Back to resultsPhase II Study of Florbetaben (BAY94-9172) PET Imaging for Detection/Exclusion of Cerebral β-amyloid.
Primary Purpose
Down Syndrome, Amyloid Beta-protein
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Florbetaben (BAY94-9172)
Sponsored by
About this trial
This is an interventional diagnostic trial for Down Syndrome focused on measuring Amyloid beta-protein, Down Syndrome
Eligibility 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
Sites / Locations
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Florbetaben (BAY94-9172)
Arm Description
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
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.
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.
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.
Full Information
NCT ID
NCT00928304
First Posted
June 24, 2009
Last Updated
January 29, 2014
Sponsor
Life Molecular Imaging SA
1. Study Identification
Unique Protocol Identification Number
NCT00928304
Brief Title
Phase II Study of Florbetaben (BAY94-9172) PET Imaging for Detection/Exclusion of Cerebral β-amyloid.
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 Type
Interventional
2. Study Status
Record Verification Date
January 2014
Overall Recruitment Status
Completed
Study Start Date
June 2009 (undefined)
Primary Completion Date
January 2011 (Actual)
Study Completion Date
January 2011 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Life Molecular Imaging SA
4. Oversight
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Down Syndrome, Amyloid Beta-protein
Keywords
Amyloid beta-protein, Down Syndrome
7. Study Design
Primary Purpose
Diagnostic
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
109 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Florbetaben (BAY94-9172)
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Florbetaben (BAY94-9172)
Intervention Description
300 megabecquerels (MBq) as single IV injection of 2 to 10 mL
Primary Outcome Measure Information:
Title
Sensitivity of the Independent Visual Assessment of Detecting Cerebral Amyloid-beta in Individuals With Down Syndrome and Specificity in Subjects Without Down Syndrome
Description
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.
Time Frame
100-120 min
Secondary Outcome Measure Information:
Title
Sensitivity Results in the Down Syndrome Age Subgroups
Description
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.
Time Frame
100 - 120 min
Title
Quantitative Parameters Standard Uptake Value Ratio
Description
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.
Time Frame
100 - 120 min p.i.
Title
Consistency Between Visual and Quantitative Efficacy
Description
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.
Time Frame
100 - 120 min
10. Eligibility
Sex
All
Minimum Age & Unit of Time
21 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Bayer Study Director
Organizational Affiliation
Bayer
Official's Role
Study Director
Facility Information:
City
Sun City
State/Province
Arizona
ZIP/Postal Code
85351
Country
United States
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
06510
Country
United States
12. IPD Sharing Statement
Citations:
PubMed Identifier
33773598
Citation
Bullich S, Roe-Vellve N, Marquie M, Landau SM, Barthel H, Villemagne VL, Sanabria A, Tartari JP, Sotolongo-Grau O, Dore V, Koglin N, Muller A, Perrotin A, Jovalekic A, De Santi S, Tarraga L, Stephens AW, Rowe CC, Sabri O, Seibyl JP, Boada M. Early detection of amyloid load using 18F-florbetaben PET. Alzheimers Res Ther. 2021 Mar 27;13(1):67. doi: 10.1186/s13195-021-00807-6.
Results Reference
derived
Learn more about this trial
Phase II Study of Florbetaben (BAY94-9172) PET Imaging for Detection/Exclusion of Cerebral β-amyloid.
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