NCT03452956

Brief Summary

This is an observational study that aims to better understand the genetic causes of frontotemporal degeneration (FTD), Multiple Systems Atrophy (MSA), and Progressive Supranuclear Palsy (PSP). It is hoped the information gathered in this study will help lead to better diagnostics and future treatments.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
997

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Jul 2003

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

July 1, 2003

Completed
14.7 years until next milestone

First Submitted

Initial submission to the registry

February 26, 2018

Completed
4 days until next milestone

First Posted

Study publicly available on registry

March 2, 2018

Completed
2.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 15, 2020

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 15, 2020

Completed
Last Updated

November 12, 2020

Status Verified

November 1, 2020

Enrollment Period

17.1 years

First QC Date

February 26, 2018

Last Update Submit

November 10, 2020

Conditions

Frontotemporal DegenerationProgressive Supranuclear PalsyMultiple System Atrophy

Outcome Measures

Primary Outcomes (1)

  • Progression

    Changes over time in MRI and cognitive testing data.

    This is a natural history study - participants are followed from date of enrollment until death, withdrawl, or funding is no longer available, or until 984 months have passed

Secondary Outcomes (1)

  • Impaired semantic memory in Semantic Dementia (SD).

    This is a natural history study - participants are followed from date of enrollment until death, withdrawl, or funding is no longer available, or until 984 months have passed

Other Outcomes (2)

  • Effortful speech in Progressive Nonfluent Aphasia (PNFA)

    This is a natural history study - participants are followed from date of enrollment until death, withdrawl, or funding is no longer available, or until 984 months have passed

  • Social disinhibition and rule violation in FTD

    This is a natural history study - participants are followed from date of enrollment until death, withdrawl, or funding is no longer available, or until 984 months have passed

Study Arms (1)

FTD Cohort

No intervention-observation only

Other: None-Observation Only

Interventions

None-Observation OnlyOTHER

No intervention-this is an observational study

FTD Cohort

Eligibility Criteria

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

All individuals with a diagnosis of FTD, PSP, or MSA, previously identified by the PI and sub-investigators through other FTD studies (protocol number 702681) and through the PI's extensive FTD clinical practice at the University of Pennsylvania and at Pennsylvania Hospital's Department of Neurology, will be recruited through letters, brochures, and study flyers.

You may qualify if:

  • Individuals who have been diagnosed with FTD, PSP, and MSA

You may not qualify if:

  • Individuals under 18 years of age
  • People with pacemakers or certain metallic implants
  • pregnant women

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

Biospecimen

Retention: SAMPLES WITH DNA

Cerebral Spinal Fluid (CSF) and blood samples will both be taken and stored for DNA and RNA extraction and analysis and University of Pennsylvania Center for Neurodegenertive Research Center (CNDR).

MeSH Terms

Conditions

Supranuclear Palsy, ProgressiveMultiple System Atrophy

Condition Hierarchy (Ancestors)

Basal Ganglia DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesMovement DisordersOphthalmoplegiaOcular Motility DisordersCranial Nerve DiseasesTauopathiesNeurodegenerative DiseasesParalysisNeurologic ManifestationsEye DiseasesSigns and SymptomsPathological Conditions, Signs and SymptomsPrimary DysautonomiasAutonomic Nervous System DiseasesSynucleinopathies

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
RETROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 26, 2018

First Posted

March 2, 2018

Study Start

July 1, 2003

Primary Completion

July 15, 2020

Study Completion

July 15, 2020

Last Updated

November 12, 2020

Record last verified: 2020-11

Data Sharing

IPD Sharing
Will share

The University of Pennsylvania is a participant in the National Alzheimer's Coordinating Center 's FTLD-NACC initiative to create a national database of data specific to FTD spectrum diseases.The FTLD NACC initiative will enable research centers focusing on FTD spectrum diseases to gather standardized data on their FTLD patients, all of which will be combined into a single database and made freely available to researchers around the world. The FTLD-NACC collects data standardly obtained in this study, including neurocognitive assessments, MRI imaging data, and biomarkers collected through lumbar puncture and blood draw.The FTLD-NACC is designed to facilitate collaborative research and will allow researchers to maximize the use of clinical information and biological material available on frontotemporal degenerative spectrum diseases. Subjects will be able to choose whether or not their research data is shared with the FTD-NACC.

Time Frame
As this is an ongoing, longitudinal study, IPD will be available through the duration of the study.

Locations

US(1)