UPenn Observational Research Repository on Neurodegenerative Disease (UNICORN)

• ClinicalTrials.gov Identifier: NCT04715399
• Recruitment Status: Recruiting
• First Posted: January 20, 2021
• Last Update Posted: January 17, 2023
• Sponsor: University of Pennsylvania
• Collaborators: National Institutes of Health (NIH); Alzheimer's Association; United States Department of Defense; National Institute on Aging (NIA)
• Information provided by (Responsible Party): Murray Grossman, University of Pennsylvania

Tracking Information

• First Submitted Date: January 11, 2021
• First Posted Date: January 20, 2021
• Last Update Posted Date: January 17, 2023
• Actual Study Start Date: May 29, 2020
• Estimated Primary Completion Date: May 30, 2070 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: January 16, 2021)

Status of progression - changes in neuropsychological tests. [ Time Frame: This is a natural history study-participants are followed from date of enrollment until death, withdraw, or funding is no longer available, or until 600 months have passed. ]

The changes of neuropsychological tests (well known cognitive measures such as: Naming test, CVLT, MoCA, Oral Trails, etc.) in neurodegenerative diseases over time.

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: July 9, 2022)

• Status of progression - changes in language processing. [ Time Frame: This is a natural history study-participants are followed from date of enrollment until death, withdraw, or funding is no longer available, or until 600 months have passed. ]
  The effect of changes in language processing (e.g. effortful speech, lexical-semantic representations) as categorized by impaired speech fluency, motor speech deficits and semantic memory deficits in neurodegenerative diseases over time.
• Status of progression - changes in social disinhibition. [ Time Frame: This is a natural history study-participants are followed from date of enrollment until death, withdraw, or funding is no longer available, or until 600 months have passed. ]
  The effect of changes in social behavioral testing, which categorized by rule violation (social disinhibition) progression in neurodegenerative diseases over time.
• Status of progression - changes in biofluids. [ Time Frame: This is a natural history study-participants are followed from date of enrollment until death, withdraw, or funding is no longer available, or until 600 months have passed. ]
  The change of disease status as categorized by biomarkers in biofluids in neurodegenerative diseases over time.
• Status of progression -changes in Neuroimaging [ Time Frame: This is a natural history study-participants are followed from date of enrollment until death, withdraw, or funding is no longer available, or until 600 months have passed. ]
  The progressive changes of images in multimodal neuroimaging techniques in neurodegenerative diseases over time.

Original Secondary Outcome Measures (submitted: January 16, 2021)

• Status of progression - changes in language processing. [ Time Frame: This is a natural history study-participants are followed from date of enrollment until death, withdraw, or funding is no longer available, or until 600 months have passed. ]
  The effect of changes in language processing (effortful speech) as categorized by impaired semantic memory in neurodegenerative diseases over time.
• Status of progression - changes in social disinhibition. [ Time Frame: This is a natural history study-participants are followed from date of enrollment until death, withdraw, or funding is no longer available, or until 600 months have passed. ]
  The effect of changes in social behavioral testing, which categorized by rule violation (social disinhibition) progression in neurodegenerative diseases over time.
• Status of progression - changes in biofluids. [ Time Frame: This is a natural history study-participants are followed from date of enrollment until death, withdraw, or funding is no longer available, or until 600 months have passed. ]
  The change of disease status as categorized by biomarkers in biofluids in neurodegenerative diseases over time.
• Status of progression -changes in Neuroimaging [ Time Frame: This is a natural history study-participants are followed from date of enrollment until death, withdraw, or funding is no longer available, or until 600 months have passed. ]
  The progressive changes of images in multimodal neuroimaging techniques in neurodegenerative diseases over time.

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: UPenn Observational Research Repository on Neurodegenerative Disease
• Official Title: University of Pennsylvania Centralized Observational Research Repository on Neurodegenerative Disease (UNICORN)
• Brief Summary: The aim of this study is to create a repository of both cross-sectional and longitudinal data, including cognitive, linguistic, imaging and biofluid biological specimens, for neurodegenerative disease research and treatment.
• Detailed Description: The Principal Investigator (PI) at the University of Pennsylvania seeks to better understand neurodegenerative diseases and is continually expanding research efforts and collaborations regarding the factors which may contribute to these illnesses. Investigators seek to better understand the basis of neurodegenerative conditions by creating a multimodal repository, including: clinical data such as demographic characteristics, vital signs and motor scales; cognitive and speech data; neuroimaging data; and biological specimens with associated biofluid biomarkers and genetic data. Investigators pursue acquiring these data from neurodegenerative disease patients, people at risk for neurodegenerative disease due to a family history, and unaffected adults. Targeted conditions include frontotemporal degeneration (FTD), primary progressive aphasia PPA), amyotrophic lateral sclerosis (ALS), progressive supranuclear palsy (PSP), corticobasal syndrome (CBS), familial frontotemporal lobar degeneration (fFTLD), non-amnestic variants of Alzheimer's disease including logopenic progressive aphasia and posterior cortical atrophy, and Lewy body disease. This study aims to collect clinical and cognitive data, imaging data, and biospecimen samples from people whose background can inform research and treatment for neurodegenerative diseases, and make these samples and data available to qualified researchers at the University of Pennsylvania and collaborating academic centers and industry partners.
• Study Type: Observational
• Study Design: Observational Model: Cohort; Time Perspective: Other
• Target Follow-Up Duration: Not Provided

Biospecimen

Retention:   Samples With DNA

Description:

Blood samples, cerebrospinal fluid (CSF) samples, and saliva or buccal samples may be taken and stored for DNA, RNA and protein extraction and analysis at the University of Pennsylvania and/or research collaborators elsewhere.

• Sampling Method: Probability Sample
• Study Population: To better understand neurodegenerative diseases, the investigators will include people living with these illnesses, people at risk of developing these illnesses, and people without neurodegenerative disease who wish to contribute to science as controls.

Condition

• Frontotemporal Degeneration(FTD)
• Primary Progressive Aphasia(PPA)
• Familial Frontotemporal Lobar Degeneration (fFTLD)
• Amyotrophic Lateral Sclerosis(ALS)
• Lewy Body Disease(LBD)
• Progressive Supranuclear Palsy(PSP)
• Corticobasal Syndrome(CBS)
• Posterior Cortical Atrophy(PCA)
• Alzheimer's Disease(AD)

Intervention

Other: No intervention

No intervention

Study Groups/Cohorts

• Cross-sectional
  Intervention: Other: No intervention
• Longitudinal
  Intervention: Other: No intervention

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: January 16, 2021): 1000
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: May 30, 2070
• Estimated Primary Completion Date: May 30, 2070 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

This protocol will include 3 groups of people:

1. People with a clinical diagnosis of a neurodegenerative disease. such as frontotemporal degeneration(FTD), primary progressive aphasia(PPA), Lewy body disease(LBD), amyotrophic lateral sclerosis(ALS), progressive supranuclear palsy(PSP), corticobasal syndrome(CBS), posterior cortical atrophy(PCA), Alzheimer's disease(AD), Parkinson's disease(PD)
2. People with a family history of neurodegenerative disease who may or may not be symptomatic, and may or may not be mutation carriers such as familial frontotemporal lobar degeneration (fFTLD) or familial ALS,
3. People with no known neurological disease who will provide control data.

Exclusion Criteria:

• Anyone who is under the age of 18.
• Anyone with a condition or in a situation which, in the Investigator's opinion, could confound the study findings or may interfere significantly with a person's participation, including but not limited to neurological, psychological and other medical conditions (such as cardiac, neurosurgical, infectious conditions).
• Individual participants may be excluded from some, but not all, study procedures for safety reasons when they have a contraindication or at the discretion of the Investigator. For example, persons with metal implants which are not MRI-safe will not be able to take part in imaging, and those on blood thinning medications may not be able to take part in lumbar puncture.
• Pregnant women; if a woman becomes pregnant during the study, research activities that may increase risk to the patient and the unborn fetus will be stopped until the end of pregnancy, at which point participation can be resumed.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: Yes

Contacts

Contact: Gillian Bradley; 215 349-5725; gillian.bradley@pennmedicine.upenn.edu

Contact: Salima Shah; salima.shah@pennmedicine.upenn.edu

• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT04715399
• Other Study ID Numbers: 842873; R01AG054519 ( U.S. NIH Grant/Contract ); P01AG066597 ( U.S. NIH Grant/Contract )
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No
• Plan Description: Information about study participants will be kept confidential and managed according to the requirements of the Health Insurance Portability and Accountability Act of 1996 (HIPAA). Only deidentified data will be shared. We will not share individual participant identifiable data with other researchers outside of the institution, unless the participant allows us to by signing a separate data release form.

• Current Responsible Party: Murray Grossman, University of Pennsylvania
• Original Responsible Party: Murray Grossman, University of Pennsylvania, Professor; Director, Penn FTD Center; Chief, Cognitive Neurology Division
• Current Study Sponsor: University of Pennsylvania
• Original Study Sponsor: Same as current

Collaborators

• National Institutes of Health (NIH)
• Alzheimer's Association
• United States Department of Defense
• National Institute on Aging (NIA)

Investigators

• Principal Investigator: David Irwin, MD; University of Pennsylvania

• PRS Account: University of Pennsylvania
• Verification Date: January 2023