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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Florbetaben (BAY94-9172) | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Florbetaben (BAY94-9172) | Drug | 300 megabecquerels (MBq) as single IV injection of 2 to 10 mL |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Inclusion Criteria:
Study participants were individuals with DS and healthy volunteers (HVs).
Main inclusion criteria for individuals without DS
Main inclusion criteria for individuals with DS
Exclusion Criteria:
Main exclusion criteria for both groups
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| Name | Affiliation | Role |
|---|---|---|
| Bayer Study Director | Bayer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Sun City | Arizona | 85351 | United States | |||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 33773598 | Derived | 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. |
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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.
The study was conducted at two sites in the US.
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| ID | Title | Description |
|---|---|---|
| FG000 | Down Syndrome Group | 39 Down Syndrome subjects received Florbetaben (BAY94-9172) : 300 megabecquerels (MBq) single IV injection of 2 to 10 mL |
| FG001 | Healthy Volunteer Group | 70 healthy volunteer subjects received Florbetaben (BAY94-9172): 300 MBq single IV injection of 2 to 10 mL |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
All Down Syndrome subjects and healthy volunteers enrolled into the study were included in the Baseline Analysis Population
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| ID | Title | Description |
|---|---|---|
| BG000 | Subjects Enrolled in Study | All Down Syndrome and healthy volunteer subjects |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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. | All subjects in the down syndrome population and healthy volunteers were included in this analysis. | Posted | Number | 95% Confidence Interval | percentage of subjects | 100-120 min |
|
8 days
Adverse event data were collected from Day 1 (baseline) until Day 8 (secondary follow-up visit)
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Down Syndrome Group | 39 Down Syndrome subjects received Florbetaben (BAY94-9172): 300 MBq single IV injection of 2 to 10 mL |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Injection site hematoma | General disorders | MedDRA (13.1) | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Juergen Hirschfeld | Piramal Imaging | 49 30 461 1246 15 | juergen.hirschfeld@piramal.com |
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| ID | Term |
|---|---|
| D004314 | Down Syndrome |
| D058225 | Plaque, Amyloid |
| ID | Term |
|---|---|
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
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| ID | Term |
|---|---|
| C527756 | 4-(N-methylamino)-4'-(2-(2-(2-fluoroethoxy)ethoxy)ethoxy)stilbene |
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| 100 - 120 min |
| 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. |
| 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 |
| New Haven |
| Connecticut |
| 06510 |
| United States |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
|
|
| 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. | All Down Syndrome subjects (n=39) were analyzed for this outcome. | Posted | Number | 95% Confidence Interval | percentage of subjects | 100 - 120 min |
|
|
|
| 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. | All Down Syndrome subjects and healthy volunteers enrolled in the study were included in this analysis | Posted | Mean | Standard Deviation | SUVR | 100 - 120 min p.i. |
|
|
|
| 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. | Posted | Mean | Standard Deviation | SUVR | 100 - 120 min |
|
|
|
| 0 |
| 39 |
| 2 |
| 39 |
| EG001 | Healthy Volunteer Group | 70 healthy volunteer subjects received Florbetaben (BAY94-9172): 300 MBq single IV injection of 2 to 10 mL | 0 | 70 | 5 | 70 |
| Headache | Nervous system disorders | MedDRA (13.1) | Systematic Assessment |
|
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| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D025063 | Chromosome Disorders |
| D030342 | Genetic Diseases, Inborn |
| D020763 | Pathological Conditions, Anatomical |
| D013568 | Pathological Conditions, Signs and Symptoms |
| lateral temporal cortex |
|
| parietal cortex |
|
| cerebellum, white matter |
|
|
| lateral temporal cortex |
|
| parietal cortex |
|
| cerebellum, white matter |
|