A Pilot Clinical Trial With the Iron Chelator Deferiprone in Parkinson's Disease
Condition: Parkinson's Disease
MeSH terms: Parkinson Disease
Intervention: Deferiprone 20mg; Placebo; Deferiprone 30mg
Intervention description: 20mg/kg/d Deferiprone divided into two equal doses (morning and evening), every day for 6 months; Feriprox placebo administered orally at the same dosing volume as the 20mg/kg/day feriprox per day; 30mg/kg/d Deferiprone divided into two equal doses (morning and evening), every day for 6 months
Intervention MeSH terms: Deferiprone
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 22.0
Sex: All
Minimum age: 50 Years
Maximum age: 75 Years
Healthy volunteers: No
Keywords: Parkinson's disease; Brain Iron; Iron chelation; Oxidative stress

Summary:

      Parkinson's disease (PD) is a common neurodegenerative disease affecting movement. Although
      drug treatments for PD are available they only treat the symptoms of the disease, fail to
      halt neuronal loss, and are associated with long term side effects and loss of efficacy.
      There is a chronic need to develop neuroprotective therapies. Increased iron and oxidative
      stress have been heavily implicated in the neurodegenerative process in PD, hence removal of
      excess iron by iron chelation represents a potential drug target. Iron chelators are
      extensively utilised to treat peripheral iron overload disorders (e.g. thalassaemia) and
      recently the investigators have demonstrated iron chelators such as Deferiprone can enter the
      brain removing excess iron and are neuroprotective in PD animal models. Although good
      tolerability and efficacy to remove brain iron has also been shown in a pilot study with the
      iron chelators Deferiprone in young patients with Friedreich Ataxia, where iron accumulates
      in the dentate nucleus, no studies have been conducted in aged individuals affected by PD.
      Hence the aims of this study are 1) to assess whether Deferiprone is well tolerated in PD
      patients, 2) whether Deferiprone can remove the excess iron levels found in the brain area
      affected by PD, the substantia nigra, as assessed by Magnetic resonance imaging (MRI) and 3)
      whether Deferiprone has any direct effect on the clinical symptoms of PD. Three groups of 12
      (total 36) early stage drug free PD patients will be treated with 20 or 30mg/kg/d Deferiprone
      or Placebo for 6 months. Over the 6 months patients will receive serial MRI scans,
      neurological examinations not only to assess PD symptoms but also psychological state, plus
      blood test to monitor for potential side effects. Positive results from this pilot will help
      support larger clinical trials to evaluate whether Deferiprone can slow down/halt PD.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Clinical diagnosis of Parkinson's disease

          -  disease duration less than 5 years

          -  stable response to standard anti-Parkinson's medication for at least 6 weeks

        Exclusion Criteria:

          -  Other neurological conditions

          -  Diabetes

          -  Renal or liver disease

          -  Blood disorders

          -  Pregnancy or breast feeding

          -  Conditions which cause immunocompromise e.g. episodes of neutropaenia or
             agranulocytosis, HIV etc

          -  Prior history of hypersensitivity to Deferiprone or its excipient

          -  Pacemaker

          -  artificial heart valves

          -  ever had surgery to the head

          -  Metalic implants in the CNS e.g. cerebral aneurysm clips

          -  history of metal entering the eye