Brief Title
PROgressive Supranuclear Palsy CorTico-Basal Syndrome Multiple System Atrophy Longitudinal Study UK
Official Title
PROgressive Supranuclear Palsy CorTico-Basal Syndrome Multiple System Atrophy Longitudinal Study UK
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.
Detailed Description
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.
Study Type
Observational [Patient Registry]
Primary Outcome
Survival status after 5 years of clinical follow-up
Secondary Outcome
Annual change in degree of disability in PSP, CBD and APS cases as determined by the PSP rating scale
Condition
Progressive Supranuclear Palsy (PSP)
Study Arms / Comparison Groups
Progressive Supranuclear Palsy
Description: Patients with a current clinical diagnosis of Progressive Supranuclear Palsy (PSP)
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Estimated Enrollment
900
Start Date
October 2014
Completion Date
July 2023
Primary Completion Date
July 2023
Eligibility Criteria
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.
Gender
All
Ages
18 Years - N/A
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Contacts
Huw Morris, PhD, FRCP, 020 310 87462, [email protected]
Location Countries
United Kingdom
Location Countries
United Kingdom
Administrative Informations
NCT ID
NCT02778607
Organization ID
14/0371
Responsible Party
Sponsor
Study Sponsor
University College, London
Collaborators
University of Cambridge
Study Sponsor
Huw Morris, PhD, FRCP, Principal Investigator, University College, London
Verification Date
October 2020