Trial Outcomes & Findings for Potential of Florbetapir F 18 PET to Inform Clinical Diagnosis and Management of Patients With Progressive Cognitive Decline (NCT NCT01400425)

Last Updated: 2013-03-29

Results Overview

The impact of a negative florbetapir F 18 PET scan on a physician's clinical diagnosis and management of a subject was evaluated on a hypothetical basis because at the start of this study Florbetapir F 18 was an investigational drug and the data collected is for research purposes only. The percentage of subjects who received a negative florbetapir scan that led to a change in hypothetical clinical diagnosis and physician management plans are presented below. A negative florbetapir PET scan is indicative of none to sparse β-amyloid neuritic plaque density according to the modified Consortium to Establish a Registry for Alzheimer's Disease (CERAD) criteria.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

239 participants

Primary outcome timeframe

6 weeks

Results posted on

2013-03-29

Participant Flow

Participant milestones

Participant milestones
Measure	Subjects With Progressive Cognitive Decline Subjects who have previously or are currently being evaluated for progressive cognitive decline. Enrolling physicians must have a confidence of less than 85% in their initial diagnosis of the subject's progressive cognitive decline and that there was at least a 15% chance that the subject's progressive cognitive decline was due to Alzheimer's disease. All subjects who did not complete the study withdrew before receiving a florbetapir F 18 injection and PET scan. Subjects received IV injection, 370 MBq (10 mCi) florbetapir F18, single dose.
Overall Study STARTED	239
Overall Study COMPLETED	229
Overall Study NOT COMPLETED	10

Reasons for withdrawal

Reasons for withdrawal
Measure	Subjects With Progressive Cognitive Decline Subjects who have previously or are currently being evaluated for progressive cognitive decline. Enrolling physicians must have a confidence of less than 85% in their initial diagnosis of the subject's progressive cognitive decline and that there was at least a 15% chance that the subject's progressive cognitive decline was due to Alzheimer's disease. All subjects who did not complete the study withdrew before receiving a florbetapir F 18 injection and PET scan. Subjects received IV injection, 370 MBq (10 mCi) florbetapir F18, single dose.
Overall Study Withdrawal by Subject	10

Baseline Characteristics

Potential of Florbetapir F 18 PET to Inform Clinical Diagnosis and Management of Patients With Progressive Cognitive Decline

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Subjects With Progressive Cognitive Decline n=229 Participants Subjects who have previously or are currently being evaluated for progressive cognitive decline. Enrolling physicians must have a confidence of less than 85% in their initial diagnosis of the subject's progressive cognitive decline and that there was at least a 15% chance that the subject's progressive cognitive decline was due to Alzheimer's disease. All subjects who did not complete the study withdrew before receiving a florbetapir F 18 injection and PET scan. Subjects received IV injection, 370 MBq (10 mCi) florbetapir F18, single dose.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	24 Participants n=5 Participants
Age, Categorical >=65 years	205 Participants n=5 Participants
Age Continuous	74.1 years STANDARD_DEVIATION 8.1 • n=5 Participants
Sex: Female, Male Female	114 Participants n=5 Participants
Sex: Female, Male Male	115 Participants n=5 Participants
Region of Enrollment United States	229 participants n=5 Participants

PRIMARY outcome

Timeframe: 6 weeks

Population: 116 of 229 subjects with progressive cognitive decline received a negative florbetapir scan.

Outcome measures

Outcome measures
Measure	Subjects With Progressive Cognitive Decline n=116 Participants Subjects who have previously or are currently being evaluated for progressive cognitive decline. Enrolling physicians must have a confidence of less than 85% in their initial diagnosis of the subject's progressive cognitive decline and that there was at least a 15% chance that the subject's progressive cognitive decline was due to Alzheimer's disease. All subjects who did not complete the study withdrew before receiving a florbetapir F 18 injection and PET scan. Subjects received IV injection, 370 MBq (10 mCi) florbetapir F18, single dose.	Subjectss With a Positive Scan Subjects with progressive cognitive decline that received a positive florbetapir scan.	Subjects With a Negative Scan Subjects with progressive cognitive decline that received a negative scan.
Percentage of Subjects Who Undergo a Hypothetical Change in Clinical Diagnosis and Physician Management Plan After Obtaining a Negative Florbetapir F 18 PET Scan. Change in Clinical Diagnosis	56.9 Percentage of subjects Interval 47.8 to 65.5	—	—
Percentage of Subjects Who Undergo a Hypothetical Change in Clinical Diagnosis and Physician Management Plan After Obtaining a Negative Florbetapir F 18 PET Scan. Change in Physician Management Plan	81.9 Percentage of subjects Interval 73.9 to 87.8	—	—

SECONDARY outcome

Timeframe: 6 weeks

Population: 113 out of 229 subjects with progressive cognitive decline received a positive florbetapir scan.

The impact of a positive florbetapir F 18 PET scan on a physician's clinical diagnosis and management of a subject was evaluated on a hypothetical basis because at the start of this study Florbetapir F 18 was an investigational drug and the data collected is for research purposes only. The percentage of subjects who received a positive florbetapir scan that led to a change in hypothetical clinical diagnosis and physician management plans are presented below. A positive florbetapir PET scan is indicative of moderate to frequent β-amyloid neuritic plaque density according to the modified Consortium to Establish a Registry for Alzheimer's Disease (CERAD) criteria.

Outcome measures

Outcome measures
Measure	Subjects With Progressive Cognitive Decline n=113 Participants Subjects who have previously or are currently being evaluated for progressive cognitive decline. Enrolling physicians must have a confidence of less than 85% in their initial diagnosis of the subject's progressive cognitive decline and that there was at least a 15% chance that the subject's progressive cognitive decline was due to Alzheimer's disease. All subjects who did not complete the study withdrew before receiving a florbetapir F 18 injection and PET scan. Subjects received IV injection, 370 MBq (10 mCi) florbetapir F18, single dose.	Subjectss With a Positive Scan Subjects with progressive cognitive decline that received a positive florbetapir scan.	Subjects With a Negative Scan Subjects with progressive cognitive decline that received a negative scan.
Percentage of Subjects Who Undergo a Hypothetical Change in Clinical Diagnosis and Physician Management Plan After Obtaining a Positive Florbetapir F 18 PET Scan Change in Clinical Diagnosis	52.2 Percentage of subjects Interval 43.1 to 61.2	—	—
Percentage of Subjects Who Undergo a Hypothetical Change in Clinical Diagnosis and Physician Management Plan After Obtaining a Positive Florbetapir F 18 PET Scan Change in Physician Management Plan	92.0 Percentage of subjects Interval 85.6 to 95.8	—	—

SECONDARY outcome

Timeframe: 6 weeks

Population: The hypothetical clinical diagnosis remained unchanged in 62 of 229 subjects who received a florbetapir scan.

Change in confidence of the clinical diagnosis prior to obtaining a florbetapir F 18 PET scan to the confidence after obtaining a florbetapir F 18 PET scan among subjects in whom the clinical diagnosis remains unchanged. Confidence levels were self-determined by physicians based on their diagnostic certainty and ranged from 0-100%. The mean (SD) change in confidence reflects the average change in diagnostic confidence along the 0-100% scale for the 62 subjects analyzed.

Outcome measures

Outcome measures
Measure	Subjects With Progressive Cognitive Decline n=62 Participants Subjects who have previously or are currently being evaluated for progressive cognitive decline. Enrolling physicians must have a confidence of less than 85% in their initial diagnosis of the subject's progressive cognitive decline and that there was at least a 15% chance that the subject's progressive cognitive decline was due to Alzheimer's disease. All subjects who did not complete the study withdrew before receiving a florbetapir F 18 injection and PET scan. Subjects received IV injection, 370 MBq (10 mCi) florbetapir F18, single dose.	Subjectss With a Positive Scan Subjects with progressive cognitive decline that received a positive florbetapir scan.	Subjects With a Negative Scan Subjects with progressive cognitive decline that received a negative scan.
Change in Confidence of the Clinical Diagnosis	23 Percent Change in Confidence Standard Deviation 11.9	—	—

SECONDARY outcome

Timeframe: 6 weeks

Population: All subjects with progressive cognitive decline who have received a florbetapir scan.

Determine the percentage of subjects that had at least one hypothetical change between pre and post scan physician management plans. Change in management is defined as the number of subjects prescribed different item-wise plans at the two assessments divided by the total number of subjects in the population with both a pre and post florbetapir F 18 PET scan physician management plan.

Outcome measures

Outcome measures
Measure	Subjects With Progressive Cognitive Decline n=229 Participants Subjects who have previously or are currently being evaluated for progressive cognitive decline. Enrolling physicians must have a confidence of less than 85% in their initial diagnosis of the subject's progressive cognitive decline and that there was at least a 15% chance that the subject's progressive cognitive decline was due to Alzheimer's disease. All subjects who did not complete the study withdrew before receiving a florbetapir F 18 injection and PET scan. Subjects received IV injection, 370 MBq (10 mCi) florbetapir F18, single dose.	Subjectss With a Positive Scan Subjects with progressive cognitive decline that received a positive florbetapir scan.	Subjects With a Negative Scan Subjects with progressive cognitive decline that received a negative scan.
Change in Physician Management Plans	86.9 Percentage of subjects Interval 81.9 to 90.7	—	—

OTHER_PRE_SPECIFIED outcome

Timeframe: 6 weeks

Population: The number of subjects analyzed for each reporting group is determined by scan status. There were 229 total subjects with progressive cognitive decline of whom 113 received a positive florbetapir scan and 116 received a negative florbetapir scan.

This outcome analyzed the percentage of subjects who had a hypothetical change in one of the medication or diagnostic categories listed below after receiving a florbetapir scan.

Outcome measures

Outcome measures
Measure	Subjects With Progressive Cognitive Decline n=229 Participants Subjects who have previously or are currently being evaluated for progressive cognitive decline. Enrolling physicians must have a confidence of less than 85% in their initial diagnosis of the subject's progressive cognitive decline and that there was at least a 15% chance that the subject's progressive cognitive decline was due to Alzheimer's disease. All subjects who did not complete the study withdrew before receiving a florbetapir F 18 injection and PET scan. Subjects received IV injection, 370 MBq (10 mCi) florbetapir F18, single dose.	Subjectss With a Positive Scan n=113 Participants Subjects with progressive cognitive decline that received a positive florbetapir scan.	Subjects With a Negative Scan n=116 Participants Subjects with progressive cognitive decline that received a negative scan.
Item Wise Changes in Physician Management Plan AD Medication	31.0 Percentage of subjects	31.9 Percentage of subjects	30.2 Percentage of subjects
Item Wise Changes in Physician Management Plan Psychiatric Medication	7.4 Percentage of subjects	3.5 Percentage of subjects	11.2 Percentage of subjects
Item Wise Changes in Physician Management Plan Refer to Clinical Trial	16.2 Percentage of subjects	22.1 Percentage of subjects	10.3 Percentage of subjects
Item Wise Changes in Physician Management Plan Brain Structural Imaging (CT/MRI)	14.0 Percentage of subjects	15.0 Percentage of subjects	12.9 Percentage of subjects
Item Wise Changes in Physician Management Plan Lumbar Puncture	10.5 Percentage of subjects	8.8 Percentage of subjects	12.1 Percentage of subjects
Item Wise Changes in Physician Management Plan Neuropsychologic Testing	22.7 Percentage of subjects	23.9 Percentage of subjects	21.6 Percentage of subjects
Item Wise Changes in Physician Management Plan FDG PET	10.0 Percentage of subjects	10.6 Percentage of subjects	9.5 Percentage of subjects
Item Wise Changes in Physician Management Plan ApoE Genotype	5.2 Percentage of subjects	5.3 Percentage of subjects	5.2 Percentage of subjects
Item Wise Changes in Physician Management Plan Lab Test	7.9 Percentage of subjects	10.6 Percentage of subjects	5.2 Percentage of subjects

OTHER_PRE_SPECIFIED outcome

Timeframe: 6 weeks

Population: Subjects who have received a florbetapir scan (either positive or negative).

The impact of a florbetapir F 18 PET scan on a physician's clinical diagnosis of a subject was evaluated on a hypothetical basis because at the start of this study Florbetapir F 18 was an investigational drug and the data collected is for research purposes only. The percentage of subjects who received a florbetapir scan that led to a change in hypothetical clinical diagnosis is presented below.

Outcome measures

Outcome measures
Measure	Subjects With Progressive Cognitive Decline n=229 Participants Subjects who have previously or are currently being evaluated for progressive cognitive decline. Enrolling physicians must have a confidence of less than 85% in their initial diagnosis of the subject's progressive cognitive decline and that there was at least a 15% chance that the subject's progressive cognitive decline was due to Alzheimer's disease. All subjects who did not complete the study withdrew before receiving a florbetapir F 18 injection and PET scan. Subjects received IV injection, 370 MBq (10 mCi) florbetapir F18, single dose.	Subjectss With a Positive Scan Subjects with progressive cognitive decline that received a positive florbetapir scan.	Subjects With a Negative Scan Subjects with progressive cognitive decline that received a negative scan.
Percentage of Subjects Who Undergo a Hypothetical Change in Clinical Diagnosis After Obtaining a Florbetapir F 18 PET Scan.	54.6 Percentage of subjects Interval 48.1 to 60.9	—	—

Adverse Events

Subjects With Progressive Cognitive Decline

Serious events: 0 serious events

Other events: 13 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Subjects With Progressive Cognitive Decline n=229 participants at risk Subjects who have previously or are currently being evaluated for progressive cognitive decline. Enrolling physicians must have a confidence of less than 85% in their initial diagnosis of the subject's progressive cognitive decline and that there was at least a 15% chance that the subject's progressive cognitive decline was due to Alzheimer's disease. All subjects who did not complete the study withdrew before receiving a florbetapir F 18 injection and PET scan. Subjects received IV injection, 370 MBq (10 mCi) florbetapir F18, single dose.
Gastrointestinal disorders Diarrhoea	0.44% 1/229 • Number of events 1
Gastrointestinal disorders Nausea	0.44% 1/229 • Number of events 1
Infections and infestations Urinary tract infection	0.44% 1/229 • Number of events 1
Musculoskeletal and connective tissue disorders Back pain	0.44% 1/229 • Number of events 1
Musculoskeletal and connective tissue disorders Musculoskeletal stiffness	0.44% 1/229 • Number of events 1
Nervous system disorders Headache	1.7% 4/229 • Number of events 4
Nervous system disorders Balance disorder	0.44% 1/229 • Number of events 1
Nervous system disorders Crying	0.44% 1/229 • Number of events 1
Nervous system disorders Dizziness	0.44% 1/229 • Number of events 1
Nervous system disorders Paraesthesia	0.44% 1/229 • Number of events 1
Nervous system disorders Somnolence	0.44% 1/229 • Number of events 1
Psychiatric disorders Anxiety	0.44% 1/229 • Number of events 1
Reproductive system and breast disorders Breast pain	0.44% 1/229 • Number of events 1
Infections and infestations Gastroenteritis viral	0.44% 1/229 • Number of events 1

Additional Information

Chief Medical Officer

Avid Radiopharmaceuticals

Phone: 215-298-0700

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60