Trial Outcomes & Findings for Flortaucipir PET Imaging in Subjects With FTD (NCT NCT03040713)

Last Updated: 2020-09-25

Results Overview

Subject scans were visually evaluated by an expert reader into three groups. Advanced Alzheimer's Disease (AD) scan pattern = In either hemisphere, increased neocortical activity in the parietal/precuneus region(s), or frontal region(s) with increased uptake in the posterolateral temporal (PLT), parietal, or occipital region(s). Moderate AD scan pattern = In either hemisphere, increased neocortical activity limited to the PLT or occipital region(s). Not AD scan pattern = No increased neocortical activity, or increased neocortical activity isolated to the mesial temporal, anterolateral temporal, and/or frontal regions.

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

16 participants

Primary outcome timeframe

baseline scan

Results posted on

2020-09-25

Participant Flow

Enrollment occurred between Apr 2017 and Oct 2018.

As a result of a positive amyloid scan at screening, 1 subject was discontinued prior to administration of flortaucipir. Two additional subjects had positive amyloid scans and received flortaucipir, but were excluded from the per protocol population results (n=13).

Participant milestones

Participant milestones
Measure	FTD Subjects Subjects diagnosed by dementia specialist with a clinical Frontotemporal Dementia (FTD) syndrome and expected tau or tar DNA binding protein (TDP)-43 pathology receiving a flortaucipir PET scan
Overall Study STARTED	16
Overall Study COMPLETED	13
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Reasons for withdrawal
Measure	FTD Subjects Subjects diagnosed by dementia specialist with a clinical Frontotemporal Dementia (FTD) syndrome and expected tau or tar DNA binding protein (TDP)-43 pathology receiving a flortaucipir PET scan
Overall Study Positive amyloid scan	3

Baseline Characteristics

Flortaucipir PET Imaging in Subjects With FTD

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	FTD Subjects n=15 Participants Subjects diagnosed by dementia specialist with a clinical Frontotemporal Dementia (FTD) syndrome and expected tau or TDP-43 pathology receiving a flortaucipir PET scan
Age, Continuous	64.7 years STANDARD_DEVIATION 6.22 • n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants
Sex: Female, Male Male	10 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	14 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	14 Participants n=5 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	15 participants n=5 Participants
MMSE	25.6 units on a scale STANDARD_DEVIATION 6.38 • n=5 Participants

PRIMARY outcome

Timeframe: baseline scan

Outcome measures

Outcome measures
Measure	FTD Subjects n=13 Participants Subjects diagnosed by dementia specialist with a clinical Frontotemporal Dementia (FTD) syndrome and expected tau or TDP-43 pathology receiving a flortaucipir PET scan
Qualitative Evaluation of Flortaucipir PET Scans Advanced AD Scan Pattern (τAD++)	0 Participants
Qualitative Evaluation of Flortaucipir PET Scans Moderate AD Scan Pattern (τAD+)	0 Participants
Qualitative Evaluation of Flortaucipir PET Scans Not AD Scan Pattern (τAD-)	13 Participants

PRIMARY outcome

Timeframe: baseline scan

Population: All amyloid negative FTD subjects who had a PET scan that was able to be quantitatively evaluated (n=12). One subject did not have magnetic resonance imaging available, so quantitation was not possible.

Standard Uptake Value Ratio (SUVr) using a weighted cortical average (MUBADA), and individual regions. For SUVr, a value of 1 or lower signifies no flortaucipir activity above background, values greater than 1 signify increasing flortaucipir activity in the brain.

Outcome measures

Outcome measures
Measure	FTD Subjects n=12 Participants Subjects diagnosed by dementia specialist with a clinical Frontotemporal Dementia (FTD) syndrome and expected tau or TDP-43 pathology receiving a flortaucipir PET scan
Quantitative Evaluation of Flortaucipir PET Scans Caudate	0.63508 standardized uptake value ratio (SUVr) Standard Deviation 0.130466
Quantitative Evaluation of Flortaucipir PET Scans MUBADA	1.00903 standardized uptake value ratio (SUVr) Standard Deviation 0.036582
Quantitative Evaluation of Flortaucipir PET Scans Parietal	0.91992 standardized uptake value ratio (SUVr) Standard Deviation 0.054753
Quantitative Evaluation of Flortaucipir PET Scans Temporal	0.94920 standardized uptake value ratio (SUVr) Standard Deviation 0.041189
Quantitative Evaluation of Flortaucipir PET Scans Frontal	0.89825 standardized uptake value ratio (SUVr) Standard Deviation 0.086118

Adverse Events

FTD Subjects Safety Population

Serious events: 0 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	FTD Subjects Safety Population n=15 participants at risk Subjects diagnosed with a clinical Frontotemporal Dementia (FTD) syndrome and expected tau or TDP-43 pathology receiving a flortaucipir PET scan
General disorders Feeling cold	6.7% 1/15 • Number of events 1 • AEs were collected at scan visit and for 48 hours after flortaucipir administration. Adverse Events were defined as occurring or worsening after injection of dose, within 48 hours post injection, at an imaging visit. AEs occurring after study drug administration, but outside that window were not recorded, unless considered attributable to study drug.
General disorders Injection site pain	6.7% 1/15 • Number of events 1 • AEs were collected at scan visit and for 48 hours after flortaucipir administration. Adverse Events were defined as occurring or worsening after injection of dose, within 48 hours post injection, at an imaging visit. AEs occurring after study drug administration, but outside that window were not recorded, unless considered attributable to study drug.
Nervous system disorders Headache	6.7% 1/15 • Number of events 1 • AEs were collected at scan visit and for 48 hours after flortaucipir administration. Adverse Events were defined as occurring or worsening after injection of dose, within 48 hours post injection, at an imaging visit. AEs occurring after study drug administration, but outside that window were not recorded, unless considered attributable to study drug.
Respiratory, thoracic and mediastinal disorders Epistaxis	6.7% 1/15 • Number of events 1 • AEs were collected at scan visit and for 48 hours after flortaucipir administration. Adverse Events were defined as occurring or worsening after injection of dose, within 48 hours post injection, at an imaging visit. AEs occurring after study drug administration, but outside that window were not recorded, unless considered attributable to study drug.

Additional Information

Medical Director

Avid Radiopharmaceuticals, Inc.

Phone: 215-298-0700

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60