PROgressive Supranuclear Palsy CorTico-Basal Syndrome Multiple System Atrophy Longitudinal Study UK

NCT02778607 | Unknown

• 1 other identifier: 14/0371
• Study Type: observational
• Target: 900
• Locations: 1 country
• Sites: 1

Brief Summary

Progressive Supranuclear Palsy (PSP), Cortico-Basal Degeneration (CBD) and Multiple System Atrophy (MSA) are degenerative brain conditions for which there are currently no curative treatments. To aid the development of new treatment trials, there is a pressing need to develop better methods for diagnosing these conditions early, and to track disease progression. The PROSPECT-M-UK study will collect standardised clinical data over time. Patients will also have the option to have a brain MRI scan, eye movement exam and donate blood, skin and spinal fluid samples, with the aim to identify "biomarkers" that can improve the accuracy of early diagnosis and track the natural time course of disease. Control participants and those not meeting criteria for Parkinson's disease or other defined conditions but are considered by the investigator group to be allied syndromes or at risk states (atypical parkinsonian syndromes), will also be examined. Patients can also participate via the CBD European registry or in a one-off study assessment through the cross-sectional study, which involves completing questionnaires and a blood sample donation.

Conditions

Progressive Supranuclear Palsy (PSP); Corticobasal Degeneration; Multiple System Atrophy (MSA)

Keywords

Atypical Parkinsonism Syndrome (APS)

Outcome Measures

Primary Outcomes (1)

• Survival status after 5 years of clinical follow-up

  To determine patient survival status after 5 years of follow-up for survival analysis using the Kaplan-Meier Method

  5 years

Secondary Outcomes (2)

• Annual change in degree of disability in PSP, CBD and APS cases as determined by the PSP rating scale

  3 years

• Annual change in degree of disability in MSA cases as determined by the Unified Multiple System Atrophy Rating Scale (UMSARS)

  3 years

Other Outcomes (5)

• CSF biomarkers

  1 year

• Brain MRI

  1 year

• MoCA Cognitive function test

  3 years

Study Arms (5)

Progressive Supranuclear Palsy

Patients with a current clinical diagnosis of Progressive Supranuclear Palsy (PSP)

Multiple System Atrophy

Patients with current clinical diagnosis of Multiple System Atrophy (MSA).

Atypical Parkinsonian Syndrome

Atypical Parkinsonian Syndrome (APS) patients who do not fulfil existing criteria for PSP/CBD/MSA, but may represent variant clinical syndromes related to tau pathology including pure akinesia with gait freezing (PAGF), PSP-parkinsonism, overlap syndromes and atypical parkinsonian disorders not meeting clinical diagnostic criteria at entry

Controls

Participants unaffected by neurological or psychiatric disease

Corticobasal Degeneration

Patients with a current clinical diagnosis of Corticobasal Degeneration (CBD)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patient recruitment for the natural history and longitudinal study arms will be through identification of patients by their physicians. The patients will have been referred or will be receiving treatment at a movement disorders, neurology or medical clinic. Patient recruitment for the cross-sectional study will be i.) through identification of patients attending relevant specialist neurology clinics ii.) through information placed on patient organization websites, PSP Association and MSA Trust, and iii.) patients with CBD/CBS can also be recruited via the British Neurological Surveillance Unit (BNSU).

You may qualify if:

• \. Written informed consent obtained prior to any study-related procedures. A consultee process will be used where participants lack the mental capacity for consent, either due to cognitive or communication deficits.
• \. Fulfills clinical criteria (PSP, MSA, CBD/CBS) or clinically defined allied disorders (at-risk states or intermediate disorders, as above) or a healthy control participant recruited from local volunteer databases or next of kin where they have expressed a wish to participate.
• \. Participant is 18 years old or older.
• \. Participant has an identified informant.

You may not qualify if:

• \. Participant has another significant medical or psychiatric illness that would interfere in completing assessments
• \. Participant is pregnant.

Sponsors & Collaborators

• University College, London: lead
• University of Cambridge: collaborator
• University of Oxford: collaborator
• University of Manchester: collaborator
• Newcastle University: collaborator
• University of Sussex: collaborator
• Royal Gwent Hospital: collaborator

Study Sites (1)

University College London Hospitals

London, United Kingdom

RECRUITING

Biospecimen

Retention: SAMPLES WITH DNA

1. Blood sample collection for: DNA extraction, Peripheral blood lymphocyte storage, plasma, serum and RNA storage. 2. Brain MRI scan 3. Skin biopsy 4. Cerebro-spinal fluid

MeSH Terms

Conditions

Supranuclear Palsy, Progressive; Corticobasal Degeneration; Multiple System Atrophy

Condition Hierarchy (Ancestors)

Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Ophthalmoplegia; Ocular Motility Disorders; Cranial Nerve Diseases; Tauopathies; Neurodegenerative Diseases; Paralysis; Neurologic Manifestations; Eye Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Primary Dysautonomias; Autonomic Nervous System Diseases; Synucleinopathies

Study Officials

• Huw Morris, PhD, FRCP

  University College, London

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Alyssa Costantini, MSc

CONTACT

020 310 87462; prospect@ucl.ac.uk

Huw Morris, PhD, FRCP

CONTACT

h.morris@ucl.ac.uk

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: PROSPECTIVE
• Target Duration: 5 Years
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 22, 2016
• First Posted: May 20, 2016
• Study Start: October 1, 2014
• Primary Completion: July 1, 2023
• Study Completion: July 1, 2023
• Last Updated: October 19, 2020

Record last verified: 2020-10

Data Sharing

• IPD Sharing: Will share

Locations

GB (1)