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A phase 2 randomized trial of ELND005, scyllo-inositol, in mild to moderate Alzheimer disease
Salloway S, Sperling R, Keren R, Porsteinsson AP, van Dyck CH, Tariot PN, Gilman S, Arnold D et al.. Neurology, 2011-09-14
Topic: Brain proteins, microglia and lab models
Objective This randomized, double-blind, placebo-controlled, dose-ranging phase 2 study explored safety, efficacy, and biomarker effects of ELND005 (an oral amyloid anti-aggregation agent) in mild to moderate Alzheimer disease (AD). Methods A total of 353 patients were randomized to ELND005 (250, 1,000, or 2,000 mg) or placebo twice daily for 78 weeks. Coprimary endpoints were the Neuropsychological Test Battery (NTB) and Alzheimer's Disease Cooperative Study-Activities of Daily Living (ADCS-ADL) scale. The primary analysis compared 250 mg (n =84) to placebo (n =82) after an imbalance of infections and deaths led to early discontinuation of the 2 higher dose groups. Results The 250 mg dose demonstrated acceptable safety. The primary efficacy analysis at 78 weeks revealed no significant differences between the treatment groups on the NTB or ADCS-ADL. Brain ventricular volume showed a small but significant increase in the overall 250 mg group (p =0.049). At the 250 mg dose, scyllo-inositol concentrations increased in CSF and brain and CSF Aβx-42 was decreased significantly compared to placebo (p =0.009). Conclusions Primary clinical efficacy outcomes were not significant. The safety and CSF biomarker results will guide selection of the optimal dose for future studies, which will target earlier stages of AD. Classification of evidence Due to the small sample sizes, this Class II trial provides insufficient evidence to support or refute a benefit of ELND005.
Key findings
- Results The 250 mg dose demonstrated acceptable safety.
- The primary efficacy analysis at 78 weeks revealed no significant differences between the treatment groups on the NTB or ADCS-ADL.
- Brain ventricular volume showed a small but significant increase in the overall 250 mg group (p =0.049).
- At the 250 mg dose, scyllo-inositol concentrations increased in CSF and brain and CSF Aβx-42 was decreased significantly compared to placebo (p =0.009).
- Conclusions Primary clinical efficacy outcomes were not significant.
Why it matters
In this phase 2 trial, the 250 mg dose of ELND005 showed no significant differences from placebo on the primary measures of cognition or daily living. The authors state that primary clinical efficacy outcomes were not significant, though safety and biomarker results will guide future studies.
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Study details
- Study designclinical trial
- Participants84
- Evidence strengthHigh
How this study scored 69/100
Published in a leading specialty journal, and it is a mid-stage phase 2 trial. Scored zero for effect reported because the record does not state it. We have no data on senior author's track record, so that was left out: it earned 66 of the 96 points we could judge it on, which is 69 out of 100.
| Component | Points | Why |
|---|---|---|
| Journal tier | 9 / 10 | Tier 2 — leading specialty journal (Neurology) |
| Journal's citation record | 4 / 4 | Neurology: journal h-index 525 (OpenAlex, 2026-10-29) |
| Study design | 8 / 12 | A mid-stage phase 2 trial (from PubMed type “Clinical Trial, Phase II”) |
| 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 | 1 / 5 | 2011 — within 20 year(s) of the newest study here |
| Sample size | 1 / 4 | 84 participants |
| Effect reported | 0 / 3 | No effect size reported — scores zero (not stated in the record — scored 0) |
| Studied in people | 3 / 3 | Studied in people (participant count stated) |
| Randomised / blinded | 2 / 2 | Randomised and blinded or placebo-controlled (as stated in the record) |
| Times cited | 5 / 7 | Cited 259 times (OpenAlex, 2026-10-10) |
| Citations per year | 3 / 5 | 15.2 citations a year over 17 year(s) since 2011 |
| Impact for its field and year | 4 / 6 | Cited 12.6× the average for papers of its field and year (OpenAlex FWCI) |
| Cited by studies here | 3 / 6 | Cited by 10 other studies in this library |
| Still being cited | 2 / 4 | Cited 29 times in 2024–2026 (2026 still in progress; OpenAlex) |
| Senior author's track record | — / 4 | Senior author's record not available — left out of this score |
| Institution's track record | 2 / 3 | Brigham and Women's Hospital: institution h-index 1,995 (OpenAlex) |
| Multi-centre collaboration | 2 / 3 | OpenAlex lists 7 institution(s) in 2 countries |
| Indexed in PubMed | 2 / 2 | Indexed in PubMed (PMID 21917766) |
| Free to read | 0 / 2 | Behind a paywall — scores zero |
| Points | 66 / 96 | Scored on 19 of 20 parts. We have no data for the rest, so they are left out: 66 of the 96 points we could judge it on is 69 out of 100. |
| Total | 69 / 100 | — |
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Read the original
- DOI: 10.1212/wnl.0b013e3182309fa5
- PubMed: 21917766
- Original source →
- Free full text: PubMed Central →
Full text: free to read — PubMed Central lists this copy as free to read: the link above (checked 10 Oct 2026).
Cite this study APA, Vancouver, BibTeX · download .bib or .ris
Built from the record alzhub holds for this paper, not looked up. Volume, issue and page numbers are not held in the record. The record's author list ends in “et al.”, so not every author is named. Check the citation against the original before you use it.
APA 7th
Salloway, S., Sperling, R., Keren, R., Porsteinsson, A. P., van Dyck, C. H., Tariot, P. N., et al. (2011). A phase 2 randomized trial of ELND005, scyllo-inositol, in mild to moderate Alzheimer disease. Neurology. https://doi.org/10.1212/wnl.0b013e3182309fa5
Vancouver
Salloway S, Sperling R, Keren R, Porsteinsson AP, van Dyck CH, Tariot PN, et al. A phase 2 randomized trial of ELND005, scyllo-inositol, in mild to moderate Alzheimer disease. Neurology. 2011. doi:10.1212/wnl.0b013e3182309fa5. PMID: 21917766.
BibTeX
@article{salloway2011-21917766,
author = {Salloway, S. and Sperling, R. and Keren, R. and Porsteinsson, A. P. and van Dyck, C. H. and Tariot, P. N. and Gilman, S. and Arnold, D. and others},
title = {{A phase 2 randomized trial of ELND005, scyllo-inositol, in mild to moderate Alzheimer disease}},
journal = {Neurology},
year = {2011},
doi = {10.1212/wnl.0b013e3182309fa5},
pmid = {21917766},
url = {https://doi.org/10.1212/wnl.0b013e3182309fa5}
}
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