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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, A. P., van Dyck, C. H., Tariot, P. N., Gilman, S., Arnold, D., Abushakra, S., Hernandez, C., Crans, G., Liang, E., Quinn, G., Bairu, M., Pastrak, A., Cedarbaum, J. M.


Neurology, Volume: 77, No.: 13, Pages.: 1253-1262

Year of Publication



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.;

Bibtex Citation

@article{Salloway_2011, doi = {10.1212/wnl.0b013e3182309fa5}, url = {}, year = 2011, month = {sep}, publisher = {Ovid Technologies (Wolters Kluwer Health)}, volume = {77}, number = {13}, pages = {1253--1262}, author = {S. Salloway and R. Sperling and R. Keren and A. P. Porsteinsson and C. H. van Dyck and P. N. Tariot and S. Gilman and D. Arnold and S. Abushakra and C. Hernandez and G. Crans and E. Liang and G. Quinn and M. Bairu and A. Pastrak and J. M. Cedarbaum}, title = {A phase 2 randomized trial of {ELND}005, scyllo-inositol, in mild to moderate Alzheimer disease}, journal = {Neurology} }


administration & dosage, administration oral, aged, aged, 80 and over, agent, alzheimer disease, amyloid, antiaggregation, apolipoprotein e4, blood, cerebrospinal fluid, double-blind method, drug therapy, female, genetics, humans, inositol, magnetic resonance imaging, male, mental status schedule, middle aged, oral, peptide fragments, pharmacokinetics, platelet aggregation inhibitors, time factors, treatment outcome

Countries of Study


Types of Dementia

Alzheimer’s Disease

Types of Study

Randomised Controlled Trial

Type of Outcomes

ADLs/IADLs, Behaviour

Type of Interventions

Pharmaceutical Interventions

Pharmaceutical Interventions