Progressive Supranuclear Palsy (PSP)PROSPECT-MNCT02778607
PROgressive Supranuclear Palsy CorTico-Basal Syndrome Multiple System Atrophy Longitudinal Study UK
An observational study of Progressive Supranuclear Palsy (PSP) and Corticobasal Degeneration, run by University College, London at 1 site in United Kingdom.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Progressive Supranuclear Palsy (PSP), Corticobasal Degeneration, Multiple System Atrophy (MSA)
- How many people
- 900 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- Oct 2014to Jul 2023
- How long will I take part?
- 5 Years
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
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).
Who may be able to join
- 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.
Who cannot join
- 1. Participant has another significant medical or psychiatric illness that would interfere in completing assessments
- 2. Participant is pregnant.
Trial locations and contacts
1 site in 1 country.
Huw Morris, PhD, FRCP contact
United Kingdom1 site, 1 recruiting
- Recruiting
University College London Hospitals
London
Alyssa Costantini, MSc, 020 310 87462, prospect@ucl.ac.uk
Huw Morris, PhD, FRCP
About this study
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.
Read the 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.
- Conditions
- Progressive Supranuclear Palsy (PSP), Corticobasal Degeneration, Multiple System Atrophy (MSA)
- Keywords
- Atypical Parkinsonism Syndrome (APS)
- Medical subject headings
- Supranuclear Palsy, Progressive, Corticobasal Degeneration, Multiple System Atrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 5 years
Secondary outcomes (2)
Annual change in degree of disability in PSP, CBD and APS cases as determined by the PSP rating scale
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
Measured: 3 years
Annual change in degree of disability in MSA cases as determined by the Unified Multiple System Atrophy Rating Scale (UMSARS)
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
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 May 2016 (estimated)
- Study startOct 2014
- Primary completionJul 2023 (estimated)
- Study completionJul 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Progressive Supranuclear Palsy (PSP)Studies of the same condition, open and under way.
- More trials run by University College, LondonThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case-control
- Time perspective
- Prospective
- Enrolment
- 900 participants (estimated)
- Target duration
- 5 Years
Sponsor and investigators
- Lead sponsor
- University College, London
- Sponsor type
- University, hospital or other
- Collaborators
- University of Cambridge, University of Oxford, University of Manchester, Newcastle University, University of Sussex, Royal Gwent Hospital
- Responsible party
- Sponsor
- Investigators
- Huw Morris, PhD, FRCP, Principal investigator, University College, London
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02778607
- Sponsor's protocol ID
- 14/0371
- Acronym
- PROSPECT-M
- Status last verified
- Oct 2020
- First submitted
- 22 Apr 2016
- Last updated
- 19 Oct 2020
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Yes: Following the first 2 years of the study a data and samples access committee will be established comprised of a representative of each study site together with representatives of the PSP Association and MSA trust, and will be chaired by an independent member who is experienced in the review of sample and tissue requests. The availability of tissue and samples will be publicized by the PSP Association and MSA trust.
Study at a glance
From the registry record, last updated by the sponsor on 19 Oct 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- PROgressive Supranuclear Palsy CorTico-Basal Syndrome Multiple System Atrophy Longitudinal Study UK
- ClinicalTrials.gov ID
- NCT02778607
- Acronym / sponsor ID
- PROSPECT-M / 14/0371
- Conditions
- Progressive Supranuclear Palsy (PSP), Corticobasal Degeneration, Multiple System Atrophy (MSA)
- Keywords
- Atypical Parkinsonism Syndrome (APS)
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 900 participants (estimated)
- Lead sponsor
- University College, LondonUniversity, hospital or other
- Collaborators
- University of Cambridge, University of Oxford, University of Manchester, Newcastle University, University of Sussex, Royal Gwent Hospital
- Study start
- Oct 2014
- Primary completion
- Jul 2023 (estimated)
- Study completion
- Jul 2023 (estimated)
- First posted
- 20 May 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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:
- 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 design
- Observational model
- Case-control
- Time perspective
- Prospective
- Target follow-up
- 5 Years
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| 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) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- 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).
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (2 listed)
- 1. Participant has another significant medical or psychiatric illness that would interfere in completing assessments
- 2. Participant is pregnant.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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
2 secondary outcomes
Annual change in degree of disability in PSP, CBD and APS cases as determined by the PSP rating scale
Time frame: 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
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Oct 2020.
- Individual participant dataSharing planned: Yes. Following the first 2 years of the study a data and samples access committee will be established comprised of a representative of each study site together with representatives of the PSP Association and MSA trust, and will be chaired by an independent member who is experienced in the review of sample and tissue requests. The availability of tissue and samples will be publicized by the PSP Association and MSA trust.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Huw Morris, PhD, FRCP contact
Huw Morris, PhD, FRCP principal investigator
University College, London
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Progressive Supranuclear Palsy (PSP) and Corticobasal Degeneration, run by University College, London at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 19 Oct 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Alyssa Costantini, MSc (020 310 87462, prospect@ucl.ac.uk), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Survival status after 5 years of clinical follow-up (time frame: 5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Oct 2020.