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Trial registered on ANZCTR

Registration number
Ethics application status
Date submitted
Date registered
Date last updated
Date data sharing statement initially provided
Date results information initially provided
Type of registration
Retrospectively registered

Titles & IDs
Public title
Deferiprone for the Treatment of Parkinson’s Disease
Scientific title
Efficacy and Safety of Deferiprone in Treatment-Naïve and Non-Treatment-Naïve Patients with Parkinson’s Disease
Secondary ID [1] 293234 0
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Early-stage Parkinson’s disease 305273 0
Condition category
Condition code
Neurological 304583 304583 0 0
Parkinson's disease

Study type
Description of intervention(s) / exposure
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.
Intervention code [1] 299493 0
Treatment: Drugs
Comparator / control treatment
No comparator. All patients will receive the same treatment in this study.
Control group

Primary outcome [1] 303802 0
The change from baseline to Month 9 in the motor examination subscale (Part III) of the Movement Disorder Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS).
Timepoint [1] 303802 0
Baseline and Month 9
Secondary outcome [1] 340203 0
The change from baseline to Month 9 on the total score of the MDS-UPDRS
Timepoint [1] 340203 0
Baseline and Month 9
Secondary outcome [2] 340205 0
The change from baseline to Month 9 on score of the individual subscales Part I (non-motor experiences of daily living), Part II (motor experiences of daily living), and Part IV (motor complications) of the MDS-UPDRS
Timepoint [2] 340205 0
Baseline and Month 9
Secondary outcome [3] 340206 0
The change from baseline to Month 9 on the combined score of Parts II and III of the MDS-UPDRS
Timepoint [3] 340206 0
Baseline and Month 9
Secondary outcome [4] 340335 0
Time elapsed until the need for rescue medication. Rescue medication is defined as the initiation (for a treatment-naïve patient) or change in regimen (for a non-treatment-naïve patient) of antiparkinsonian medication. Patients who show a worsening of condition that meets certain criteria and who wish to start on rescue medication will be withdrawn from the trial.
Timepoint [4] 340335 0
Any time during the 9-month trial
Secondary outcome [5] 340338 0
The safety of deferiprone oral solution will be assessed through laboratory testing (hematology, blood chemistry, and urinalysis), ECG, vital signs, physical examination, administration of the Columbia Suicide Severity Rating Scale (as required by current guidelines for clinical trials of investigational neurological drugs), and collection of adverse events.
Timepoint [5] 340338 0
Site visits will be made at baseline (Month 0) and Months 1, 2, 3, 4, 5, 6, and 9.

• Hematology: Weekly after the start of dosing until Month 9
• Blood chemistry, vital signs, Columbia Suicide Severity Rating Scale: Each site visit
• Urinalysis: Screening and Months 3, 6, and 9
• ECG: Screening and Month 9
• Physical examination: Screening, baseline, and Months 2, 4, 6, and 9
• Adverse events: Collected throughout the study, from the time of the first dose up to 1 month after the final dose

Key inclusion criteria
Main inclusion criteria (all patients):

1. Male or female aged 18 to < 80 years
2. 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.
3. 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:

1. At an early stage of the disease, without motor fluctuations

Inclusion criteria for non-treatment-naïve patients only:

1. 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
2. At an early stage of the disease, without motor fluctuations and/or L-dopa–induced dyskinesia
Minimum age
18 Years
Maximum age
79 Years
Both males and females
Can healthy volunteers participate?
Key exclusion criteria
Main exclusion criteria (all patients):

1. Diagnosis of Parkinson’s disease more than 3 years prior to screening visit
2. 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.
3. Atypical or secondary Parkinsonism without dopa-sensitivity (e.g., vascular parkinsonism, supranuclear palsy, multisystem atrophy)
4. 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
5. Current treatment with bromocriptine
6. 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.)
7. Current use of a Deep Brain Stimulation (DBS) system. (Patients who previously had a DBS system but have had it removed may be enrolled.)

Exclusion criteria for treatment-naïve patients only:

1. Previous or current treatment with antiparkinsonian medication, or likely to require such medication over the duration of the trial
2. 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.)

Exclusion criteria for non-treatment-naïve patients only:

1. 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
2. Current treatment with any antiparkinsonian drug other than those listed in the inclusion criteria

Study design
Purpose of the study
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Allocation is not concealed
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Not applicable
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?

Intervention assignment
Single group
Other design features
Phase 2
Type of endpoint(s)
Statistical methods / analysis
Determination of sample size:
There is no formal sample size and power calculation. Based on preliminary data from other investigators, it is estimated that 20 patients per patient group will be sufficient to provide meaningful assessment of the treatment effect for the study objectives. Patients who withdraw during the first 6 months of the study will be replaced.

Treatment-naïve and non-treatment-naïve patients will be analyzed as two patient subgroups. All data and analysis results will be presented for these two patient groups separately and combined.

Efficacy Analysis:
The data on the change from baseline to each follow-up visit will be summarized by the two patient groups separately and combined, using descriptive statistics for each efficacy endpoint except time elapsed until the need for rescue medication.

For time elapsed until the need for rescue medication, the Kaplan-Meier survival curve will be presented for the two patient groups separately and combined. An analysis of covariance (ANCOVA) approach will be used for assessing the significance of the change from baseline to study end for all efficacy variables except time elapsed until the need for rescue medication, with baseline value as a covariate and patient group and visit as the main factors in the model. A Mixed-Effect Model Repeated Measure (MMRM) model will be used as the primary analysis method to analyze the observed data.

Safety Analysis:
The safety data for continuous variables will be summarized using descriptive statistics, and the safety data for discrete variables will be tabulated with frequency tables.

Recruitment status
Active, not recruiting
Date of first participant enrolment
Date of last participant enrolment
Date of last data collection
Sample size
Accrual to date
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 9290 0
Brain and Mind Centre - University of Sydney - Camperdown
Recruitment hospital [2] 9291 0
Monash Medical Centre - Clayton campus - Clayton
Recruitment hospital [3] 12510 0
Royal Melbourne Hospital - City campus - Parkville
Recruitment postcode(s) [1] 17955 0
2050 - Camperdown
Recruitment postcode(s) [2] 17956 0
3168 - Clayton
Recruitment postcode(s) [3] 24890 0
3050 - Parkville
Recruitment outside Australia
Country [1] 9327 0
State/province [1] 9327 0
Ontario, Alberta, and British Columbia

Funding & Sponsors
Funding source category [1] 297864 0
Commercial sector/Industry
Name [1] 297864 0
ApoPharma Inc.
Address [1] 297864 0
200 Barmac Drive
Toronto, Ontario M9L 2Z7
Country [1] 297864 0
Primary sponsor type
Commercial sector/Industry
ApoPharma Inc.
200 Barmac Drive
Toronto, Ontario M9L 2Z7
Secondary sponsor category [1] 296908 0
Commercial sector/Industry
Name [1] 296908 0
Apotex Pty Limited
Address [1] 296908 0
16 Giffnock Ave, Macquarie Park
NSW 2113
Country [1] 296908 0

Ethics approval
Ethics application status
Ethics committee name [1] 298913 0
Schulman IRB
Ethics committee address [1] 298913 0
4445 Lake Forest Drive
Suite 300
Cincinnati, OH 45242
Ethics committee country [1] 298913 0
Date submitted for ethics approval [1] 298913 0
Approval date [1] 298913 0
Ethics approval number [1] 298913 0
Ethics committee name [2] 302007 0
St Vincent's Hospital Melbourne
Ethics committee address [2] 302007 0
41 Victoria Parade Fitzroy VIC 3065
PO Box 2900 Fitzroy VIC 3065
Ethics committee country [2] 302007 0
Date submitted for ethics approval [2] 302007 0
Approval date [2] 302007 0
Ethics approval number [2] 302007 0

Brief 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.

Trial website
Trial related presentations / publications
Public notes

Principal investigator
Name 78630 0
Dr Simon Lewis
Address 78630 0
University of Sydney
Brain and Mind Centre
Level 2, 100 Mallett Street
Camperdown NSW 2050
Country 78630 0
Phone 78630 0
+61 420-754-663
Fax 78630 0
Email 78630 0
Contact person for public queries
Name 78631 0
Dr Caroline Fradette
Address 78631 0
ApoPharma Inc.
200 Barmac Drive
Toronto, Ontario M9L 2Z7
Country 78631 0
Phone 78631 0
Fax 78631 0
Email 78631 0
Contact person for scientific queries
Name 78632 0
Dr Caroline Fradette
Address 78632 0
ApoPharma Inc.
200 Barmac Drive
Toronto, Ontario M9L 2Z7
Country 78632 0
Phone 78632 0
Fax 78632 0
Email 78632 0

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No/undecided IPD sharing reason/comment
What supporting documents are/will be available?
No other documents available
Summary results
Have study results been published in a peer-reviewed journal?
Other publications
Have study results been made publicly available in another format?
Results – basic reporting
Results – plain English summary