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

Down Syndrome Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00928304
• Other study ID #: 14311
• Status: Completed
• Phase: Phase 2
• First received: June 24, 2009
• Last updated: January 29, 2014
• Start date: June 2009
• Est. completion date: January 2011

Study information

• Verified date: January 2014
• Source: Piramal Imaging SA
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 109
• Est. completion date: January 2011
• Est. primary completion date: January 2011
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: Both
• Age group: 21 Years and older

Eligibility

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

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Diagnostic

Related Conditions & MeSH terms

• Amyloid Beta-protein
• Down Syndrome

Intervention

Drug:
• Florbetaben (BAY94-9172)
300 megabecquerels (MBq) as single IV injection of 2 to 10 mL

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Piramal Imaging SA

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Sensitivity of the Independent Visual Assessment of Detecting Cerebral Amyloid-beta in Individuals With Down Syndrome and Specificity in Subjects Without Down Syndrome	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.	100-120 min	No
Secondary	Sensitivity Results in the Down Syndrome Age Subgroups	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.	100 - 120 min	No
Secondary	Quantitative Parameters Standard Uptake Value Ratio	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.	100 - 120 min p.i.	No
Secondary	Consistency Between Visual and Quantitative Efficacy	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.	100 - 120 min	No

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