NCT02941822

Ambroxol in Disease Modification in Parkinson Disease

A Phase IIA Prospective, Single-Centre, Open Label Clinical Trial to Evaluate the Safety, Tolerability and Pharmacodynamic Effects of Ambroxol in Patients With Parkinson Disease: Ambroxol in Disease Modification in Parkinson Disease

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 23 (actual)
• Sponsor: University College, London · Academic / Other
• Sex: All
• Age: 40 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

This study will evaluate the safety, tolerability and pharmacodynamics of ambroxol in participants with Parkinson Disease. Participants will administer ambroxol at five dose levels and will undergo clinical assessments, lumbar punctures, venepuncture, biomarker blood analysis and cognitive assessment throughout the course of the study.

Detailed description

The AiM-PD study will recruit 20 patients (10 GBA-positive \& 10 GBA-negative status) diagnosed with Parkinson disease (PD). Each patient will self-administer the study drug, ambroxol (60 mg per tablet) at 5 intra-dose escalations over the course of 6 months, as shown below: 1. Day 1-7: 60 mg three times a day 2. Day 8-14: 120 mg three times a day 3. Day 15-21: 180 mg three times a day 4. Day 22-28: 300 mg three times a day 5. Day 29-186: 420 mg three times a day The study drug is licensed in the EU as an over-the-counter drug to treat respiratory conditions by reducing mucus production. Previous studies have shown that ambroxol can penetrate the brain in rodent and non-human primate models, and may have an effect in slowing PD. The results also indicate individuals who express the GBA mutation (increased risk of PD) are able to reduce the growth of cells that cause PD by stimulating an enzyme called glucocerebrosidase. The study will collect cerebrospinal fluid, blood, and urine samples before, during and after the drug has been taken over a 6 month period. In these samples the Investigators will measure ambroxol drug levels, assess whether the glucocerebrosidase enzyme has been stimulated and the levels of other substances thought to be associated with the development of PD and confirm whether the study drug has penetrated the cerebrospinal fluid and CNS. The study will administer clinical and cognitive assessments to determine if there is any improvement in patient's PD symptoms. If the study proves ambroxol penetrates the CNS and replicates our current findings in the laboratory, the Investigators shall move on to a much larger drug trial to test whether the study drug may be able to slow the progression of PD.

Conditions

• Parkinson Disease

Interventions

Type	Name	Description
DRUG	Ambroxol	Details outlines in the intervention description.

Timeline

• Start date: 2016-12-01
• Primary completion: 2018-04-01
• Completion: 2018-05-01
• First posted: 2016-10-21
• Last updated: 2020-04-15

Locations

1 site across 1 country: United Kingdom