A phase 3 trial of IV immunoglobulin for Alzheimer disease

Neurology. 2017 May 2;88(18):1768-1775. doi: 10.1212/WNL.0000000000003904. Epub 2017 Apr 5.

Abstract

Objective: We tested biweekly infusions of IV immunoglobulin (IVIg) as a possible treatment for mild to moderate Alzheimer disease (AD) dementia.

Methods: In a phase 3, double-blind, placebo-controlled trial, we randomly assigned 390 participants with mild to moderate AD to receive placebo (low-dose albumin) or IVIg (Gammagard Liquid; Baxalta, Bannockburn, IL) administered IV at doses of 0.2 or 0.4 g/kg every 2 weeks for 18 months. The primary cognitive outcome was change from baseline to 18 months on the 11-item cognitive subscale of the Alzheimer's Disease Assessment Scale; the primary functional outcome was 18-month change on the Alzheimer's Disease Cooperative Study-Activities of Daily Living Inventory. Safety and tolerability data, as well as serial MRIs and plasma samples, were collected throughout the study from all enrolled participants.

Results: No beneficial effects were observed in the dual primary outcome measures for the 2 IVIg doses tested. Significant decreases in plasma Aβ42 (but not Aβ40) levels were observed in IVIg-treated participants. Analysis of safety data showed no difference between IVIg and placebo in terms of the rate of occurrence of amyloid-related imaging abnormalities (brain edema or microhemorrhage). IVIg-treated participants had more systemic reactions (chills, rashes) but fewer respiratory infections than participants receiving placebo.

Conclusions: Participants with mild to moderate AD showed good tolerability of treatment with low-dose human IVIg for 18 months but did not show beneficial effects on cognition or function relative to participants who received placebo.

Clinicaltrialsgov identifier: NCT00818662.

Classification of evidence: This study provides Class II evidence that IVIg infusions performed every 2 weeks do not improve cognition or function at 18 months in patients with mild to moderate AD.

Publication types

  • Clinical Trial, Phase III
  • Multicenter Study
  • Randomized Controlled Trial

MeSH terms

  • Aged
  • Aged, 80 and over
  • Alzheimer Disease / diagnostic imaging
  • Alzheimer Disease / drug therapy*
  • Alzheimer Disease / genetics
  • Amyloid beta-Peptides / blood
  • Apolipoprotein E4 / genetics
  • Biomarkers / blood
  • Brain / diagnostic imaging
  • Brain / drug effects
  • Canada
  • Cognition / drug effects
  • Double-Blind Method
  • Female
  • Humans
  • Immunoglobulins, Intravenous / adverse effects
  • Immunoglobulins, Intravenous / therapeutic use*
  • Male
  • Middle Aged
  • Nootropic Agents / adverse effects
  • Nootropic Agents / therapeutic use*
  • Peptide Fragments / blood
  • Treatment Failure
  • United States

Substances

  • Amyloid beta-Peptides
  • Apolipoprotein E4
  • Biomarkers
  • Immunoglobulins, Intravenous
  • Nootropic Agents
  • Peptide Fragments
  • amyloid beta-protein (1-40)
  • amyloid beta-protein (1-42)

Associated data

  • ClinicalTrials.gov/NCT00818662