NCT03545126

Brief Summary

The goal of this study is to characterize tau kinetics and tau aggregation in the human CNS and to test the hypothesis that tau kinetics are altered (i.e. increased production, decreased clearance, and increased aggregation rate) in tauopathies.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
27

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Aug 2017

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 21, 2017

Completed
7 months until next milestone

First Submitted

Initial submission to the registry

March 12, 2018

Completed
3 months until next milestone

First Posted

Study publicly available on registry

June 4, 2018

Completed
3.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 4, 2022

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 4, 2022

Completed
Last Updated

April 13, 2022

Status Verified

April 1, 2022

Enrollment Period

4.5 years

First QC Date

March 12, 2018

Last Update Submit

April 12, 2022

Conditions

Progressive Supranuclear Palsy (PSP)Corticobasal Degeneration (CBD)Frontotemporal Dementia (FTD MAPT Mutation)

Keywords

Tauopathies

Outcome Measures

Primary Outcomes (1)

  • Tau Fractional Turnover Rate (FTR)

    Calculated by using CSF tau labeling and plasma free leucine.

    6 months

Secondary Outcomes (2)

  • CSF Tau Absolute Concentration

    6 months

  • Tau Production Rate

    6 months

Study Arms (3)

Progressive Supranuclear Palsy (PSP)

N=12 Age: 18 and older Recruited participants will be given 13C6 Leucine through intravenous infusion (4mg/kg/hr for 16hrs), and CSF will be collected five times over 120 days (on approximately days 1-4, 5-10, 11-20, 21-60 and 61-120) after labeling.

Other: 13C6 Leucine

Corticobasal Degeneration (CBD)

N=8 Age: 18 and older Recruited participants will be given 13C6 Leucine through intravenous infusion (4mg/kg/hr for 16hrs), and CSF will be collected five times over 120 days (on approximately days 1-4, 5-10, 11-20, 21-60 and 61-120) after labeling.

Other: 13C6 Leucine

Frontotemporal Dementia: MAPT

N=12 Family members with or at-risk of tau mutations (e.g. P301L) Age: 18 and older Recruited participants will be given 13C6 Leucine through intravenous infusion (4mg/kg/hr for 16hrs), and CSF will be collected five times over 120 days (on approximately days 1-4, 5-10, 11-20, 21-60 and 61-120) after labeling.

Other: 13C6 Leucine

Interventions

13C6 LeucineOTHER

Recruited participants will be given 13C6-labeled leucine through intravenous infusion (4mg/kg/hr for 16hrs)

Corticobasal Degeneration (CBD)Frontotemporal Dementia: MAPTProgressive Supranuclear Palsy (PSP)

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Progressive Supranuclear Palsy (PSP): N=12 Corticobasal Degeneration (CBD): N=8 Frontotemporal Dementia (FTD): MAPT: Family members with or at-risk of tau mutations (e.g. P301L) N=12

You may qualify if:

  • Diagnosed with PSP, CBD, or FTD MAPT

You may not qualify if:

  • Clotting disorder
  • Active anticoagulation therapy
  • Active infection
  • Meningitis
  • Recent syncope
  • Current experimental treatment targeting Aβ or medications thought to influence Aβ production or clearance rates (benzodiazepines, muscarinic agents, or anti-epileptics)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Washington University in St. Louis School of Medicine

St Louis, Missouri, 63110, United States

Location

Biospecimen

Retention: SAMPLES WITHOUT DNA

Plasma CSF

MeSH Terms

Conditions

Supranuclear Palsy, ProgressiveCorticobasal DegenerationFrontotemporal DementiaTauopathies

Condition Hierarchy (Ancestors)

Basal Ganglia DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesMovement DisordersOphthalmoplegiaOcular Motility DisordersCranial Nerve DiseasesNeurodegenerative DiseasesParalysisNeurologic ManifestationsEye DiseasesSigns and SymptomsPathological Conditions, Signs and SymptomsFrontotemporal Lobar DegenerationDementiaTDP-43 ProteinopathiesProteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic DiseasesNeurocognitive DisordersMental Disorders

Study Officials

  • Randall Bateman, MD

    Washington University in Saint Louis Medical School

    PRINCIPAL INVESTIGATOR

  • Nupur Ghoshal, MD, PhD

    Washington University in Saint Louis Medical School

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
CASE CONTROL
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 12, 2018

First Posted

June 4, 2018

Study Start

August 21, 2017

Primary Completion

March 4, 2022

Study Completion

March 4, 2022

Last Updated

April 13, 2022

Record last verified: 2022-04

Locations

US(1)