Phase 3 clinical trial of ambroxol for Parkinson’s confirmed

Cure Parkinson’s is delighted to announce that following promising results reported at phase 2 in 2020, a large-scale phase 3 clinical trial of ambroxol, a drug currently in use to treat respiratory conditions, will shortly begin.

The ASPro-PD trial is a world-first phase 3 trial aimed at establishing ambroxol’s potential to slow the progression of Parkinson’s. Following collaboration with the Parkinson’s community, driven by Cure Parkinson’s in partnership with Van Andel Institute and the John Black Charitable Foundation, the trial will investigate the ability of ambroxol to slow the progression of Parkinson’s. 

This ground-breaking trial, led by Professor Anthony Schapira at University College London (UCL), will involve 330 people with Parkinson’s across 10-12 clinical centres in the UK. The trial is placebo controlled and participants will take ambroxol for two years. The effectiveness of ambroxol will be measured by its ability to slow the progression of Parkinson’s using a scale including quality of life and movement. Preparations for recruitment of trial participants have already started however trial criteria are still to be finalised.

Ambroxol is one of the drugs prioritised by the International Linked Clinical Trials (iLCT) programme, created and operated by Cure Parkinson’s and Van Andel Institute. The iLCT mission is to slow, stop and reverse the progression of Parkinson’s; it aims to significantly reduce the time to bring disease-modifying treatments to clinic for the Parkinson’s community by testing promising drugs that already have extensive safety data and, in some cases, have been approved by regulators for other medical conditions.

This trial will cost £5.5 million, which is being funded by Cure Parkinson’s alongside its strategic partners, Van Andel Institute and the John Black Charitable Foundation, and by the Parkinson’s Virtual Biotech, the drug discovery and development arm of Parkinson’s UK. Cure Parkinson’s is committing to fund £2.2 million of the total cost, with the other funders contributing £1.1 million each.

This trial is a big step forward in the search to find new treatments for Parkinson’s. Once the ambroxol trial is underway, it will be one of only six phase 3 trials on public record of potentially disease-modifying drugs in Parkinson’s, worldwide. We at Cure Parkinson’s are working hard – through our efforts within the iLCT programme and in our fundraising efforts – to increase this number significantly in the next few years, to accelerate our progress towards a cure for Parkinson’s.”

Will Cook, CEO, Cure Parkinson’s