Trial Outcomes & Findings for Tau Imaging in Subjects With Progressive Supranuclear Palsy, Corticobasal Degeneration and Healthy Volunteers (NCT NCT02167594)

Last Updated: 2020-08-28

Results Overview

A PSP/CBD (PSP = progressive supranuclear palsy; CBD = corticobasal degeneration) targeted composite Standardized Uptake Value ratio (SUVr) was calculated as the voxel-weighted average from the regional SUVr values of the left, right, and total globus pallidus and the dentate nuclei of the cerebellum for both the baseline and 9 month PET scans. Additionally, a global cortical Alzheimer's Disease (AD)-targeted cortical SUVr was calculated. For SUVr, a value of 1 signifies no flortaucipir activity above background, values greater than 1 signify increasing flortaucipir activity in the brain.

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

29 participants

Primary outcome timeframe

baseline and 9 month scans

Results posted on

2020-08-28

Participant Flow

Subjects recruited between Aug 2014 and Jul 2016.

Participant milestones

Participant milestones
Measure	PSP Subjects Amyloid negative subjects with Progressive Supranuclear Palsy (PSP) receiving a flortaucipir PET scan	CBD Subjects Amyloid negative subjects with Corticobasal Degeneration (CBD) receiving a flortaucipir PET scan	Healthy Volunteers Healthy volunteers receiving a flortaucipir PET scan
Overall Study STARTED	20	6	3
Overall Study COMPLETED	18	5	3
Overall Study NOT COMPLETED	2	1	0

Reasons for withdrawal

Reasons for withdrawal
Measure	PSP Subjects Amyloid negative subjects with Progressive Supranuclear Palsy (PSP) receiving a flortaucipir PET scan	CBD Subjects Amyloid negative subjects with Corticobasal Degeneration (CBD) receiving a flortaucipir PET scan	Healthy Volunteers Healthy volunteers receiving a flortaucipir PET scan
Overall Study Withdrawal by Subject	2	0	0
Overall Study Death	0	1	0

Baseline Characteristics

Tau Imaging in Subjects With Progressive Supranuclear Palsy, Corticobasal Degeneration and Healthy Volunteers

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	PSP Subjects n=20 Participants Amyloid negative subjects with PSP receiving a flortaucipir PET scan at baseline and at 9 months. Flortaucipir F18 PET scan: IV injection, 370 MBq (10 mCi)	CBD Subjects n=6 Participants Amyloid negative subjects with CBD receiving a flortaucipir PET scan at baseline and at 9 months. Flortaucipir F18 PET scan: IV injection, 370 MBq (10 mCi)	Healthy Volunteers n=3 Participants Healthy volunteers receiving a flortaucipir PET scan at baseline. Flortaucipir F18 PET scan: IV injection, 370 MBq (10 mCi)	Total n=29 Participants Total of all reporting groups
Age, Continuous	69.2 years STANDARD_DEVIATION 5.88 • n=5 Participants	72.2 years STANDARD_DEVIATION 7.41 • n=7 Participants	68.7 years STANDARD_DEVIATION 10.41 • n=5 Participants	69.8 years STANDARD_DEVIATION 6.53 • n=4 Participants
Sex: Female, Male Female	6 Participants n=5 Participants	4 Participants n=7 Participants	0 Participants n=5 Participants	10 Participants n=4 Participants
Sex: Female, Male Male	14 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants	19 Participants n=4 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	19 Participants n=5 Participants	6 Participants n=7 Participants	3 Participants n=5 Participants	28 Participants n=4 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) White	18 Participants n=5 Participants	6 Participants n=7 Participants	3 Participants n=5 Participants	27 Participants n=4 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants
Region of Enrollment United States	20 Participants n=5 Participants	6 Participants n=7 Participants	3 Participants n=5 Participants	29 Participants n=4 Participants
Mini Mental Status Exam (MMSE)	26.5 units on a scale STANDARD_DEVIATION 3.12 • n=5 Participants	26.3 units on a scale STANDARD_DEVIATION 4.32 • n=7 Participants	29.3 units on a scale STANDARD_DEVIATION 0.58 • n=5 Participants	26.7 units on a scale STANDARD_DEVIATION 3.28 • n=4 Participants

PRIMARY outcome

Timeframe: baseline and 9 month scans

Population: Healthy volunteers did not receive 9 month follow-up scans. 9 month data presented only for those subjects who completed both a baseline and 9 month scan.

Outcome measures

Outcome measures
Measure	PSP Subjects n=20 Participants Amyloid negative subjects with PSP receiving a flortaucipir PET scan at baseline and at 9 months. Flortaucipir F18 PET scan: IV injection, 370 MBq (10 mCi)	CBD Subjects n=6 Participants Amyloid negative subjects with CBD receiving a flortaucipir PET scan at baseline and at 9 months. Flortaucipir F18 PET scan: IV injection, 370 MBq (10 mCi)	Healthy Volunteers n=3 Participants Healthy volunteers receiving a flortaucipir PET scan at baseline. Flortaucipir F18 PET scan: IV injection, 370 MBq (10 mCi)	All Subjects n=29 Participants All subjects combined
Flortaucipir Imaging in PSP, CBD and Healthy Volunteers Right Dentate 9 months	1.17 standardized uptake value ratio (SUVr) Standard Deviation 0.11	1.11 standardized uptake value ratio (SUVr) Standard Deviation 0.05	—	1.15 standardized uptake value ratio (SUVr) Standard Deviation 0.10
Flortaucipir Imaging in PSP, CBD and Healthy Volunteers Left Globus Pallidus 9 Months	1.62 standardized uptake value ratio (SUVr) Standard Deviation 0.15	1.62 standardized uptake value ratio (SUVr) Standard Deviation 0.40	—	1.62 standardized uptake value ratio (SUVr) Standard Deviation 0.21
Flortaucipir Imaging in PSP, CBD and Healthy Volunteers Global PSP/CBD Summary Baseline	1.38 standardized uptake value ratio (SUVr) Standard Deviation 0.10	1.29 standardized uptake value ratio (SUVr) Standard Deviation 0.11	1.17 standardized uptake value ratio (SUVr) Standard Deviation 0.03	1.34 standardized uptake value ratio (SUVr) Standard Deviation 0.12
Flortaucipir Imaging in PSP, CBD and Healthy Volunteers Right Dentate Baseline	1.17 standardized uptake value ratio (SUVr) Standard Deviation 0.12	1.12 standardized uptake value ratio (SUVr) Standard Deviation 0.04	1.03 standardized uptake value ratio (SUVr) Standard Deviation 0.12	1.15 standardized uptake value ratio (SUVr) Standard Deviation 0.12
Flortaucipir Imaging in PSP, CBD and Healthy Volunteers Left Dentate Baseline	1.16 standardized uptake value ratio (SUVr) Standard Deviation 0.10	1.09 standardized uptake value ratio (SUVr) Standard Deviation 0.04	1.08 standardized uptake value ratio (SUVr) Standard Deviation 0.07	1.14 standardized uptake value ratio (SUVr) Standard Deviation 0.09
Flortaucipir Imaging in PSP, CBD and Healthy Volunteers Left Dentate 9 months	1.13 standardized uptake value ratio (SUVr) Standard Deviation 0.11	1.08 standardized uptake value ratio (SUVr) Standard Deviation 0.05	—	1.12 standardized uptake value ratio (SUVr) Standard Deviation 0.10
Flortaucipir Imaging in PSP, CBD and Healthy Volunteers Right Globus Pallidus Baseline	1.60 standardized uptake value ratio (SUVr) Standard Deviation 0.15	1.40 standardized uptake value ratio (SUVr) Standard Deviation 0.20	1.29 standardized uptake value ratio (SUVr) Standard Deviation 0.06	1.53 standardized uptake value ratio (SUVr) Standard Deviation 0.19
Flortaucipir Imaging in PSP, CBD and Healthy Volunteers Right Globus Pallidus 9 Months	1.58 standardized uptake value ratio (SUVr) Standard Deviation 0.13	1.47 standardized uptake value ratio (SUVr) Standard Deviation 0.19	—	1.55 standardized uptake value ratio (SUVr) Standard Deviation 0.15
Flortaucipir Imaging in PSP, CBD and Healthy Volunteers Left Globus Pallidus Baseline	1.62 standardized uptake value ratio (SUVr) Standard Deviation 0.15	1.56 standardized uptake value ratio (SUVr) Standard Deviation 0.32	1.30 standardized uptake value ratio (SUVr) Standard Deviation 0.07	1.58 standardized uptake value ratio (SUVr) Standard Deviation 0.21
Flortaucipir Imaging in PSP, CBD and Healthy Volunteers Global PSP/CBD Summary 9 Months	1.37 standardized uptake value ratio (SUVr) Standard Deviation 0.09	1.32 standardized uptake value ratio (SUVr) Standard Deviation 0.13	—	1.36 standardized uptake value ratio (SUVr) Standard Deviation 0.10
Flortaucipir Imaging in PSP, CBD and Healthy Volunteers AD Cortical Baseline	0.99 standardized uptake value ratio (SUVr) Standard Deviation 0.03	0.99 standardized uptake value ratio (SUVr) Standard Deviation 0.06	1.02 standardized uptake value ratio (SUVr) Standard Deviation 0.02	0.99 standardized uptake value ratio (SUVr) Standard Deviation 0.04
Flortaucipir Imaging in PSP, CBD and Healthy Volunteers AD Cortical 9 Months	0.99 standardized uptake value ratio (SUVr) Standard Deviation 0.03	1.00 standardized uptake value ratio (SUVr) Standard Deviation 0.05	—	0.99 standardized uptake value ratio (SUVr) Standard Deviation 0.04

Adverse Events

PSP Subjects

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

CBD Subjects

Serious events: 0 serious events

Other events: 1 other events

Deaths: 1 deaths

Healthy Volunteers

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	PSP Subjects n=20 participants at risk Amyloid negative subjects with Progressive Supranuclear Palsy (PSP) receiving a flortaucipir PET scan	CBD Subjects n=6 participants at risk Amyloid negative subjects with Corticobasal Degeneration (CBD) receiving a flortaucipir PET scan	Healthy Volunteers n=3 participants at risk Healthy volunteers receiving a flortaucipir PET scan
Respiratory, thoracic and mediastinal disorders bronchial secretion retention	5.0% 1/20 • Number of events 1 • End of study for AE reporting was 48 hours after flortaucipir administration at each imaging visit. 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 flortaucipir.	0.00% 0/6 • End of study for AE reporting was 48 hours after flortaucipir administration at each imaging visit. 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 flortaucipir.	0.00% 0/3 • End of study for AE reporting was 48 hours after flortaucipir administration at each imaging visit. 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 flortaucipir.
Musculoskeletal and connective tissue disorders muscle spasms	0.00% 0/20 • End of study for AE reporting was 48 hours after flortaucipir administration at each imaging visit. 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 flortaucipir.	16.7% 1/6 • Number of events 1 • End of study for AE reporting was 48 hours after flortaucipir administration at each imaging visit. 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 flortaucipir.	0.00% 0/3 • End of study for AE reporting was 48 hours after flortaucipir administration at each imaging visit. 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 flortaucipir.
General disorders asthenia	5.0% 1/20 • Number of events 1 • End of study for AE reporting was 48 hours after flortaucipir administration at each imaging visit. 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 flortaucipir.	0.00% 0/6 • End of study for AE reporting was 48 hours after flortaucipir administration at each imaging visit. 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 flortaucipir.	0.00% 0/3 • End of study for AE reporting was 48 hours after flortaucipir administration at each imaging visit. 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 flortaucipir.

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