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A Pilot Clinical Trial With the Iron Chelator Deferiprone in Parkinson's Disease (DeferipronPD)

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ClinicalTrials.gov Identifier: NCT01539837
Recruitment Status : Completed
First Posted : February 28, 2012
Results First Posted : June 16, 2020
Last Update Posted : June 16, 2020
Sponsor:
Information provided by (Responsible Party):
Imperial College London

Brief 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.

Condition or disease Intervention/treatment Phase
Parkinson's Disease Drug: Deferiprone 20mg Drug: Placebo Drug: Deferiprone 30mg Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 22 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Pilot Clinical Trial With the Iron Chelator Deferiprone in Parkinson's Disease
Actual Study Start Date : February 2012
Actual Primary Completion Date : September 2014
Actual Study Completion Date : December 2014

Resource links provided by the National Library of Medicine

Drug Information available for: Deferiprone

Arm Intervention/treatment
Placebo Comparator: Placebo
Drug excipient
Drug: Placebo
Feriprox placebo administered orally at the same dosing volume as the 20mg/kg/day feriprox per day
Other Name: Feriprox placebo

Active Comparator: Deferiprone 20mg
20mg/kg/day deferiprone
Drug: Deferiprone 20mg
20mg/kg/d Deferiprone divided into two equal doses (morning and evening), every day for 6 months
Other Name: Ferriprox

Active Comparator: Deferiprone 30mg
30mg/kg/day Deferiprone
Drug: Deferiprone 30mg
30mg/kg/d Deferiprone divided into two equal doses (morning and evening), every day for 6 months
Other Name: Ferriprox




Primary Outcome Measures :
  1. Number of Participants With Serious Adverse Events [ Time Frame: 6 months ]
    To assess whether there were any serious adverse events in 6-month treatment with Deferiprone.


Secondary Outcome Measures :
  1. Iron Concentrations in the Dentate Nucleus [ Time Frame: 6 months ]
    Assess whether Deferiprone therapy directly affects the symptoms of Parkinson's disease, modify regional brain mineralization (iron concentration) as assessed with T2* MRI in PD patients in the dentate nucleus. In previous animal studies, Deferiprone treatment reduced dentate nucleus iron content, as assessed by MRI. An increase in the T2*MRI value represents an increase in mineralization.



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Ages Eligible for Study:   50 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01539837


Locations
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United Kingdom
Centre for Neuroscience, Imperial College London
London, United Kingdom, W120NN
Sponsors and Collaborators
Imperial College London
Investigators
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Principal Investigator: David T Dexter, PhD Imperial College London
Publications of Results:
Other Publications:
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Responsible Party: Imperial College London
ClinicalTrials.gov Identifier: NCT01539837    
Other Study ID Numbers: ICL-11/SC/0101
2011-001148-31 ( EudraCT Number )
11/SC/0101 ( Other Identifier: Research Ethics Committee )
First Posted: February 28, 2012    Key Record Dates
Results First Posted: June 16, 2020
Last Update Posted: June 16, 2020
Last Verified: June 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Imperial College London:
Parkinson's disease
Brain Iron
Iron chelation
Oxidative stress
Additional relevant MeSH terms:
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Parkinson Disease
Parkinsonian Disorders
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Movement Disorders
Neurodegenerative Diseases
Deferiprone
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action