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Study Evaluating the Safety,Tolerability and Efficacy of PF-04360365 in Adults With Probable Cerebral Amyloid Angiopathy

This study has been completed.
Information provided by (Responsible Party):
Pfizer Identifier:
First received: March 4, 2013
Last updated: May 6, 2016
Last verified: May 2016
Cerebral Amyloid Angiopathy (CAA) is a condition caused by the build-up of a protein called amyloid, predominantly Aβ40, within the walls of brain blood vessels, especially those blood vessels in the occipital lobe of the brain. Probable CAA may be defined as two or more hemorrhages in the brain cortex in individuals 55 years of age or older. This study will examine the study drug (PF-04360365) vs. placebo (saline) at 10 mg/kg - Day 1 and the maintenance dose of the study drug (PF-04360365) vs. placebo (saline) at 7.5mg/kg on Days 30 and 60. Subjects will be followed for 6 months after receiving the last dose of study medication.

Condition Intervention Phase
Cerebral Amyloid Angiopathy
Biological: Ponezumab
Other: placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 2, Randomized, Double Blind Placebo Controlled Trial To Evaluate The Safety, Tolerability, Pharmacokinetics And Efficacy Of Pf-04360365 (Ponezumab) In Adult Subjects With Probable Cerebral Amyloid Angiopathy

Resource links provided by NLM:

Further study details as provided by Pfizer:

Primary Outcome Measures:
  • Change from baseline to Day 2 or day 90 in cerebrovascular reactivity. [ Time Frame: screening +90 days ] [ Designated as safety issue: No ]
    Change from baseline to Day 2 or day 90 in cerebrovascular reactivity as measured by the slope (amplitude over time to peak) from visual task-evoked fMRI.

Secondary Outcome Measures:
  • Change from baseline to Day 2 or Day 90 in cerebrovascular reactivity. [ Time Frame: screening +90 days ] [ Designated as safety issue: No ]
    Change from baseline to Day 2 or Day 90 in cerebrovascular reactivity as measured by the time to peak, magnitude, and time to bseline from visual task-evoked fMRI.

  • Change from baseline of total plasma AB species [ Time Frame: screening +240 days ] [ Designated as safety issue: No ]
    Change from baseline of total plasma AB species at specified endpoints after the intial dose of study medication on the concentration

Enrollment: 36
Study Start Date: June 2013
Study Completion Date: September 2015
Primary Completion Date: September 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1 Biological: Ponezumab
Infusion of Ponezumab (Day 1=10mg/kg; Day 30 and Day 60 dose = 7.5mg/kg) or placebo (saline); administered via infusion for a total infusion time of 20 minutes.
Placebo Comparator: 2 Other: placebo
placebo (saline)- given via infusion total infusion time of 20 minutes


Ages Eligible for Study:   55 Years to 90 Years   (Adult, Senior)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Patients diagnosed with probable CAA using the Boston criteria; with no clinical cognitive impairment
  • In general good health

Exclusion Criteria:

  • Co-morbid diagnosis of clinically documented Alzheimer's disease or significant cognitive impairment
  • Clinically significant syncope, epilepsy, head trauma or clinically significant unexplained loss of consciousness within the last 5 years
  • Subject's body weight exceeding 100kg
  • Women of childbearing potential.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01821118

United States, California
UCLA Ronald Reagan Medical Center
Los Angeles, California, United States, 90095
United States, Massachusetts
MGH Stroke Research Center
Boston, Massachusetts, United States, 02114
Boston Medical Center - Menino Pavilion
Boston, Massachusetts, United States, 02118
Boston Medical Center - Shapiro Center
Boston, Massachusetts, United States, 02118
Anthinoula A. Martinos Center for Biomedical Imaging
Charlestown, Massachusetts, United States, 02129
United States, Missouri
Barnes Jewish Hospital
St. Louis, Missouri, United States, 63110
United States, New York
Columbia University
New York, New York, United States, 10032
Canada, Alberta
University of Calgary/Foothills Medical Centre
Calgary, Alberta, Canada, T2N 2T9
Canada, Ontario
Sunnybrook Health Sciences Centre
Toronto, Ontario, Canada, M4N 3M5
CHRU de Lille - Hôpital Roger Salengro
Lille Cedex, NAP, France, 59037
CHRU de Lille
Lille Cedex, NAP, France, 59037
University Medical Center Utrecht
Utrecht, Netherlands, 3584 CX
United Kingdom
University College of London
London, United Kingdom, WC1N 3BG
Sponsors and Collaborators
Study Director: Pfizer Call Center Pfizer
  More Information

Additional Information:
Responsible Party: Pfizer Identifier: NCT01821118     History of Changes
Other Study ID Numbers: A9951024  2013-001557-27 
Study First Received: March 4, 2013
Last Updated: May 6, 2016
Health Authority: United States: Food and Drug Administration

Keywords provided by Pfizer:
Cerebral amyloid angiopathy (CAA)
cerebrovascular reactivity
functional MRI
double blind

Additional relevant MeSH terms:
Cerebral Amyloid Angiopathy
Cerebral Amyloid Angiopathy, Familial
Proteostasis Deficiencies
Metabolic Diseases
Cerebral Arterial Diseases
Intracranial Arterial Diseases
Cerebrovascular Disorders
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Vascular Diseases
Cardiovascular Diseases
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Cerebral Small Vessel Diseases
Amyloidosis, Familial
Metabolism, Inborn Errors
Genetic Diseases, Inborn processed this record on October 28, 2016