A phase 2 study of tramiprosate for cerebral amyloid angiopathy

Steven M. Greenberg, Jonathan Rosand, Alexander T. Schneider, L. Creed Pettigrew, Samuel E. Gandy, Barry Rovner, Brian Fred Fitzsimmons, Eric E. Smith, M. Edip Gurol, Kristin Schwab, Julie Laurin, Denis Garceau

Research output: Contribution to journalArticlepeer-review

36 Scopus citations


BACKGROUND AND PURPOSE: No treatments have been identified to lower the risk of intracerebral hemorrhage due to cerebral amyloid angiopathy (CAA). A potential approach to prevention is the use of agents that interfere with the pathogenic cascade initiated by the β-amyloid peptide (Aβ). Tramiprosate (3-amino-1-propanesulfonic acid) is a candidate molecule shown in preclinical studies to reduce CAA in a transgenic mouse model. METHODS: We performed a 5-center phase 2 double-blinded trial to evaluate the safety, tolerability, and pharmacokinetics of tramiprosate in subjects with lobar intracerebral hemorrhage. Twenty-four subjects age ≥55 years with possible or probable CAA were randomized to receive 12 weeks of tramiprosate at 1 of 3 oral doses (50, 100, or 150 mg twice daily). Subjects were followed for clinical adverse effects, laboratory, vital sign, electrocardiogram, cognitive, or functional changes, appearance of new symptomatic or asymptomatic hemorrhages, and pharmacokinetic parameters. RESULTS: Enrolled subjects were younger (mean age 70.8±5.4, range 61 to 78) and had more advanced baseline disease (measured by number of previous hemorrhages) than consecutive subjects in a CAA natural history cohort. No concerning safety issues were encountered with treatment. Nausea and vomiting were the most common adverse events and were more frequent at high doses. Nine subjects had new symptomatic or asymptomatic hemorrhages during treatment; all occurred in subjects with advanced baseline disease, with no apparent effect of drug dosing assignment. CONCLUSIONS: These data suggest that tramiprosate can be given safely to subjects with suspected CAA and support future efficacy trials.

Original languageEnglish
Pages (from-to)269-274
Number of pages6
JournalAlzheimer Disease and Associated Disorders
Issue number4
StatePublished - Oct 2006


  • Cerebral amyloid angiopathy
  • Clinical drug trial
  • Glycosaminoglycans

ASJC Scopus subject areas

  • Clinical Psychology
  • Gerontology
  • Geriatrics and Gerontology
  • Psychiatry and Mental health


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