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73/100

✓ Peer-reviewed alzhub usefulness score 73 out of 100. Treatments

Topic: Brain proteins, microglia and lab models

Results A total of 68 participants (38 men [56%]; median age, 65 [range, 50-85] years) were included in the modified intention-to-treat analysis, of whom 26 had AD (14 women [54%]; median age, 63 [range, 50-76] years), and 42 had 4RT (16 women [38%]; median age, 69 [range, 54-83] years). Despite undetectable TPI-287 levels in CSF, CSF biomarkers demonstrated decreased chitinase-3-like protein-1 (YKL-40) levels in the 4RT treatment arm (mean [SD], -8.4 [26.0] ng/mL) compared with placebo (mean [SD], 10.4 [42.3] ng/mL; median difference, -14.6 [95% CI, -30.0 to 0.2] ng/mL; P = .048, Mann-Whitney test). The ability to reveal different tau therapeutic effects in various tauopathy syndromes suggests that basket trials are a valuable approach to tau therapeutic early clinical development.

Cite this study APA, Vancouver, BibTeX · download .bib or .ris

APA 7th

Tsai, R. M., Miller, Z., Koestler, M., Rojas, J. C., Ljubenkov, P. A., Rosen, H. J., et al. (2020). Reactions to Multiple Ascending Doses of the Microtubule Stabilizer TPI-287 in Patients With Alzheimer Disease, Progressive Supranuclear Palsy, and Corticobasal Syndrome: A Randomized Clinical Trial. JAMA neurology. https://doi.org/10.1001/jamaneurol.2019.3812

Vancouver

Tsai RM, Miller Z, Koestler M, Rojas JC, Ljubenkov PA, Rosen HJ, et al. Reactions to Multiple Ascending Doses of the Microtubule Stabilizer TPI-287 in Patients With Alzheimer Disease, Progressive Supranuclear Palsy, and Corticobasal Syndrome: A Randomized Clinical Trial. JAMA neurology. 2020. doi:10.1001/jamaneurol.2019.3812. PMID: 31710340.

BibTeX

@article{tsai2020-31710340,
  author  = {Tsai, R. M. and Miller, Z. and Koestler, M. and Rojas, J. C. and Ljubenkov, P. A. and Rosen, H. J. and Rabinovici, G. D. and Fagan, A. M. and others},
  title   = {{Reactions to Multiple Ascending Doses of the Microtubule Stabilizer TPI-287 in Patients With Alzheimer Disease, Progressive Supranuclear Palsy, and Corticobasal Syndrome: A Randomized Clinical Trial}},
  journal = {JAMA neurology},
  year    = {2020},
  doi     = {10.1001/jamaneurol.2019.3812},
  pmid    = {31710340},
  url     = {https://doi.org/10.1001/jamaneurol.2019.3812}
}

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Key findings

Results A total of 68 participants (38 men [56%]; median age, 65 [range, 50-85] years) were included in the modified intention-to-treat analysis, of whom 26 had AD (14 women [54%]; median age, 63 [range, 50-76] years), and 42 had 4RT (16 women [38%]; median age, 69 [range, 54-83] years).
Despite undetectable TPI-287 levels in CSF, CSF biomarkers demonstrated decreased chitinase-3-like protein-1 (YKL-40) levels in the 4RT treatment arm (mean [SD], -8.4 [26.0] ng/mL) compared with placebo (mean [SD], 10.4 [42.3] ng/mL; median difference, -14.6 [95% CI, -30.0 to 0.2] ng/mL; P = .048, Mann-Whitney test).
The ability to reveal different tau therapeutic effects in various tauopathy syndromes suggests that basket trials are a valuable approach to tau therapeutic early clinical development.

Why it matters

In this trial of 68 participants with Alzheimer disease, progressive supranuclear palsy, or corticobasal syndrome, TPI-287 levels were undetectable in cerebrospinal fluid, and only the 4-repeat tauopathy group showed decreased YKL-40 levels compared with placebo. The authors conclude that basket trials are a useful approach for early tau therapeutic development, so this is an early-stage finding and not evidence of clinical benefit in Alzheimer disease.

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Study details

Study designclinical trial
Participants39
Effect sizeor 20.0
Evidence strengthHigh

How this study scored 73/100

Published in a leading specialty journal, and it is a randomized controlled trial.

Component	Points	Why
Journal tier	9 / 10	Tier 2 — leading specialty journal (JAMA neurology)
Journal's citation record	2 / 4	JAMA Neurology: journal h-index 235 (OpenAlex, 2026-10-29)
Study design	11 / 12	A randomized controlled trial (from PubMed type “Randomized Controlled Trial”)
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	2 / 5	2020 — within 10 year(s) of the newest study here
Sample size	1 / 4	39 participants
Effect reported	2 / 3	Effect reported as a number, without an interval or test
Studied in people	3 / 3	Studied in people (participant count stated)
Randomized / blinded	1 / 2	Randomized (as stated in the record)
Times cited	5 / 7	Cited 131 times (OpenAlex, 2026-10-10)
Citations per year	3 / 5	16.4 citations a year over 8 year(s) since 2020
Impact for its field and year	4 / 6	Cited 8.3× the average for papers of its field and year (OpenAlex FWCI)
Cited by studies here	2 / 6	Cited by 5 other studies in this library
Still being cited	3 / 4	Cited 51 times in 2024–2026 (2026 still in progress; OpenAlex)
Senior author's track record	3 / 4	Last-listed author Adam L. Boxer: h-index 98 (OpenAlex, whole career)
Institution's track record	3 / 3	Stanford University: institution h-index 2,684 (OpenAlex)
Multicenter collaboration	2 / 3	OpenAlex lists 5 institution(s) in 1 country
Indexed in PubMed	2 / 2	Indexed in PubMed (PMID 31710340)
Free to read	0 / 2	Behind a paywall — scores zero
Total	73 / 100	—

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