Clinical Trial Details
— Status: Completed
Administrative data
| NCT number |
NCT03421899 |
| Other study ID # |
17/0126 |
| Secondary ID |
|
| Status |
Completed |
| Phase |
|
| First received |
|
| Last updated |
|
| Start date |
August 18, 2017 |
| Est. completion date |
April 1, 2020 |
Study information
| Verified date |
May 2023 |
| Source |
University College, London |
| Contact |
n/a |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Observational
|
Clinical Trial Summary
Parkinson's disease (PD) is a progressive neurological disorder that is increasingly common
with age, with the incidence rising from approximately 4 people per 10,000 in their forties
to 2 in 100 over the age of eighty.
Our understanding of the causes of PD has rapidly developed in the past two decades, but this
has not yet translated into any clinically established neuroprotective treatment that slows
disease progression. There is a growing consensus that the failure of previous efforts is
mainly due to the causative diversity of PD i.e. that PD may have many different causes. For
example, it is known that variants in mitochondrial (cellular power house) genes can cause
specific forms of PD and this may be relevant to other forms of PD.
The aim of this study is to attempt to group PD patients based on markers of biochemical
dysfunction (e.g. into groups of patients that do and those who do not have evidence of
mitochondrial dysfunction) to aid in the development of new candidate neuro-protective
compounds.
The investigators hope by grouping people with Parkinson's into those with and without
impaired mitochondrial function the investigators will be better able to develop more
targeted treatments aimed at protecting further loss of brain cells that occurs in
Parkinson's disease.
To achieve this the investigators will study people, in two study sites in London, with both
genetic forms of PD and those with idiopathic PD (i.e. those where there is not a known
genetic variant causing PD), as well as a healthy control group. All groups will undergo
standardised clinical assessment to collect information on several aspects of their condition
(e.g. disease severity, memory problems and sleep problems).
Participants will be asked to provide blood, urine and optionally cerebrospinal fluid & skin
samples from which various biochemical assays and genetic analysis will be performed in
attempt to group participants based on the results of these tests. The study is funded for 3
years with participants being asked to attend for up to 3 study visits each over this time
period.
Description:
Parkinson's disease (PD) is a progressive neurological disorder that is increasingly
prevalent with age, with the incidence rising from approximately 4 people per 10,000 in their
forties to 2 in 100 over the age of eighty. Besides motor symptoms, such as tremor, rigidity,
bradykinesia, and postural instability, PD patients often experience a variety of non-motor
symptoms, such as fatigue, depression, sleep disturbance, and dementia.
Although symptomatic treatments exist to partially compensate for motor dysfunction, no
neuroprotective treatment has yet been established to slow PD progression, which inevitably
renders patients incapable of living independently. Compared to age- and sex-matched
controls, PD patients are about 5 times more likely to require nursing home care and this
care costs about 5 times more than average nursing home care. This, combined with the
European demographic shift toward an increasingly larger fraction of aged individuals,
creates a social and economic challenge to develop new medications to slow the progression of
PD.
Our understanding of the aetiopathogenesis of PD has rapidly developed in the past two
decades, but this has not yet translated into any clinically established neuroprotective
treatment that slows disease progression. There is a growing consensus that the failure of
previous efforts is mainly due to the aetiopathogenic diversity of PD and the estrangement of
existing preclinical models from clinical PD. For example, it is known that mutations in
mitochondrial genes can cause monogenic PD and biochemical evidence indicates that in a
proportion of cases, idiopathic PD is associated with detectable mitochondrial dysfunction.
Therefore, the investigators focus is on monogenic forms of PD that involve mitochondrial
abnormalities as a primary (e.g., Parkin, PINK1), or secondary (e.g., LRRK2, GBA1)
phenomenon, in order to extrapolate to idiopathic PD (IPD) patients with and without
mitochondrial dysfunction (Mito-IPD and Amito-IPD, respectively). Biochemical pathways
focusing on, but not restricted to mitochondrial function, will be assessed using a variety
of techniques including biochemical assays on blood, urine, CSF and tissue samples. The
investigators will explore both the relevance and measurement of specific biochemical
pathways in Parkinson's and related disorders
The main overall objective is to stratify PD patients based on dysfunction in biochemical
pathways related to PD. This will aid in developing new candidate neuroprotection compounds
to slow the progression of neurodegeneration.
