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Huntington's Disease Drug Development: A Phase 3 Pipeline Analysis
Hannah J Van de Roovaart, Nguyen Nguyen, Timothy D Veenstra. Pharmaceuticals (Basel, Switzerland), 2023-10-24
Topic: Drug trials: efficacy and safety
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.
Cite this study APA, Vancouver, BibTeX · download .bib or .ris
APA 7th
Van de Roovaart, H. J., Nguyen, N., & Veenstra, T. D. (2023). Huntington's Disease Drug Development: A Phase 3 Pipeline Analysis. Pharmaceuticals (Basel, Switzerland). https://doi.org/10.3390/ph16111513
Vancouver
Van de Roovaart HJ, Nguyen N, Veenstra TD. Huntington's Disease Drug Development: A Phase 3 Pipeline Analysis. Pharmaceuticals (Basel, Switzerland). 2023. doi:10.3390/ph16111513. PMID: 38004378.
BibTeX
@article{vanderoovaart2023-38004378,
author = {Van de Roovaart, Hannah J and Nguyen, Nguyen and Veenstra, Timothy D},
title = {{Huntington's Disease Drug Development: A Phase 3 Pipeline Analysis}},
journal = {Pharmaceuticals (Basel, Switzerland)},
year = {2023},
doi = {10.3390/ph16111513},
pmid = {38004378},
url = {https://doi.org/10.3390/ph16111513}
}
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Key findings
- The disorder is a result of a trinucleotide repeat expansion mutation that is inherited in an autosomal dominant manner.
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Study details
- Study designsystematic review
- Evidence strengthHigh
How this study scored 70/100
Published in a peer-reviewed journal, and it is a systematic review of all the studies on the question. Scored zero for sample size, effect reported and studied in people because the record does not state it. We have no data on journal's citation record, times cited, citations per year, impact for its field and year, cited by studies here, still being cited, senior author's track record, institution's track record, multi-centre collaboration and free to read, so those were left out: it earned 39 of the 56 points we could judge it on, which is 70 out of 100.
| Component | Points | Why |
|---|---|---|
| Journal tier | 7 / 10 | Tier 3 — peer-reviewed journal (Pharmaceuticals (Basel, Switzerland)) |
| Journal's citation record | — / 4 | No journal record from OpenAlex — left out of this score |
| Study design | 12 / 12 | A systematic review of all the studies on the question |
| Strength of evidence | 5 / 5 | Rated “high” in our summary |
| Peer-review validation | 10 / 10 | Peer-reviewed. Published as an article in a named peer-reviewed journal. |
| How recent | 3 / 5 | 2023 — within 6 year(s) of the newest study here |
| Sample size | 0 / 4 | Participant count not stated — scores zero (not stated in the record — scored 0) |
| Effect reported | 0 / 3 | No effect size reported — scores zero (not stated in the record — scored 0) |
| Studied in people | 0 / 3 | Not stated whether this was studied in people — scores zero (not stated in the record — scored 0) |
| Randomised / blinded | 0 / 2 | A review of other studies — randomisation is not credited here |
| Times cited | — / 7 | No citation count (not found in OpenAlex) — left out of this score |
| Citations per year | — / 5 | No citation count or no year — left out of this score |
| Impact for its field and year | — / 6 | No field-weighted citation impact (OpenAlex has none yet) — left out of this score |
| Cited by studies here | — / 6 | Not yet checked against this library's citation graph — left out of this score |
| Still being cited | — / 4 | No yearly citation counts — left out of this score |
| Senior author's track record | — / 4 | Senior author's record not available — left out of this score |
| Institution's track record | — / 3 | No institution listed in OpenAlex — left out of this score |
| Multi-centre collaboration | — / 3 | Institutions not listed — left out of this score |
| Indexed in PubMed | 2 / 2 | Indexed in PubMed (PMID 38004378) |
| Free to read | — / 2 | Access not recorded — left out of this score |
| Points | 39 / 56 | Scored on 10 of 20 parts. We have no data for the rest, so they are left out: 39 of the 56 points we could judge it on is 70 out of 100. |
| Total | 70 / 100 | — |
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