A mammoth £26m project – and the world’s largest-ever clinical trial of treatments to slow or stop the progression of Parkinson’s disease – has just launched, led by researchers at University College London and Newcastle University, reports News-Medical.net.
The aim is to accelerate the search for effective treatments with an innovative, flexible trial design testing multiple treatments in parallel, which could shave up to three years off the time needed to test a single drug candidate.
The trial team is recruiting up to 1 600 participants in its first phase, from more than 40 hospitals across England, Wales, Scotland and Northern Ireland, at the London (UCLH) and Newcastle (Clinical Ageing Research Unit) sites, with the other trial sites kicking off between now and next April.
Co-chief investigator Professor Thomas Foltynie of the UCL Queen Square Institute of Neurology, and consultant neurologist at UCLH’s National Hospital for Neurology and Neurosurgery (NHNN), said: “Although Parkinson’s is the second most common neurodegenerative disease worldwide, as yet there are no treatments that can slow its relentless progression. We are prioritising drugs that already show promise as potential treatments, based on an extensive review of prior evidence, as we seek to identify one that does more than just provide symptom relief.”
The new trial is using a multi-arm, multi-stage design, enabling several treatments to be tested at the same time, in comparison to a single group of participants taking a placebo, a method which has not been used before for Parkinson’s.
Initially, it will be testing two drugs known to be safe and effective at treating other conditions: a blood pressure medication and a drug used to treat an enlarged prostate.
By analysing results on an ongoing basis, ineffective treatments can be identified and dropped from the trial, with more promising drugs progressing. The design's flexibility also allows new treatment arms to be introduced within the same trial infrastructure.
The current standard clinical trials process is hugely time and resource consuming and stop-start in nature, taking up to 10 years for a single potential treatment to complete assessment.
Compared to running individual trials for each treatment, the structure of this new EJS ACT-PD trial can accelerate the assessment process by close to 25% (or up to three years).
The sheer scale of the trial provides a unique opportunity to embed research studies within the trial. Sub-studies funded by The Michael J Fox Foundation are assessing whether wearable technologies could be used to digitally monitor symptoms, and to search for specific and sensitive molecular signatures of Parkinson’s in samples from participants.
Professor Sonia Gandhi (UCL Queen Square Institute of Neurology and The Francis Crick Institute), who is co-leading the EJS ACT-PD trial innovation programme, said: “This research will tell us which drugs might be effective, but also, critically, why and how a drug may be working, and who may respond to it – it will change the way we monitor Parkinson’s in future trials.”
The trial will begin by testing two repurposed drugs: telmisartan, a medication for high blood pressure, and terazosin, most commonly used to treat an enlarged prostate. The scientists aim to add a third drug, ursodeoxycholic acid (UDCA), currently used for liver disease, in 2026.
Participants are people with the disease who were diagnosed aged 30 or older, are taking dopaminergic treatment, and who have not had deep brain stimulation surgery nor infusion treatments.
Eligible participants, once they start taking medication, will be invited to study visits, either in-person or remotely, every six months for up to three years. Trial medication will be delivered to participants’ homes.
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