Cure Parkinson’s is a major supporter of clinical trials for disease modification in Parkinson’s. For a new Parkinson’s treatment to be approved for use in hospitals, it must first pass through three phases of clinical testing.

Phase 1 is a small, short test of safety involving both healthy volunteers and individuals with the condition; phase 2 is a larger and longer test of safety with additional measures looking for evidence of efficacy, and phase 3 is a very large, multi-year study to determine safety and efficacy for long-term use.

Cure Parkinsons has many clinical trials underway across these different phases of clinical development. The drugs listed below are part of the clinical trials being progressed under our International Linked Clinical Trials programme (iLCT). As of 2023, there are now 60 drugs that have been prioritised by the iLCT committee for Parkinson’s with 17 drugs already in, or about to go into, clinical trials. There are only three disease-modifying phase 3 trials for Parkinson’s currently underway or imminent, and two of these have been directed through the iLCT process.

Our phase 3 International Linked Clinical Trials (iLCT)


Ambroxol is already being used to treat respiratory conditions. Cure Parkinson’s has supported a phase 2 clinical trial to test efficacy in treating Parkinson’s, and is supporting a further phase 3 clinical trial, designed to test the long-term efficacy of ambroxol in slowing the progression of Parkinson’s.

Ambroxol and Parkinson’s

Exenatide (also known as Bydureon) is a clinically available and widely used drug for the treatment of Type 2 diabetes. Cure Parkinson’s is investigating the repurposing of exenatide to treat the progression of Parkinson’s.

Exenatide and Parkinson’s

Clinical trials recruiting now

Find out more

The phase 2 International Linked Clinical Trials

The ‘PROSEEK Study’ – KO706 for Parkinson’s (awaiting full results to be published)

The drug named KO706 (a c-Abl inhibitor) is very similar to a drug called nilotinib (also a c-Abl inhibitor) which was supported into trial by Cure Parkinson’s. KO706 has been designed to better access the brain. This trial finished in early 2024 and we are now awaiting the results.

Read more about c-Abl inhibitors.

Read more about the KO706 PROSEEK Trial.

The Anle138b Phase 1b Study (awaiting full results to be published)

Anle138b is a promising drug that targets the toxic accumulation of alpha-synuclein with the aim of alleviating neurodegenerative symptoms. This study aimed to evaluate the safety and tolerability of Anle138b in people with mild to moderate Parkinson’s and the results were presented at the 2023 MDS conference. We are now awaiting publication of the full results.

The Anle138b Trial – find out more.

Read the MDS conference abstract here. 

The ‘AZA-PD Study’ – Azathioprine and Parkinson’s (recruitment complete)

This medication is already in use as a treatment for inflammation and the AZA-PD trial is looking at dampening down the immune response as a way of slowing the progression of Parkinson’s. The trial is being conducted in Cambridge (UK) and is supported by Cure Parkinson’s.

Read more about Azathioprine and Parkinson’s.

The ‘NLY-01’ Trial (results published)

A similar drug to exenatide, NLY-01 is designed to better access the brain. This drug was prioritised by the International Linked Clinical Trials (iLCT) committee in 2017. Similar drugs to NLY-01 already approved for the treatment of type 2 diabetes include exenatide, lixisenatide, and liraglutide – all three of these drugs have been or are being trialled in Parkinson’s through our iLCT programme.

The results of the phase 2 clinical trial of NLY-01 were published in Lancet Neurology at the beginning of 2024. Although researchers reported no significant difference in the motor or non-motor symptoms of participants taking NLY-01 and those taking the placebo (dummy drug), there were some signs that it improved motor symptoms in younger participants. Based on this, Neuraly, the company in charge of NLY-01, are now investigating how best to take this drug forward.

Find out more about NLY-01 here.

Read the full NLY01 trial results here. 

The ‘FAIRPARK-II Study’ – Deferiprone and Parkinson’s (results published)

Deferiprone is an iron chelator – a class of medication used to remove excess iron from the body. There is evidence that reducing iron in the brain can be beneficial for Parkinson’s.

The results of the FAIRPARK-II study were published in the New England Journal of Medicine in late 2022. Though researchers found deferiprone did reduce iron levels in the brain, but contrary to the study’s original proposal, clinical measurements detected a worsening of movement symptoms in those people taking deferiprone. Given that the findings from FAIRPARK-II do not confirm the findings from previous studies, important research questions need to be investigated further.

Read more about Deferiprone and Parkinson’s.

Read the full results here.

Liraglutide and lixisenatide in Parkinson’s (awaiting full results to be published)

Like exenatide, liraglutide and lixisenatide are part of a group of drugs called GLP-1R agonists, used to treat type 2 diabetes. Cure Parkinson’s is supporting these trials to determine their impact on the progression of Parkinson’s.

Read more about the liraglutide trial here.

Read more about the lixisenatide trial here.

The ‘UP-Study’ – UDCA and Parkinson’s (results published)

UDCA is used in the treatment of gallstones and has been determined to have beneficial effects on mitochondrial function in pre-clinical models of Parkinson’s. The UP study – UDCA in Parkinson’s, supported by Cure Parkinson’s, has now completed. Published in the journal Movement Disorders, the research team found UDCA to be safe and tolerable when taken by participants, with some evidence of improved cell energy production.

Read more about UDCA and Parkinson’s.

Read the full results here.

The APM Trials – Australian Parkinson’s Mission

The Australian Parkinson’s Mission (APM) is a 5-year clinical trial project funded by the Australian Federal Government. The first trial is testing three drugs that have been prioritised by the International Linked Clinical Trials initiative, comparing them with a single placebo. Further APM trials are being set up.

Read more about the APM

Other research studies recruiting

We also support other studies to help shape Parkinson’s research science.

Find out more here

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