- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02778607
PROgressive Supranuclear Palsy CorTico-Basal Syndrome Multiple System Atrophy Longitudinal Study UK (PROSPECT-M)
Study Overview
Status
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:
- a detailed study of the change in patients' clinical state over time;
- 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
Enrollment (Anticipated)
Contacts and Locations
Study Contact
- Name: Alyssa Costantini, MSc
- Phone Number: 020 310 87462
- Email: prospect@ucl.ac.uk
Study Contact Backup
- Name: Huw Morris, PhD, FRCP
- Email: h.morris@ucl.ac.uk
Study Locations
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-
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London, United Kingdom
- Recruiting
- University College London Hospitals
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Principal Investigator:
- Huw Morris, PhD, FRCP
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Contact:
- Alyssa Costantini, MSc
- Phone Number: 020 310 87462
- Email: prospect@ucl.ac.uk
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Sampling Method
Study Population
Description
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.
Study Plan
How is the study designed?
Design Details
- Observational Models: Case-Control
- Time Perspectives: Prospective
Cohorts and Interventions
Group / Cohort |
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Progressive Supranuclear Palsy
Patients with a current clinical diagnosis of Progressive Supranuclear Palsy (PSP)
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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
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Corticobasal Degeneration
Patients with a current clinical diagnosis of Corticobasal Degeneration (CBD)
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Survival status after 5 years of clinical follow-up
Time Frame: 5 years
|
To determine patient survival status after 5 years of follow-up for survival analysis using the Kaplan-Meier Method
|
5 years
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Annual change in degree of disability in PSP, CBD and APS cases as determined by the PSP rating scale
Time Frame: 3 years
|
Change in clinical symptoms and motor function in PSP, CBD and APS cases to determine degree of disability and rate of disease progression using a 0-100 rating scale
|
3 years
|
Annual change in degree of disability in MSA cases as determined by the Unified Multiple System Atrophy Rating Scale (UMSARS)
Time Frame: 3 years
|
Change in clinical symptoms and motor function in MSA cases to determine degree of disability and rate of disease progression with scores ranging from 0 to 104
|
3 years
|
Other Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
CSF biomarkers
Time Frame: 1 year
|
Changes in CSF biomarkers of neurodegeneration after one year of follow-up including: neurofilament light chain, total tau, tau isoforms, phosphorylated tau
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1 year
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Brain MRI
Time Frame: 1 year
|
Whole and regional brain atrophy (%/year) and functional connectivity measured across distinct brain regions (using low frequency BOLD signal) will be examined after 1 year of follow-up using brain MRI.
|
1 year
|
MoCA Cognitive function test
Time Frame: 3 years
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Cognitive function will be reviewed annually for 3 years using the Montreal Cognitive Assessment (MoCA).
The total score is out of 30 with higher scores indicating better cognitive functioning.
|
3 years
|
ACE-3 Cognitive function test
Time Frame: 3 years
|
Cognitive function will be reviewed annually for 3 years using the Addenbrookes Cognitive Examination (ACE-3).
The total score is out of 100 with higher scores indicating better cognitive functioning.
|
3 years
|
ECAS Cognitive function test
Time Frame: 3 years
|
Cognitive function will also viewed annually for 3 years using the Edinburgh Cognitive and Behavioural ALS Screen (ECAS).
The total score is out of 136 with higher scores indicating better cognitive functioning.
|
3 years
|
Collaborators and Investigators
Sponsor
Collaborators
Investigators
- Principal Investigator: Huw Morris, PhD, FRCP, University College, London
Study record dates
Study Major Dates
Study Start
Primary Completion (Anticipated)
Study Completion (Anticipated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Cardiovascular Diseases
- Vascular Diseases
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Eye Diseases
- Neurologic Manifestations
- Basal Ganglia Diseases
- Movement Disorders
- Synucleinopathies
- Neurodegenerative Diseases
- Pathological Conditions, Anatomical
- Tauopathies
- Cranial Nerve Diseases
- Autonomic Nervous System Diseases
- Ocular Motility Disorders
- Paralysis
- Primary Dysautonomias
- Hypotension
- Ophthalmoplegia
- Atrophy
- Multiple System Atrophy
- Shy-Drager Syndrome
- Supranuclear Palsy, Progressive
Other Study ID Numbers
- 14/0371
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
IPD Plan Description
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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