The GDNF study was a single-centre, investigator-led, randomised, placebo-controlled – followed by open-label clinical trial, to assess the safety and efficacy of intermittent bilateral intraputamenal Glial Cell Line-Derived Neurotrophic Factor (GDNF) infusions on a monthly basis via a skull-mounted port in people with Parkinson’s. 41 subjects were randomised and over 1000 brain infusions were administered between 2012 and 2017.
This £2.5million study was funded by Parkinson's UK, with support from the Cure Parkinson's Trust and in association with the North Bristol NHS Trust.
The trial is complete and initial analysis suggested the treatment is safe. The full results are coming and will help us understand whether GDNF has the potential to be a future treatment for Parkinson's.
Further information about this study can be found here: https://www.parkinsons.org.uk/research/clinical-trial-gdnf
CUSTOM DBS was a prospective, multi-centre, double-blind, randomized controlled trial. This study compared various program settings for the bilateral stimulation of the subthalmic nucleus (STN) using the Boston Scientific Corporation (BSC) implantable Vercise™ Deep Brain Stimulation (DBS) System for the treatment of levodopa-responsive, moderate to severe idiopathic PD.
The CUSTOM-DBS clinical study demonstrated that shorter stimulation pulses may offer a clinical advantage over DBS therapy using conventional pulses. The Vercise DBS System is the only commercially available platform with the capability to generate stimulation pulses at the shorter pulse width settings.
Further information about this study can be found here:
PD SURG was a large, pragmatic, multicentre “real-life” randomised trial to evaluate the role of surgery as therapy for PD. The fundamental question being addressed in this trial was:
Does early surgery provide more or less effective long-term control than medical therapy (with surgery deferred for as long as possible)?
In order to obtain the large number of patients needed to provide reliable answers, and to maximise the clinical relevance of the findings, the trial was designed to fit in with routine practice as far as possible and to impose minimal additional workload by keeping extra clinic-based tests and evaluations to a minimum (the majority of assessments are by postal questionnaires to patients and carers). Publication of the results will be in the name of the collaborative group and not those of the central organisers.
This study was run by the University of Birmingham through the Birmingham Clinical Trials Unit. It was funded by a grant from the UK Medical Research Council and UK Parkinson’s Disease Society awarded to the University of Birmingham.
Further information about this study can be found here: https://www.birmingham.ac.uk/research/activity/mds/trials/bctu/trials/p…
ReSPonD was a successful investigator-led single-centre phase II clinical trial to assess if the cholinesterase inhibitor Rivastigmine could be repositioned, through its effect on increased attention, to reduce gait instability and falls in people with Parkinson’s without dementia. The primary endpoint, improvement in step time variability, and a secondary endpoint, a reduction in falls (40%), was reached.
For the results and further discussion: http://www.thelancet.com/journals/laneur/article/PIIS1474-4422(15)00389-0/abstract
This study was funded as a Research Training Fellowship by Parkinson’s UK for Emily Henderson. An HTA has been awarded in 2017 by the NIHR for a confirmatory phase III multi-centre study where falls are the primary outcome and as part of which there will be an economic cost benefit analysis.
PD MED was a large, simple, "real-life" trial that aims to determine much more reliably which class of drug provides the most effective control, with the fewest side-effects, for both early and later PD. Patients with early PD were randomised between DA (Dopamine Agonist), MAOBI (monoamine oxidase type B inhibitors) and LD (Levodopa) alone, with the option to omit either the MAOBI or LD alone arm. Those whose disease was no longer controlled by their first class of drug, after dose titration and/or addition of LD, were randomised between COMTI (catechol-O-methyltransferase inhibitors), MAOBI and DA, with the option to omit either the MAOBI or the DA arm. The main outcome measure was the patient-rated PDQ-39 quality of life scale, which assesses all aspects of the patient's life, and is sensitive to changes considered important to patients but not identified by clinical rating scales.
In order to recruit the large number of patients needed to provide reliable answers, and to maximise the clinical relevance of the findings, the trial was designed to fit in with routine practice as far as possible and to impose minimal additional workload: clinicians could use the specific drug within each class that they prefer, treatments were prescribed in the usual way, and extra clinic-based tests and evaluations were kept to a minimum (the majority of assessments were by postal questionnaires to patients and carers).
This study was run by the University of Birmingham through the Birmingham Clinical Trials Unit. It was funded by the NHS Health Technology Assessment programme and was supported by the European Parkinson’s Disease Association, the Parkinson’s Disease Society and the Parkinson’s Disease Nurse Specialist Association.
Further study information can be found here: https://www.birmingham.ac.uk/research/activity/mds/trials/bctu/trials/pd/pdmed/index.aspx