safety and efficacy of AMT-130 in European adults with early manifest Huntington’s disease.
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UniQure Biopharma B.V. uniQure announces positive interim data update demonstrating slowing of disease progression in Phase I/II Trials of AMT-130 for Huntington’s disease, https://uniqure.gcs-web.com/news-releases/news-release-details/uniqure-announces-positive-interim-data-update-demonstrating (2024, accessed 14 August 2024).
