Progressive Supranuclear Palsy (PSP)TANGLESNCT03545126
Human CNS Tau Kinetics in Tauopathies
An observational study of Progressive Supranuclear Palsy (PSP) and Corticobasal Degeneration (CBD), run by Washington University School of Medicine at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Progressive Supranuclear Palsy (PSP), Corticobasal Degeneration (CBD), Frontotemporal Dementia (FTD MAPT Mutation)
- What is being tested?
- 13C6 Leucine
- Type of treatment
- Injection or infusion
- How many people
- 27 participants
- Where
- 1 siteUnited States
- Study dates
- 21 Aug 2017to 4 Mar 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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
Who may be able to join
- Diagnosed with PSP, CBD, or FTD MAPT
Who cannot join
- 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)
Trial locations and contacts
1 site in 1 country.
United States1 site
Washington University in St. Louis School of Medicine
St Louis, Missouri, 63110
About this study
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.
Read the detailed description
Tauopathies are neurodegenerative diseases with tau pathology. These tauopathies are the most common pathology in neurodegenerative diseases, and they are reaching epidemic proportions. The rates of tau kinetics are central to understanding normal and abnormal processing and production and clearance of tau kinetics in humans to help understand the causes of tauopathy and evaluate tau-targeted therapeutics.
This study will utilize the Stable Isotope Labeling Kinetics (SILK) method to elucidate tau kinetics in vivo in the human central nervous system (CNS) and its alteration in tauopathies. A total of ~34 participants from 3 different neurodegenerative diseases: Frontotemporal Dementia (FTD), Corticobasal Degeneration (CBD), and Progressive Supranuclear Palsy (PSP), will be invited to enroll in the study.
Participants will be labeled with stable isotopes via 16hr intravenous infusion and CSF samples collected during subsequent lumbar puncture visits over ~120 days. CSF will be analyzed over time for the quantitation of labeled tau.
- Conditions
- Progressive Supranuclear Palsy (PSP), Corticobasal Degeneration (CBD), Frontotemporal Dementia (FTD MAPT Mutation)
- Being tested
- 13C6 Leucine
- Keywords
- Tauopathies
- Medical subject headings
- Supranuclear Palsy, Progressive, Corticobasal Degeneration, Frontotemporal Dementia, Tauopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
13C6 LeucineOther
Recruited participants will be given 13C6-labeled leucine through intravenous infusion (4mg/kg/hr for 16hrs)
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: 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. Receives: Other: 13C6 Leucine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Tau Fractional Turnover Rate (FTR)
Calculated by using CSF tau labeling and plasma free leucine.
Measured: 6 months
Secondary outcomes (2)
CSF Tau Absolute Concentration
Measured using labeled and unlabeled tau protein isoforms that will be immunoprecipitated and analyzed by mass spectrometry.
Measured: 6 months
Tau Production Rate
Measured by FTR multiplied by CSF tau concentration.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jun 2018
- Study start21 Aug 2017
- Primary completion4 Mar 2022
- Study completion4 Mar 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Progressive Supranuclear Palsy (PSP)Studies of the same condition, open and under way.
- More trials run by Washington University School of MedicineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case-control
- Time perspective
- Prospective
- Enrolment
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- Washington University School of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- Association of Frontotemporal Degeneration, Tau Consortium
- Responsible party
- Sponsor
- Investigators
- Randall Bateman, MD, Principal investigator, Washington University in Saint Louis Medical SchoolNupur Ghoshal, MD, PhD, Principal investigator, Washington University in Saint Louis Medical School
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03545126
- Sponsor's protocol ID
- 201703052
- Acronym
- TANGLES
- Status last verified
- Apr 2022
- First submitted
- 12 Mar 2018
- Last updated
- 13 Apr 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided: Undecided at this time.
Study at a glance
From the registry record, last updated by the sponsor on 13 Apr 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Human CNS Tau Kinetics in Tauopathies
- ClinicalTrials.gov ID
- NCT03545126
- Acronym / sponsor ID
- TANGLES / 201703052
- Conditions
- Progressive Supranuclear Palsy (PSP), Corticobasal Degeneration (CBD), Frontotemporal Dementia (FTD MAPT Mutation)
- Keywords
- Tauopathies
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 27 participants (actual)
- Lead sponsor
- Washington University School of MedicineUniversity, hospital or other
- Collaborators
- Association of Frontotemporal Degeneration, Tau Consortium
- Study start
- 21 Aug 2017 (actual)
- Primary completion
- 4 Mar 2022 (actual)
- Study completion
- 4 Mar 2022 (actual)
- First posted
- 4 Jun 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Tauopathies are neurodegenerative diseases with tau pathology. These tauopathies are the most common pathology in neurodegenerative diseases, and they are reaching epidemic proportions. The rates of tau kinetics are central to understanding normal and abnormal processing and production and clearance of tau kinetics in humans to help understand the causes of tauopathy and evaluate tau-targeted therapeutics.
This study will utilize the Stable Isotope Labeling Kinetics (SILK) method to elucidate tau kinetics in vivo in the human central nervous system (CNS) and its alteration in tauopathies. A total of ~34 participants from 3 different neurodegenerative diseases: Frontotemporal Dementia (FTD), Corticobasal Degeneration (CBD), and Progressive Supranuclear Palsy (PSP), will be invited to enroll in the study.
Participants will be labeled with stable isotopes via 16hr intravenous infusion and CSF samples collected during subsequent lumbar puncture visits over ~120 days. CSF will be analyzed over time for the quantitation of labeled tau.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Progressive Supranuclear Palsy (PSP) | – | Other: 13C6 LeucineN=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. |
| Corticobasal Degeneration (CBD) | – | Other: 13C6 LeucineN=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. |
| Frontotemporal Dementia: MAPT | – | Other: 13C6 LeucineN=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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- 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
Key inclusion criteria (1 listed)
- Diagnosed with PSP, CBD, or FTD MAPT
Key exclusion criteria (6 listed)
- Clotting disorder
- Active anticoagulation therapy
- Active infection
- Meningitis
- Recent syncope
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 13C6 LeucineRecruited participants will be given 13C6-labeled leucine through intravenous infusion (4mg/kg/hr for 16hrs) | Other | Corticobasal Degeneration (CBD), Frontotemporal Dementia: MAPT, Progressive Supranuclear Palsy (PSP) |
Primary outcome
Tau Fractional Turnover Rate (FTR)
Time frame: 6 months
Calculated by using CSF tau labeling and plasma free leucine.
2 secondary outcomes
CSF Tau Absolute Concentration
Time frame: 6 months
Tau Production Rate
Time frame: 6 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Apr 2022.
- Individual participant dataSharing planned: Undecided. Undecided at this time.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Randall Bateman, MD principal investigator
Washington University in Saint Louis Medical School
Nupur Ghoshal, MD, PhD principal investigator
Washington University in Saint Louis Medical School
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Progressive Supranuclear Palsy (PSP) and Corticobasal Degeneration (CBD), run by Washington University School of Medicine at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 Apr 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Tau Fractional Turnover Rate (FTR) (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Apr 2022.