AiM-PD

• Research type

  Research Study

• Full title

  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

• IRAS ID

  191779

• Contact name

  Professor Anthony Schapira

• Contact email

  a.schapira@ucl.ac.uk

• Sponsor organisation

  Joint Research Office

• Eudract number

  2015-002571-24

• Clinicaltrials.gov Identifier

  Z6364106/2015/06/17, UCL Data Protection

• Duration of Study in the UK

  2 years, 0 months, 1 days

• Research summary

  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 (DE) over the duration of 6 months that will be taken three times a day, see below:

  1 Day 1-7, 60 mg
  2 Day 8-14, 120 mg
  3 Day 15-21, 180 mg
  4 Day 22-28, 300 mg
  5 Day 29-186, 420 mg
  
  The study drug is licensed in the EU as over the counter drug to treat the respiratory conditions to reduce mucus production. Our studies have shown that ambroxol can penetrate the brain in rodent and non-human primate models, and may have an effect in slowing. The results also indicate individuals who express the GBA mutation (increased risk of PD) is able to reduce the growth of cells that cause PD by stimulating an enzyme called glucocerebrosidase.

  We will collect spinal fluid, blood and urine samples before, during and after the study drug has been taken over 6 months. In these samples we shall measure ambroxol drug levels, assess whether 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 spinal fluid and brain. We shall also assess patients through clinical, cognitive assessments and questionnaires to determine if there is any improvement of their PD. If we are able to prove the study drug penetrates the brain and replicate our current findings in the laboratory, we shall move on to a much larger drug trial to test whether the study drug may be able to slow the progression of PD.

• REC name

  London - Bloomsbury Research Ethics Committee

• REC reference

  16/LO/1341

• Date of REC Opinion

  11 Aug 2016

• REC opinion

  Favourable Opinion