Huntington’s Disease Drug Development: A Phase 3 Pipeline Analysis
Abstract
:1. Introduction
2. Results
2.1. Metformin
2.1.1. Overview of Evidence Supporting Use of Metformin in HD
2.1.2. Overview of Current Phase III Trial
2.2. Dextromethorphan/Quinidine
2.2.1. Overview of Evidence Supporting Use of Dextromethorphan/Quinidine in HD
2.2.2. Overview of Current Phase III Trial
2.3. Deutetrabenazine
2.3.1. Overview of Evidence Supporting Use of Deutetrabenazine in HD
2.3.2. Overview of Current Phase III Trial
2.4. Valbenazine
2.4.1. Overview of Evidence Supporting Use of Valbenazine in HD
2.4.2. Overview of Phase III Clinical Trials
2.5. Cellavita HD
2.5.1. Overview of Evidence Supporting Use of Cellavita HD in HD
2.5.2. Overview of Phase III Clinical Trial
2.6. Pridopidine
2.6.1. Overview of Evidence Supporting Use of Pridopidine in HD
2.6.2. Overview of Phase III Clinical Trial
2.7. SAGE-718
2.7.1. Overview of Evidence Supporting Use of SAGE-718 in HD
2.7.2. Overview of Phase III Clinical Trial
2.8. Tominersen (RO7234292 or RG6042)
2.8.1. Overview of Evidence Supporting Use of Tominersen for HD
2.8.2. Overview of Phase III Clinical Trial
3. Discussion
4. Conclusions
Author Contributions
Funding
Institutional Review Board Statement
Informed Consent Statement
Data Availability Statement
Acknowledgments
Conflicts of Interest
References
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Drug | Clinical Trial Name | Identifier | Primary Outcome |
---|---|---|---|
Metformin | Testing METformin against cognitive decline in HD | NCT04826692 | Evaluate drug’s effect on scores obtained in different cognitive subtests that make up the Unified Huntington’s Disease Rating Scale (UHDRS) |
Dextromethorphan/ quinidine | Evaluating the Efficacy of Dextromethorphan/Quinidine in Treating Irritability in HD | NCT03854019 | Measure patient’s irritability using The Irritability Scale |
Deutetrabenazine | Impact of Deutetrabenazine on Functional Speech and Gait Dynamics in Huntington Disease | NCT04713982 | Improvement on Sentence Intelligibility Test and Motor Speech Evaluation |
Valbenazine | Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Chorea Associated with Huntington Disease (KINECT-HD) | NCT04102579 | Improvement in chorea symptoms and evaluation of treatment-emergent adverse events |
An Open-Label Rollover Study for Continuing Valbenazine Administration for the Treatment of Chorea Associated With Huntington Disease | NCT04400331 | Evaluation of treatment-emergent adverse events (TEAEs) | |
Cellavita HD | Clinical Extension Study for Safety and Efficacy Evaluation of the Cellavita HD Product in Huntington’s Patients (ADORE-EXT) | NCT04219241 | Evaluate treatment’s effectiveness as verified by comparing the total UHDRS |
Pridopidine | Pridopidine’s Outcome on Function in Huntington Disease, PROOF-HD | NCT04556656 | Measure change from baseline in UHDRS-Total Functional Capacity score |
SAGE-718 | A Study to Evaluate the Safety and Tolerability of SAGE-718 in Participants With Huntington’s Disease | NCT05655520 | Measure number and severity of TEAEs Measure number of participants that withdraw due to adverse events Measure change from baseline in clinical laboratory and electrocardiogram parameters Measure change in baseline in C-SSRS responses |
RO7234292 (RG6042) | An Open-Label Extension Study to Evaluate Long-Term Safety and Tolerability of RO7234292 (RG6042) in Huntington’s Disease | NCT03842969 | Measure number and severity of TEAEs Measure number of participants with suicidal ideation/behavior and self-injurious behavior without suicidal intent based on C-SSRS scale Measure change from baseline in MoCA |
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Van de Roovaart, H.J.; Nguyen, N.; Veenstra, T.D. Huntington’s Disease Drug Development: A Phase 3 Pipeline Analysis. Pharmaceuticals 2023, 16, 1513. https://doi.org/10.3390/ph16111513
Van de Roovaart HJ, Nguyen N, Veenstra TD. Huntington’s Disease Drug Development: A Phase 3 Pipeline Analysis. Pharmaceuticals. 2023; 16(11):1513. https://doi.org/10.3390/ph16111513
Chicago/Turabian StyleVan de Roovaart, Hannah J., Nguyen Nguyen, and Timothy D. Veenstra. 2023. "Huntington’s Disease Drug Development: A Phase 3 Pipeline Analysis" Pharmaceuticals 16, no. 11: 1513. https://doi.org/10.3390/ph16111513