Deferiprone for the Treatment of Parkinson’s Disease
Efficacy and Safety of Deferiprone in Treatment-Naïve and Non-Treatment-Naïve Patients with Parkinson’s Disease
ApoPharma Inc.
40 participants
Nov 28, 2016
Interventional
Conditions
Summary
In Parkinson’s disease, there is a loss of the cells that produce dopamine, a chemical that sends signals between brain cells, and this leads to problems in movement and other functions. Drugs exist that act to increase levels of dopamine, but while doing this may relieve symptoms, it does not have any impact on the underlying cause. Patients with Parkinson’s disease have been found to have a build-up of extra iron in the brain. While some iron is necessary for good health, too much of it can be toxic, and it is possible that the symptoms of Parkinson’s disease are due at least in part to extra iron acting to destroy the dopamine-producing cells in this brain region. Deferiprone is an iron chelator, meaning that it binds to free molecules of iron in the body and moves them or gets rid of them. This study is looking to see if giving this drug to Parkinson’s patients will act to remove the extra iron from the brain and so prevent further destruction of dopamine-producing cells, thereby slowing progression of the disease. A total of 40 patients in different countries will be enrolled. Both patients who are already on standard antiparkinsonian medication and patients who have not yet started on such medications will be enrolled, in order to see how other treatments affect the response to deferiprone. All subjects will receive deferiprone twice a day for 9 months. The dose will be 15 milligrams (mg) of deferiprone for every kilogram of body weight, for a total daily dosage of 30 mg/kg. Patients will undergo regular tests to check the safety of the drug and to see if it is acting to slow disease progression, as assessed by the change from baseline in scores on a scale that measures the severity of Parkinson's disease symptoms. In addition, because a possible side effect of deferiprone is a drop in a certain type of white blood cell, patients must have their blood tested weekly, but this can be done at a local laboratory.
Eligibility
Inclusion Criteria11
- Male or female aged 18 to < 80 years
- Parkinson’s disease diagnosed according to UK Parkinson’s Disease Society Brain Bank Clinical Diagnostic Criteria and based on the presence of at least two of the three cardinal features of the disease (rest tremor, bradykinesia, and rigidity). If rest tremor is not present, patients must have unilateral onset of symptoms.
- Absolute neutrophil count (ANC) greater than or equal to 1.5 x 10^9/L (greater than or equal to 1.0 x 10^9/L for Black population)
- Inclusion criterion for treatment-naïve patients only:
- At an early stage of the disease, without motor fluctuations
- On a stable dose for at least 3 months prior to the screening visit of any of the following treatments at an L-dopa equivalent total daily dose of up to 600 mg:
- Dopaminergic agonist alone
- L-dopa alone
- Combination therapy with dopaminergic agonist and L-dopa
- Rasagiline
- At an early stage of the disease, without motor fluctuations and/or L-dopa–induced dyskinesia
Exclusion Criteria10
- Diagnosis of Parkinson’s disease more than 3 years prior to screening visit
- Hoehn and Yahr stage greater than or equal to 3 in the “Off” state, indicating the need for walking assistance in the absence of treatment.
- Atypical or secondary Parkinsonism without dopa-sensitivity (e.g., vascular parkinsonism, supranuclear palsy, multisystem atrophy)
- Progressing Axis I psychiatric disorders (psychosis, hallucinations, compulsive disorders, substance addiction, bipolar disorder, severe depression, anxiety) as assessed in a semi-structured interview in accordance with the Diagnostic and Statistical Manual of Mental Disorders
- Current treatment with bromocriptine
- Current treatment with coenzyme Q10 or idebenone. (Patients who are on these medications but stop taking them at least 2 weeks prior to baseline may be enrolled.)
- Current use of a Deep Brain Stimulation (DBS) system. (Patients who previously had a DBS system but have had it removed may be enrolled.)
- Previous or current treatment with antiparkinsonian medication, or likely to require such medication over the duration of the trial
- Current treatment with coenzyme Q10 at any dosage. (Patients who are on this medication at a dosage <300 mg/day but stop taking it at least 2 weeks prior to the first dose of study medication may be enrolled.)
- Not stabilized in terms of the current antiparkinsonian therapeutic regimen, and likely to require any change in dopamine therapy over the duration of the trial
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Interventions
Participants will receive 15 mg/kg dose of deferiprone oral solution, taken orally, twice daily for 9 months. Compliance will be assessed at each post-baseline visit by reviewing the diary card in which patients are to record the daily volume of medication taken, and by collecting the medication containers, whether empty, partly used, or unopened, and checking the amount of solution remaining by weighing the bottles.
Locations(4)
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ACTRN12617001578392