Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 54 of 165 for:    trisomy21 NOT prenatal

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00928304
Recruitment Status : Completed
First Posted : June 25, 2009
Results First Posted : January 28, 2014
Last Update Posted : March 5, 2014
Sponsor:
Information provided by (Responsible Party):
Life Molecular Imaging SA

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Diagnostic
Conditions Down Syndrome
Amyloid Beta-protein
Intervention Drug: Florbetaben (BAY94-9172)
Enrollment 109
Recruitment Details The study was conducted at two sites in the US.
Pre-assignment Details A total of 130 subjects were screened. 21 subjects were considered as screen failures and were not enrolled into the study. 9 subjects did not meet the inclusion/exclusion criteria. 10 subjects withdrew their consent. 1 subject was lost to follow-up and 1 subjected exceeded the weight limit for the camera.
Arm/Group Title Down Syndrome Group Healthy Volunteer Group
Hide Arm/Group Description 39 Down Syndrome subjects received Florbetaben (BAY94-9172) : 300 megabecquerels (MBq) single IV injection of 2 to 10 mL 70 healthy volunteer subjects received Florbetaben (BAY94-9172): 300 MBq single IV injection of 2 to 10 mL
Period Title: Overall Study
Started 39 70
Completed 39 70
Not Completed 0 0
Arm/Group Title Subjects Enrolled in Study
Hide Arm/Group Description All Down Syndrome and healthy volunteer subjects
Overall Number of Baseline Participants 109
Hide Baseline Analysis Population Description
All Down Syndrome subjects and healthy volunteers enrolled into the study were included in the Baseline Analysis Population
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 109 participants
<=18 years
0
   0.0%
Between 18 and 65 years
109
 100.0%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 109 participants
Down Syndrome Group 46.3  (4.7)
Healthy Volunteer Group 27.7  (5.1)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 109 participants
Female
60
  55.0%
Male
49
  45.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 109 participants
109
1.Primary Outcome
Title Sensitivity of the Independent Visual Assessment of Detecting Cerebral Amyloid-beta in Individuals With Down Syndrome and Specificity in Subjects Without Down Syndrome
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects in the down syndrome population and healthy volunteers were included in this analysis.
Arm/Group Title Majority Read
Hide Arm/Group Description:
Majority read of the visual assessment made by 3 independent readers of PET images from all subjects.
Overall Number of Participants Analyzed 109
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of subjects
Sensitivity
46.15
(30.51 to 61.80)
Specificity
100.00
(100.00 to 100.00)
2.Secondary Outcome
Title Sensitivity Results in the Down Syndrome Age Subgroups
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
All Down Syndrome subjects (n=39) were analyzed for this outcome.
Arm/Group Title Majority Read (DS-Young) Majority Read (DS-Old)
Hide Arm/Group Description:
Majority Read results for Down Syndrome subjects with age <= 46 yrs
Majority Read results for Down Syndrome subjects with age >46 yrs
Overall Number of Participants Analyzed 21 18
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of subjects
23.81
(5.59 to 42.03)
72.22
(51.53 to 92.91)
3.Secondary Outcome
Title Quantitative Parameters Standard Uptake Value Ratio
Hide 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.
Hide Outcome Measure Data
Hide Analysis Population Description
All Down Syndrome subjects and healthy volunteers enrolled in the study were included in this analysis
Arm/Group Title Down Syndrome Group Healthy Volunteer Group
Hide Arm/Group Description:
Subjects with Down Syndrome enrolled in the study
Healthy volunteers enrolled in the study
Overall Number of Participants Analyzed 39 70
Mean (Standard Deviation)
Unit of Measure: SUVR
frontal cortex 1.396  (0.2488) 1.146  (0.0749)
posterior cingulate 1.525  (0.2549) 1.329  (0.1118)
lateral temporal cortex 1.350  (0.2265) 1.132  (0.0623)
parietal cortex 1.391  (0.2594) 1.138  (0.0742)
cerebellum, white matter 1.730  (0.2115) 1.769  (0.1302)
4.Secondary Outcome
Title Consistency Between Visual and Quantitative Efficacy
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Down Syndrome PET-positive (Majority Read) Down Syndrome PET-negative (Majority Read) Healthy Volunteer Group
Hide Arm/Group Description:
Down Syndrome subjects positive for cerebral beta-amyloid as determined by majority read
Down Syndrome subjects negative for cerebral beta-amyloid as determined by majority read
All healthy volunteers enrolled in the study
Overall Number of Participants Analyzed 18 21 70
Mean (Standard Deviation)
Unit of Measure: SUVR
frontal cortex 1.566  (0.2372) 1.249  (0.1451) 1.146  (0.0749)
posterior cingulate 1.626  (0.2372) 1.438  (0.2421) 1.329  (0.1118)
lateral temporal cortex 1.498  (0.2396) 1.223  (0.1092) 1.132  (0.0623)
parietal cortex 1.557  (0.2567) 1.250  (0.1620) 1.138  (0.0742)
cerebellum, white matter 1.698  (0.2396) 1.758  (0.1856) 1.769  (0.1302)
Time Frame 8 days
Adverse Event Reporting Description Adverse event data were collected from Day 1 (baseline) until Day 8 (secondary follow-up visit)
 
Arm/Group Title Down Syndrome Group Healthy Volunteer Group
Hide Arm/Group Description 39 Down Syndrome subjects received Florbetaben (BAY94-9172): 300 MBq single IV injection of 2 to 10 mL 70 healthy volunteer subjects received Florbetaben (BAY94-9172): 300 MBq single IV injection of 2 to 10 mL
All-Cause Mortality
Down Syndrome Group Healthy Volunteer Group
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Down Syndrome Group Healthy Volunteer Group
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/39 (0.00%)      0/70 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Down Syndrome Group Healthy Volunteer Group
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   2/39 (5.13%)      5/70 (7.14%)    
General disorders     
Injection site hematoma  1  2/39 (5.13%)  2 1/70 (1.43%)  1
Nervous system disorders     
Headache  1  0/39 (0.00%)  0 5/70 (7.14%)  5
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (13.1)
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Dr. Juergen Hirschfeld
Organization: Piramal Imaging
Phone: 49 30 461 1246 15
Responsible Party: Life Molecular Imaging SA
ClinicalTrials.gov Identifier: NCT00928304     History of Changes
Other Study ID Numbers: 14311
First Submitted: June 24, 2009
First Posted: June 25, 2009
Results First Submitted: December 9, 2013
Results First Posted: January 28, 2014
Last Update Posted: March 5, 2014