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Which patients with Parkinson's disease participate in clinical trials? : One centre's experiences with a new cell based therapy trial (TRANSEURO)

Moore, Sarah F; Guzman, Natalie Valle; Mason, Sarah L; Williams-Gray, Caroline H and Barker, Roger A LU (2014) In Journal of Parkinson's Disease 4(4). p.6-671
Abstract

BACKGROUND: There is currently little evidence regarding the selection of patients for clinical trials in Parkinson's Disease (PD), especially those involving experimental therapies delivered using invasive techniques.

OBJECTIVE: Understanding which patients are recruited will increase awareness of issues regarding parity of access to clinical trials and have an impact on the wider applicability of results, as well as provoking discussion regarding future improvements in the enrolment process.

METHODS: TRANSEURO is an open label multi-centre surgical transplant trial which seeks to investigate the feasibility and efficacy of grafts of human foetal ventral mesencephalic tissue in patients with PD. The Cambridge based cohort... (More)

BACKGROUND: There is currently little evidence regarding the selection of patients for clinical trials in Parkinson's Disease (PD), especially those involving experimental therapies delivered using invasive techniques.

OBJECTIVE: Understanding which patients are recruited will increase awareness of issues regarding parity of access to clinical trials and have an impact on the wider applicability of results, as well as provoking discussion regarding future improvements in the enrolment process.

METHODS: TRANSEURO is an open label multi-centre surgical transplant trial which seeks to investigate the feasibility and efficacy of grafts of human foetal ventral mesencephalic tissue in patients with PD. The Cambridge based cohort of TRANSEURO participants (n = 26) was compared with a population representative sample of patients with PD eligible for, but not enrolled in, TRANSEURO (n = 33). Measurements were available in both populations for demographics, neuropsychological tests, tests of motor and non-motor function and quality of life.

RESULTS: Patients enrolled in TRANSEURO were younger and had significantly more years of education with higher scores on the revised Addenbrooke's Cognitive Examination. This difference was accounted for by memory, fluency and visuospatial subscores. There were significant differences in the Movement Disorder Society Unified PD Rating Scale scores with better motor function but more motor complications in the enrolled group. Those enrolled were also more likely to be under the care of the principal investigator of the study.

CONCLUSIONS: In this trial our population was younger and more educated with higher cognitive scores and better motor function than eligible PD patients not enrolled. This raises interesting questions about the parity of access to clinical trials of this nature amongst patients with PD.

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author
publishing date
type
Contribution to journal
publication status
published
keywords
Adult, Aged, Cell- and Tissue-Based Therapy, Cohort Studies, Female, Humans, Male, Middle Aged, Motor Activity, Neuropsychological Tests, Parkinson Disease, Psychiatric Status Rating Scales, Severity of Illness Index, Statistics, Nonparametric, Treatment Outcome, Journal Article, Multicenter Study, Randomized Controlled Trial, Research Support, Non-U.S. Gov't
in
Journal of Parkinson's Disease
volume
4
issue
4
pages
6 pages
publisher
IOS Press
external identifiers
  • scopus:84914697822
ISSN
1877-718X
DOI
10.3233/JPD-140432
language
English
LU publication?
no
id
8def3c9e-d99b-443b-a892-2d4bed02cc1f
date added to LUP
2016-11-24 15:02:25
date last changed
2017-11-19 04:35:10
@article{8def3c9e-d99b-443b-a892-2d4bed02cc1f,
  abstract     = {<p>BACKGROUND: There is currently little evidence regarding the selection of patients for clinical trials in Parkinson's Disease (PD), especially those involving experimental therapies delivered using invasive techniques.</p><p>OBJECTIVE: Understanding which patients are recruited will increase awareness of issues regarding parity of access to clinical trials and have an impact on the wider applicability of results, as well as provoking discussion regarding future improvements in the enrolment process.</p><p>METHODS: TRANSEURO is an open label multi-centre surgical transplant trial which seeks to investigate the feasibility and efficacy of grafts of human foetal ventral mesencephalic tissue in patients with PD. The Cambridge based cohort of TRANSEURO participants (n = 26) was compared with a population representative sample of patients with PD eligible for, but not enrolled in, TRANSEURO (n = 33). Measurements were available in both populations for demographics, neuropsychological tests, tests of motor and non-motor function and quality of life.</p><p>RESULTS: Patients enrolled in TRANSEURO were younger and had significantly more years of education with higher scores on the revised Addenbrooke's Cognitive Examination. This difference was accounted for by memory, fluency and visuospatial subscores. There were significant differences in the Movement Disorder Society Unified PD Rating Scale scores with better motor function but more motor complications in the enrolled group. Those enrolled were also more likely to be under the care of the principal investigator of the study.</p><p>CONCLUSIONS: In this trial our population was younger and more educated with higher cognitive scores and better motor function than eligible PD patients not enrolled. This raises interesting questions about the parity of access to clinical trials of this nature amongst patients with PD.</p>},
  author       = {Moore, Sarah F and Guzman, Natalie Valle and Mason, Sarah L and Williams-Gray, Caroline H and Barker, Roger A},
  issn         = {1877-718X},
  keyword      = {Adult,Aged,Cell- and Tissue-Based Therapy,Cohort Studies,Female,Humans,Male,Middle Aged,Motor Activity,Neuropsychological Tests,Parkinson Disease,Psychiatric Status Rating Scales,Severity of Illness Index,Statistics, Nonparametric,Treatment Outcome,Journal Article,Multicenter Study,Randomized Controlled Trial,Research Support, Non-U.S. Gov't},
  language     = {eng},
  number       = {4},
  pages        = {6--671},
  publisher    = {IOS Press},
  series       = {Journal of Parkinson's Disease},
  title        = {Which patients with Parkinson's disease participate in clinical trials? : One centre's experiences with a new cell based therapy trial (TRANSEURO)},
  url          = {http://dx.doi.org/10.3233/JPD-140432},
  volume       = {4},
  year         = {2014},
}