Trial Outcomes & Findings for Assessment of Hyperphosphorylated Tau PET Binding in Primary Progressive Aphasia and Frontotemporal Dementia (NCT NCT02736695)

Last Updated: 2025-11-21

Results Overview

The percentage of subjects who showed elevated tau PET binding in the following five categories: PPA Clinical Variant-logopenic (lvPPA), PPA Clinical Variant-semantic (svPPA), PPA Clinical Variant-agrammatic (agPPA), no clinical variant-healthy, and Unclassified Variant (PPA-U).

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

81 participants

Primary outcome timeframe

1 week

Results posted on

2025-11-21

Participant Flow

We leveraged data that had already been collected on healthy controls from our NIH funded Mayo Clinic Study of Aging (U01 AG006786). That study recruited healthy subjects of which 102 were controls, who had completed the identical tau-PET protocol with AV-1451 and MRI and clinical protocols. We selected a subset of 80 age and gender match healthy controls from the 102 for use in this study. These 80 participants were not consented or enrolled in this study.

Participant milestones

Participant milestones
Measure	Tau PET Scan, F-18 AV 1451 All subjects will receive a Tau PET scan. F-18 AV 1451: Tau binding agent	Healthy Controls Subjects from our NIH funded Mayo Clinic Study of Aging (U01 AG006786) who have completed the identical tau-PET protocol with AV-1451 and MRI and clinical protocols. These participants were not consented or enrolled in this study.
Overall Study STARTED	81	80
Overall Study COMPLETED	75	80
Overall Study NOT COMPLETED	6	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Tau PET Scan, F-18 AV 1451 All subjects will receive a Tau PET scan. F-18 AV 1451: Tau binding agent	Healthy Controls Subjects from our NIH funded Mayo Clinic Study of Aging (U01 AG006786) who have completed the identical tau-PET protocol with AV-1451 and MRI and clinical protocols. These participants were not consented or enrolled in this study.
Overall Study Ineligible	6	0

Baseline Characteristics

Assessment of Hyperphosphorylated Tau PET Binding in Primary Progressive Aphasia and Frontotemporal Dementia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Tau PET Scan, F-18 AV 1451 n=81 Participants All subjects will receive a Tau PET scan. F-18 AV 1451: Tau binding agent	Healthy Controls n=80 Participants Subjects from our NIH funded Mayo Clinic Study of Aging (U01 AG006786) who have completed the identical tau-PET protocol with AV-1451 and MRI and clinical protocols. These participants were not consented or enrolled in this study.	Total n=161 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=68 Participants	0 Participants n=76 Participants	0 Participants n=48 Participants
Age, Categorical Between 18 and 65 years	35 Participants n=68 Participants	37 Participants n=76 Participants	72 Participants n=48 Participants
Age, Categorical >=65 years	46 Participants n=68 Participants	43 Participants n=76 Participants	89 Participants n=48 Participants
Sex: Female, Male Female	45 Participants n=68 Participants	50 Participants n=76 Participants	95 Participants n=48 Participants
Sex: Female, Male Male	36 Participants n=68 Participants	30 Participants n=76 Participants	66 Participants n=48 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=68 Participants	0 Participants n=76 Participants	1 Participants n=48 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	77 Participants n=68 Participants	80 Participants n=76 Participants	157 Participants n=48 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	3 Participants n=68 Participants	0 Participants n=76 Participants	3 Participants n=48 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=68 Participants	0 Participants n=76 Participants	0 Participants n=48 Participants
Race (NIH/OMB) Asian	4 Participants n=68 Participants	0 Participants n=76 Participants	4 Participants n=48 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=68 Participants	0 Participants n=76 Participants	0 Participants n=48 Participants
Race (NIH/OMB) Black or African American	0 Participants n=68 Participants	0 Participants n=76 Participants	0 Participants n=48 Participants
Race (NIH/OMB) White	77 Participants n=68 Participants	80 Participants n=76 Participants	157 Participants n=48 Participants
Race (NIH/OMB) More than one race	0 Participants n=68 Participants	0 Participants n=76 Participants	0 Participants n=48 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=68 Participants	0 Participants n=76 Participants	0 Participants n=48 Participants
Region of Enrollment United States	81 participants n=68 Participants	80 participants n=76 Participants	161 participants n=48 Participants

PRIMARY outcome

Timeframe: 1 week

Population: Subjects were subclassified into 1 of 5 categories based on clinical features. Classification for Tau PET Scan, F-18 AV 1451 arm: 4 lvPPA; 32 svPPA; 24 agPPA; 0 healthy; 9 PPA-U. Classification for Health Controls: 0 lvPPA; 0 svPPA; 0 agPPA; 80 healthy; 0 PPA-U.

Outcome measures

Outcome measures
Measure	Tau PET Scan, F-18 AV 1451 n=69 Participants All subjects will receive a Tau PET scan. F-18 AV 1451: Tau binding agent	Healthy Controls n=80 Participants Subjects from our NIH funded Mayo Clinic Study of Aging (U01 AG006786) who have completed the identical tau-PET protocol with AV-1451 and MRI and clinical protocols. These participants were not consented or enrolled in this study.
Proportion of PPA Subjects That Showed Elevated Tau PET Binding Compared to Normal Controls No Clinical Variant-healthy	0 Participants	6 Participants
Proportion of PPA Subjects That Showed Elevated Tau PET Binding Compared to Normal Controls PPA Clinical Variant-Unclassified (PPA-U)	1 Participants	0 Participants
Proportion of PPA Subjects That Showed Elevated Tau PET Binding Compared to Normal Controls PPA Clinical Variant-semantic (svPPA)	17 Participants	0 Participants
Proportion of PPA Subjects That Showed Elevated Tau PET Binding Compared to Normal Controls PPA Clinical Variant-agrammatic (agPPA)	5 Participants	0 Participants
Proportion of PPA Subjects That Showed Elevated Tau PET Binding Compared to Normal Controls PPA Clinical Varient-logopenic (lvPPA)	3 Participants	0 Participants

SECONDARY outcome

Timeframe: 1 Week

Population: 40 subjects were included in the analysis as there was enough data from these 40 subjects to address aim 2. Subjects were subclassified into 1 of 3 PPA variants based on clinical features. Classification: 14 lvPPA; 13 svPPA; 13 agPPA. Signature patterns of regional tau PET binding in clinical PPA variants were not measured in the Mayo Clinic Study of Aging trial, therefore the healthy controls arm does not apply to this outcome as no data exists for that arm.

To determine whether each of the three clinical PPA variants (logopenic (lvPPA), semantic (svPPA) and agrammatic (agPPA)) has a signature pattern of regional tau PET binding by measuring the level of tau PET binding in each PPA variant. 0=represents no specific uptake pattern; 1=specific uptake pattern

Outcome measures

Outcome measures
Measure	Tau PET Scan, F-18 AV 1451 n=40 Participants All subjects will receive a Tau PET scan. F-18 AV 1451: Tau binding agent	Healthy Controls Subjects from our NIH funded Mayo Clinic Study of Aging (U01 AG006786) who have completed the identical tau-PET protocol with AV-1451 and MRI and clinical protocols. These participants were not consented or enrolled in this study.
Signature Patterns of Regional Tau PET Binding in Clinical PPA Variants logopenic (lvPPA)	1 units on a scale	—
Signature Patterns of Regional Tau PET Binding in Clinical PPA Variants semantic (svPPA)	1 units on a scale	—
Signature Patterns of Regional Tau PET Binding in Clinical PPA Variants agrammatic (agPPA)	1 units on a scale	—

OTHER_PRE_SPECIFIED outcome

Timeframe: 1 Week

Population: 40 subjects were included in the analysis as there was enough data from these 40 subjects to address aim 3. Subjects were classified into groups based on uptake patterns. Classification: 5 Low PC1/High PC2; 10 Low PC1/Low PC2; 13 High PC1/High PC2, 12 High PC1/Low PC2.Variability in patterns of tau PET binding was not measured in the Mayo Clinic Study of Aging trial; therefore, the healthy controls arm does not apply to this outcome as no data exists for that arm.

To identify variability in patterns of tau PET binding using principal component (PC) analysis based on regional \[18F\]AV-1451 uptake to identify patterns between the three clinical variants: lvPPA, svPPA and agPPA independent of clinical diagnosis among PPA subjects. Two PCs were utilized to determine the number of participants who fell into the following 4 categories based on regional \[18F\]AV-1451 uptake patterns: low PC1/high PC2, low PC1/low PC2, high PC1/high PC2 and high PC1/low PC2. The first principal component (PC1) is a weighted sum of regional data where the weights are chosen so that PC1 has maximum variation across subjects. PC1 can be thought of as the "best" single-number summary of the regional data in a given modality. The second principal component (PC2) is a weighted sum of regional data with weight chosen so that (1) PC2 is completely uncorrelated with PC1 and (2) PC2 has maximum variation after accounting for PC1.

Outcome measures

Outcome measures
Measure	Tau PET Scan, F-18 AV 1451 n=40 Participants All subjects will receive a Tau PET scan. F-18 AV 1451: Tau binding agent	Healthy Controls Subjects from our NIH funded Mayo Clinic Study of Aging (U01 AG006786) who have completed the identical tau-PET protocol with AV-1451 and MRI and clinical protocols. These participants were not consented or enrolled in this study.
Variability in Patterns of Tau PET Binding High PC1/Low PC2 · svPPA	0 Participants	—
Variability in Patterns of Tau PET Binding Low PC1/High PC2 · lvPPA	5 Participants	—
Variability in Patterns of Tau PET Binding Low PC1/High PC2 · agPPA	0 Participants	—
Variability in Patterns of Tau PET Binding Low PC1/High PC2 · svPPA	0 Participants	—
Variability in Patterns of Tau PET Binding Low PC1/Low PC2 · lvPPA	9 Participants	—
Variability in Patterns of Tau PET Binding Low PC1/Low PC2 · agPPA	1 Participants	—
Variability in Patterns of Tau PET Binding Low PC1/Low PC2 · svPPA	0 Participants	—
Variability in Patterns of Tau PET Binding High PC1/High PC2 · lvPPA	0 Participants	—
Variability in Patterns of Tau PET Binding High PC1/High PC2 · agPPA	0 Participants	—
Variability in Patterns of Tau PET Binding High PC1/High PC2 · svPPA	13 Participants	—
Variability in Patterns of Tau PET Binding High PC1/Low PC2 · lvPPA	0 Participants	—
Variability in Patterns of Tau PET Binding High PC1/Low PC2 · agPPA	12 Participants	—

Adverse Events

Tau PET Scan, F-18 AV 1451

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Healthy Controls

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Keith Josephs, M.D.

Mayo Clinic

Phone: 507-266-4106

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place