Phase II Study of Florbetaben (BAY94-9172) PET Imaging for Detection/Exclusion of Cerebral β-amyloid.

Study Description

Go to 

Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Brief Summary:

To determine the sensitivity of the visual assessment of BAY94-9172 PET images in detecting cerebral β-amyloid in individuals with Down Syndrome (DS) and specificity in individuals without DS. Given that individuals with Down Syndrome develop β-amyloid pathology over the age of 40, the clinical diagnosis of Down Syndrome will serve as the standard of truth.

Condition or disease	Intervention/treatment	Phase
Down Syndrome; Amyloid Beta-protein	Drug: Florbetaben (BAY94-9172)	Phase 2

Study Design

Go to 

Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Study Type :	Interventional (Clinical Trial)
Actual Enrollment :	109 participants
Intervention Model:	Single Group Assignment
Masking:	None (Open Label)
Primary Purpose:	Diagnostic
Official Title:	An Open-Label, Non-Randomized Study to Evaluate the Efficacy and Safety of BAY94-9172 (ZK 6013443) Positron Emission Tomography (PET) for Detection of Cerebral ß-Amyloid in Individuals With Down Syndrome Compared to Individuals Without Down Syndrome
Study Start Date :	June 2009
Actual Primary Completion Date :	January 2011
Actual Study Completion Date :	January 2011

Arms and Interventions

Go to 

Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Arm	Intervention/treatment
Experimental: Florbetaben (BAY94-9172)	Drug: Florbetaben (BAY94-9172); 300 megabecquerels (MBq) as single IV injection of 2 to 10 mL

Outcome Measures

Go to 

Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Primary Outcome Measures :

1. Sensitivity of the Independent Visual Assessment of Detecting Cerebral Amyloid-beta in Individuals With Down Syndrome and Specificity in Subjects Without Down Syndrome [ Time Frame: 100-120 min ]
   The primary variables are sensitivity (percentage of DS subjects positive for cerebral beta-amyloid) and specificity (percentage of healthy volunteers negative for cerebral beta-amyloid) of brain uptake of florbetaben based on the majority read of the visual assessment by three independent blinded readers of PET images obtained 100 to 120 minutes post-injection of florbetaben.

Secondary Outcome Measures :

1. Sensitivity Results in the Down Syndrome Age Subgroups [ Time Frame: 100 - 120 min ]
   The majority read sensitivity (percentage of DS subjects positive for cerebral beta-amyloid by majority read) was computed for the group of DS subjects with age equal to or below the median age (DS-young) and for DS subjects with age above the median age (DS-old). The median age was 46 yrs, with 3 subjects being exactly 46 yrs old. As defined, these subjects were assigned to the DS-young group.
2. Quantitative Parameters Standard Uptake Value Ratio [ Time Frame: 100 - 120 min p.i. ]
   The Standard Uptake Value Ratios for florbetaben signal in the frontal cortex, posterior cingulate, lateral temporal cortex, parietal cortex, and cerebellum (white matter) were determined as a quantitative measure of tracer uptake. The SUV is defined as the ratio of (1) the tissue radioactivity concentration c (in MBq/kg) at time point t, and (2) the injected activity (in MBq, extrapolated to the same time t) divided by the body weight (in kg). These SUV numbers from regions of interest were then used to derive SUV ratios (SUVR) using the SUV from the cerebellar cortex as reference.
3. Consistency Between Visual and Quantitative Efficacy [ Time Frame: 100 - 120 min ]
   For a comparison of the results of the visual assessment with the quantitative assessment, descriptive Standardized Uptake Value Ratio (SUVR) statistics were computed separately for DS subjects with an abnormal/normal majority read of the PET scan (DS-PET+/DS-PET-). The SUV is defined as the ratio of (1) the tissue radioactivity concentration c (in MBq/kg) at time point t, and (2) the injected activity (in MBq, extrapolated to the same time t) divided by the body weight (in kg). These SUV numbers from regions of interest were then used to derive SUV ratios (SUVR) using the SUV from the cerebellar cortex as reference.

Eligibility Criteria

Go to 

Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Ages Eligible for Study:	21 Years and older (Adult, Senior)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	Yes

Criteria

Inclusion Criteria:

Study participants were individuals with DS and healthy volunteers (HVs).

• Main inclusion criteria for individuals without DS

  • >=21 and <= 40 years of age
  • Mini-Mental State Examination (MMSE) >= 28
  • Clinical Dementia Rating (CDR) of 0

• Main inclusion criteria for individuals with DS

  • >= 40 years of age

Exclusion Criteria:

• Main exclusion criteria for both groups

  • Unstable medical or psychiatric condition, study specific screening procedures with the understanding that the patient has the right to withdraw from the study at any time

Contacts and Locations

Go to 

Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Locations

United States, Arizona
Sun City, Arizona, United States, 85351
United States, Connecticut
New Haven, Connecticut, United States, 06510

Sponsors and Collaborators

Piramal Imaging SA

Investigators

Study Director:	Bayer Study Director	Bayer

More Information

Go to 

Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Additional Information:

Click here and search for drug information provided by the FDA. 

Click here and search for information on any recalls, market or product safety alerts by the FDA which might have occurred with this product. 

Responsible Party:	Piramal Imaging SA
ClinicalTrials.gov Identifier:	NCT00928304 History of Changes
Other Study ID Numbers:	14311
First Posted:	June 25, 2009
Results First Posted:	January 28, 2014
Last Update Posted:	March 5, 2014
Last Verified:	January 2014

Keywords provided by Piramal Imaging SA:

Amyloid beta-protein; Down Syndrome

Additional relevant MeSH terms:

Syndrome; Amyloidosis; Down Syndrome; Disease; Pathologic Processes; Proteostasis Deficiencies; Metabolic Diseases; Intellectual Disability	Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn