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A phase 2 randomized trial of ELND005, scyllo-inositol, in mild to moderate Alzheimer disease.

AbstractOBJECTIVE:
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.
AuthorsS Salloway, R Sperling, R Keren, A P Porsteinsson, C H van Dyck, P N Tariot, S Gilman, D Arnold, S Abushakra, C Hernandez, G Crans, E Liang, G Quinn, M Bairu, A Pastrak, J M Cedarbaum, ELND005-AD201 Investigators
JournalNeurology (Neurology) Vol. 77 Issue 13 Pg. 1253-62 (Sep 27 2011) ISSN: 1526-632X [Electronic] United States
PMID21917766 (Publication Type: Clinical Trial, Phase II, Journal Article, Multicenter Study, Randomized Controlled Trial, Research Support, Non-U.S. Gov't)
Chemical References
  • Amyloid beta-Peptides
  • Apolipoprotein E4
  • Peptide Fragments
  • Platelet Aggregation Inhibitors
  • amyloid beta-protein (1-42)
  • scyllitol
  • Inositol
Topics
  • Administration, Oral
  • Aged
  • Aged, 80 and over
  • Alzheimer Disease (blood, cerebrospinal fluid, drug therapy, genetics)
  • Amyloid beta-Peptides (cerebrospinal fluid)
  • Apolipoprotein E4 (genetics)
  • Dose-Response Relationship, Drug
  • Double-Blind Method
  • Female
  • Follow-Up Studies
  • Humans
  • Inositol (administration & dosage, blood, pharmacokinetics)
  • Magnetic Resonance Imaging
  • Male
  • Mental Status Schedule
  • Middle Aged
  • Peptide Fragments (cerebrospinal fluid)
  • Platelet Aggregation Inhibitors (administration & dosage, blood, pharmacokinetics)
  • Time Factors
  • Treatment Outcome

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