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

NCT ID: NCT02778607

Last Updated: 2020-10-19

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Total Enrollment

900 participants

Study Classification

OBSERVATIONAL

Study Start Date

2014-10-31

Study Completion Date

2023-07-31

Brief Summary

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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.

Detailed Description

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There are a group of neurodegenerative disorders which are often initially diagnosed to be Parkinson's disease (PD), but which are biologically and clinically distinct, and follow a malignant disease course. The three most common conditions are PSP, CBD and MSA. These conditions have a median survival of approximately 6-7 years and unlike PD, do not respond well to dopamine replacement therapy.

PSP and CBD are characterized by tau-pathology and MSA by alpha-synuclein pathology. A great deal of pre-clinical work has been carried out on tau and alpha-synuclein disease models, yet there are no disease modifying agents for these conditions. There are a number of potential therapeutic compounds in development and in order to improve the likelihood of their success, there is a pressing need to increase the number of early case patients recruited into these new treatment trails. Thus, better methods for improved accuracy of early diagnosis and for tracking progression need to be developed. This can be achieved through:

1. a detailed study of the change in patients' clinical state over time;
2. studying "biomarkers" such as blood, skin, spinal fluid and brain MRI.

The investigators will recruit patients with PSP, CBD and MSA who are referred to specialist clinics for assessment and treatment. An additional group of Atypical parkinsonian syndrome (APS) cases who do not meet criteria for Parkinson's disease or other defined conditions, but are considered by the investigator group to be allied syndromes or at risk states will also be invited to participate in the study. People unaffected by neurological disease will be invited to participate on a one-off occasion.

Being involved in the PROSPECT-M-UK longitudinal study will involve attending a research assessment on 5 occasions over 3 years in our natural history cohort, and for 2 occasions over 2 years for our longitudinal cohort. Study procedures consist of: having a neurological examination; completing questionnaires to provide details of clinical history, self/carer reported functional scales and quality of life; neuropsychology assessment; eye movement exam; donating blood and skin samples; some patients will be invited to have a lumbar puncture for spinal fluid collection and have a brain MRI scan on two occasions (at baseline and after 1 year follow-up). Patients can also agree to be contacted by phone or at a clinic appointment for remote monitoring of symptoms after face to face visits have completed.

In addition, a cross-sectional cohort will be established, to enable participation of patients who cannot travel to a study centre. This will involve donating blood samples,returning study questionnaires, and being monitored remotely. A CBD European registry will also be created which will involve a structured neurological assessment, a medical notes review and blood sample donation.

The primary outcome for the study is duration of disease, with the aim to improve methods for early diagnosis and tracking disease progression. Importantly, the study will link together centres and researchers from across Europe to establish the infrastructure and create a trial ready cohort for future therapeutic study into PSP/CBD/MSA.

Conditions

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

Study Design

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Observational Model Type

CASE_CONTROL

Study Time Perspective

PROSPECTIVE

Study Groups

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Progressive Supranuclear Palsy

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

No interventions assigned to this group

Multiple System Atrophy

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

No interventions assigned to this group

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

No interventions assigned to this group

Controls

Participants unaffected by neurological or psychiatric disease

No interventions assigned to this group

Corticobasal Degeneration

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

No interventions assigned to this group

Eligibility Criteria

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Inclusion Criteria

* 1\. 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.
* 2\. 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.
* 3\. Participant is 18 years old or older.
* 4\. Participant has an identified informant.

Exclusion Criteria

* 1\. Participant has another significant medical or psychiatric illness that would interfere in completing assessments
* 2\. Participant is pregnant.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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University of Cambridge

OTHER

Sponsor Role collaborator

University of Oxford

OTHER

Sponsor Role collaborator

University of Manchester

OTHER

Sponsor Role collaborator

Newcastle University

OTHER

Sponsor Role collaborator

University of Sussex

OTHER

Sponsor Role collaborator

Royal Gwent Hospital

UNKNOWN

Sponsor Role collaborator

University College, London

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Huw Morris, PhD, FRCP

Role: PRINCIPAL_INVESTIGATOR

University College, London

Locations

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University College London Hospitals

London, , United Kingdom

Site Status RECRUITING

Countries

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United Kingdom

Central Contacts

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Alyssa Costantini, MSc

Role: CONTACT

020 310 87462

Huw Morris, PhD, FRCP

Role: CONTACT

Facility Contacts

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Alyssa Costantini, MSc

Role: primary

020 310 87462

Other Identifiers

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14/0371

Identifier Type: -

Identifier Source: org_study_id

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