Tau PET Imaging With 18F-AV-1451 in Subjects With MAPT Mutations
Study Details
Study Description
Brief Summary
The study will investigate the ability of a new PET tracer, 18F-AV-1451, to detect depositions of a protein, called tau, in the brains of people with a mutation in the tau gene that causes deposition of the protein, and in people without the mutation. Up to three 18F-AV-1451 scans will be performed (one per year) on control subjects without MAPT mutations, presymptomatic mutation carriers, and symptomatic mutation carriers.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 2 |
Detailed Description
Approximately 40% of cases of Frontotemporal lobar Degeneration (FTLD) are also associated with abnormal deposition of tau protein. The purpose of this study is to image MAPT mutation carriers and their non-carrier relatives in order to study the use of this tracer as a biomarker in Frontotemporal Lobar Degeneration with tau deposition (FTLD-tau).
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: 18F-AV-1451 Subjects who are microtubule associated protein tau (MAPT) family carriers and non-carriers will receive 18F-AV-1451 by injection, and undergo a Positron Emission Tomography (PET) scan, which will then be qualitatively analyzed to examine tau deposition in the brain. |
Drug: 18F-AV-1451
A single injection of up to 10 millicuries of 18F-AV-1451 will be administered to subjects, followed by a 20-minute PET scan.
Other Names:
|
Outcome Measures
Primary Outcome Measures
- SUVR of 18F-AV-1451 [Baseline, 12-month follow up]
Regional tau deposition will be measured as standardized uptake value ratio (SUVR) of 18F-AV-1451. SUVR (80-100 min post-injection) for 18F-AV-1451 will be calculated two ways: 1) using cerebellar crus as a reference region, and 2) using the Parametric Estimation of Reference Signal Intensity (PERSI) method to create individual white matter reference regions. Binding in the inferior temporal lobe/cortex was used as the primary outcome.
Eligibility Criteria
Criteria
Inclusion Criteria:
- Members of families with established MAPT mutations, who either have the capacity to consent to participate in the protocol, or else have designated a surrogate/proxy to consent to participate in this study
Exclusion Criteria:
-
Unwillingness to participate
-
Usage of medication which significantly prolongs QT interval
-
Pregnancy or plans for pregnancy within 90 days after participating in study
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Morton A. Kreitchman PET Center | New York | New York | United States | 10032 |
Sponsors and Collaborators
- Columbia University
- National Institute of Neurological Disorders and Stroke (NINDS)
Investigators
- Principal Investigator: Edward Huey, MD, Columbia University
Study Documents (Full-Text)
More Information
Publications
None provided.- AAAP4551
- R01NS076837
Study Results
Participant Flow
| Recruitment Details | |
|---|---|
| Pre-assignment Detail |
| Arm/Group Title | 18F-AV-1451 |
|---|---|
| Arm/Group Description | Subjects will receive 18F-AV-1451 by injection, and undergo a Positron Emission Tomography (PET) scan, which will then be qualitatively analyzed to examine tau deposition in the brain. 18F-AV-1451: A single injection of up to 10 millicuries of 18F-AV-1451 will be administered to subjects, followed by a 20-minute PET scan. |
| Period Title: Overall Study | |
| STARTED | 7 |
| COMPLETED | 7 |
| NOT COMPLETED | 0 |
Baseline Characteristics
| Arm/Group Title | 18F-AV-1451 |
|---|---|
| Arm/Group Description | Subjects will receive 18F-AV-1451 by injection, and undergo a Positron Emission Tomography (PET) scan, which will then be qualitatively analyzed to examine tau deposition in the brain. 18F-AV-1451: A single injection of up to 10 millicuries of 18F-AV-1451 will be administered to subjects, followed by a 20-minute PET scan. |
| Overall Participants | 7 |
| Age, Customized (Count of Participants) | |
| 18-21 years |
0
0%
|
| 22-29 years |
1
14.3%
|
| 30-39 years |
1
14.3%
|
| 40-49 years |
0
0%
|
| 50-59 years |
4
57.1%
|
| 60-69 years |
1
14.3%
|
| Sex: Female, Male (Count of Participants) | |
| Female |
5
71.4%
|
| Male |
2
28.6%
|
| Ethnicity (NIH/OMB) (Count of Participants) | |
| Hispanic or Latino |
0
0%
|
| Not Hispanic or Latino |
7
100%
|
| Unknown or Not Reported |
0
0%
|
| Race (NIH/OMB) (Count of Participants) | |
| American Indian or Alaska Native |
0
0%
|
| Asian |
0
0%
|
| Native Hawaiian or Other Pacific Islander |
0
0%
|
| Black or African American |
0
0%
|
| White |
7
100%
|
| More than one race |
0
0%
|
| Unknown or Not Reported |
0
0%
|
| Region of Enrollment (participants) [Number] | |
| United States |
7
100%
|
Outcome Measures
| Title | SUVR of 18F-AV-1451 |
|---|---|
| Description | Regional tau deposition will be measured as standardized uptake value ratio (SUVR) of 18F-AV-1451. SUVR (80-100 min post-injection) for 18F-AV-1451 will be calculated two ways: 1) using cerebellar crus as a reference region, and 2) using the Parametric Estimation of Reference Signal Intensity (PERSI) method to create individual white matter reference regions. Binding in the inferior temporal lobe/cortex was used as the primary outcome. |
| Time Frame | Baseline, 12-month follow up |
Outcome Measure Data
| Analysis Population Description |
|---|
| 5 out of 7 subjects had data collected and analyzed (evaluable repeat imaging after 12 months). |
| Arm/Group Title | 18F-AV-1451 - Non-carriers | 18F-AV-1451 - Carriers |
|---|---|---|
| Arm/Group Description | Subjects who are microtubule associated protein tau (MAPT) family non-carriers will receive 18F-AV-1451 by injection, and undergo a Positron Emission Tomography (PET) scan, which will then be qualitatively analyzed to examine tau deposition in the brain. 18F-AV-1451: A single injection of up to 10 millicuries of 18F-AV-1451 will be administered to subjects, followed by a 20-minute PET scan. | Subjects who are MAPT family carriers will receive 18F-AV-1451 by injection, and undergo a Positron Emission Tomography (PET) scan, which will then be qualitatively analyzed to examine tau deposition in the brain. 18F-AV-1451: A single injection of up to 10 millicuries of 18F-AV-1451 will be administered to subjects, followed by a 20-minute PET scan. |
| Measure Participants | 2 | 3 |
| Inferior temporal cortex (Cerebellar crus) |
1.07
|
1.26
|
| Inferior temporal cortex (PERSI) |
1.03
|
1.34
|
Adverse Events
| Time Frame | Up to 2 years | |
|---|---|---|
| Adverse Event Reporting Description | ||
| Arm/Group Title | 18F-AV-1451 | |
| Arm/Group Description | Subjects will receive 18F-AV-1451 by injection, and undergo a Positron Emission Tomography (PET) scan, which will then be qualitatively analyzed to examine tau deposition in the brain. 18F-AV-1451: A single injection of up to 10 millicuries of 18F-AV-1451 will be administered to subjects, followed by a 20-minute PET scan. | |
All Cause Mortality |
||
| 18F-AV-1451 | ||
| Affected / at Risk (%) | # Events | |
| Total | 0/7 (0%) | |
Serious Adverse Events |
||
| 18F-AV-1451 | ||
| Affected / at Risk (%) | # Events | |
| Total | 0/7 (0%) | |
Other (Not Including Serious) Adverse Events |
||
| 18F-AV-1451 | ||
| Affected / at Risk (%) | # Events | |
| Total | 4/7 (57.1%) | |
| General disorders | ||
| Insomnia | 2/7 (28.6%) | 5 |
| Headache | 2/7 (28.6%) | 5 |
Limitations/Caveats
More Information
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
| Name/Title | William Kreisl, MD |
|---|---|
| Organization | Columbia University |
| Phone | 212-342-2380 |
| wck2107@cumc.columbia.edu |
- AAAP4551
- R01NS076837