Multi-arm, multi-stage trial platform

Improving clinical trial design for Parkinson鈥檚 therapies
Clinical trials

Stopping the progression of Parkinson鈥檚

Despite 30 years of research, not a single therapy has been found to successfully delay or stop the progression of Parkinson鈥檚 disease. Many potential disease-modifying therapies have been identified as suitable for clinical evaluation in Parkinson鈥檚. Each potential cure for Parkinson鈥檚 has to go through three clinical trial phases to test its safety investigating if it shows signs of improving Parkinson鈥檚 and if there are any meaningful benefit to people with Parkinson鈥檚.

Running a clinical trial is a huge logistical, costly and time-consuming undertaking. For a single new therapy this process can take the best part of a decade. Currently, phase II and phase III clinical trials are set up in isolation from each other 鈥 a process that is lengthy, costly and inefficient.

Photo of carer and person with walking disability, Copyright: Photographee.eu, courtesy of Shutterstock
Dr Camille Carroll, Post-Doctoral Research Fellow in Clinical Neuroscience

鈥淭he current way we do trials in Parkinson鈥檚 is too slow and inefficient. We need to develop new ways of doing trials, which will speed up the process and bring us closer to finding a cure, faster. We have the opportunity to learn from the experience in these other conditions and design a new trial that will work for people with Parkinson鈥檚.鈥
Dr Camille Carroll, Associate Professor and Honorary Consultant Neurologist

Evaluating several Parkinson鈥檚 therapies at once

Dr Camille Carroll and Dr Marie-Louise Zeissler report on the possibility of using a multi-arm, multi-stage (MAMS) trial platform to evaluate several potential therapies at once, using lessons learned from other diseases such as prostate, renal and oropharyngeal cancer, as well as other neurogenerative disorders, progressive multiple sclerosis (PMS) and motor neuron disease (MND).

MAMS trials test many potential therapies in parallel (multi-arm), transitioning seamlessly through various phases (multi-stage), i.e., from a phase II safety and efficacy study to a phase III trial. Early analyses allow unsuccessful therapies to be replaced. At the interim checkpoint, ineffective arms can be dropped and replaced by new treatment arms, thereby allowing for the continuous evaluation of interventions.

Speeding up the search for a cure

To maximise the potential of a MAMS platform trial over many years, it is crucial that there is a pipeline in place that will continuously identify and evaluate suitable drug candidates. 

Furthermore, outcome measures have to be chosen that are sensitive enough to changes in disease progression over interim stages as well as the full duration of the trial.

In 2020 and 2021, Dr Carroll, Dr Zeissler and their team will be working with The Cure Parkinson鈥檚 Trust, the Edmond J. Safra Foundation and other organisations national and internally to achieve just this.

It is hoped this will dramatically speed up the search for a cure.

Report analyses why complaints about doctors to the GMC have risen

Associated publications

Zeissler, Marie-Louisea; Li, Vivienb; Parmar, Mahesh K.B.; Carroll, Camille Buchholza, 2020  Journal of Parkinson's Disease, vol. 10, no. 2, pp. 413-428, 2020.