Study of Parkinson's Early Stage With Deferiprone (SKY)
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ClinicalTrials.gov Identifier: NCT02728843 |
Recruitment Status :
Completed
First Posted : April 5, 2016
Last Update Posted : November 18, 2019
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Condition or disease | Intervention/treatment | Phase |
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Parkinson's Disease | Drug: Deferiprone Drug: Placebo | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 140 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
Official Title: | A Dose-Ranging Study of the Efficacy, Safety, and Pharmacokinetics of Deferiprone Delayed Release Tablets in Patients With Parkinson's Disease |
Actual Study Start Date : | October 12, 2016 |
Actual Primary Completion Date : | August 2, 2019 |
Actual Study Completion Date : | September 4, 2019 |

Arm | Intervention/treatment |
---|---|
Experimental: Deferiprone 300 mg
One-half of a 600 mg tablet of deferiprone twice a day, for a total daily dosage of 600 mg
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Drug: Deferiprone
600 mg tablets
Other Name: DFP |
Experimental: Deferiprone 600 mg
One 600 mg tablet of deferiprone twice a day, for a total daily dosage of 1200 mg
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Drug: Deferiprone
600 mg tablets
Other Name: DFP |
Experimental: Deferiprone 900 mg
One and a half 600 mg tablets of deferiprone twice a day, for a total daily dosage of 1800 mg
|
Drug: Deferiprone
600 mg tablets
Other Name: DFP |
Experimental: Deferiprone 1200 mg
Two 600 mg tablets of deferiprone twice a day, for a total daily dosage of 2400 mg
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Drug: Deferiprone
600 mg tablets
Other Name: DFP |
Placebo Comparator: Placebo
Depending on dosage cohort, either one half-tablet, one tablet, one and a half tablets, or two tablets of placebo, twice a day
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Drug: Placebo
Tablets that match the deferiprone tablets in appearance |
- Score on the Part III subscale of the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) [ Time Frame: Nine months ]Change from baseline to Month 9 in motor examination, as assessed by score on Part III of the MDS-UPDRS
- Total score on the MDS-UPDRS [ Time Frame: Nine months ]Change from baseline to Month 9 in total score on the MDS-UPDRS
- Scores on the Part I, Part II, and Part IV subscales of the MDS-UPDRS [ Time Frame: Nine months ]Change from baseline to Month 9 in non-motor experiences of daily living, motor experiences of daily living, and motor complications, as assessed by scores on Parts I, II, and IV, respectively of the MDS-UPDRS
- Combined scores from Parts II and III of the MDS-UPDRS [ Time Frame: Nine months ]Change from baseline to Month 9 in combined scores from Parts II and III of the MDS-UPDRS
- Score on the Montreal Cognitive Assessment (MoCA) test [ Time Frame: Nine months ]Change from baseline to Month 9 in overall cognitive function as assessed by MoCA score
- Pharmacodynamics measures of oxidative stress biomarkers [ Time Frame: Nine months ]Change from baseline to Month 9 in oxidative stress
- Pharmacodynamics measures of inflammatory factor biomarkers [ Time Frame: Nine months ]Change from baseline to Month 9 in inflammatory factor
- Time until need for rescue medication [ Time Frame: Up to nine months ]Time elapsed until the patient is deemed to require a change or increase in antiparkinsonian medication
- Safety of deferiprone [ Time Frame: Nine months ]Number of subjects with adverse events
- Cmax for serum deferiprone and deferiprone 3-O-glucuronide [ Time Frame: 4 hours ]Maximum measured serum concentration. Blood samples collected at baseline and Month 3, pre-dose and at 2 hours and 4 hours post-dose.
- Tmax for serum deferiprone and deferiprone 3-O-glucuronide [ Time Frame: 4 hours ]Time to maximum observed serum concentration. Blood samples collected at baseline and Month 3, pre-dose and at 2 hours and 4 hours post-dose.
- AUC0-∞for Serum Deferiprone and Deferiprone 3-O-glucuronide [ Time Frame: 4 hours ]Area under the serum concentration time curve extrapolated to infinity. Blood samples collected at baseline and Month 3, pre-dose and at 2 hours and 4 hours post-dose.

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Ages Eligible for Study: | 18 Years to 80 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Male or female aged ≥18 to < 80 years
- Body weight ≥60 kg but ≤100 kg
- Parkinson's disease diagnosed
- Absolute neutrophil count (ANC) ≥1.5 x 10^9/L (≥1.0 x 10^9/L for Black population) at screening
- 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 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 Criteria:
- Diagnosis of Parkinson's disease more than 3 years prior to screening visit
- Hoehn and Yahr stage ≥ 3
- 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
- Not stabilized in terms of the current antiparkinsonian therapeutic regimen: already requires dose adaptation and/or is likely to require any change in dopamine therapy over the duration of the trial
- Current treatment with bromocriptine
- Current treatment with any antiparkinsonian drug other than those listed in the inclusion criteria
- 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.)

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): NCT02728843

Study Director: | Caroline Fradette, PhD | ApoPharma Inc. |
Responsible Party: | ApoPharma |
ClinicalTrials.gov Identifier: | NCT02728843 |
Other Study ID Numbers: |
LA48-0215 |
First Posted: | April 5, 2016 Key Record Dates |
Last Update Posted: | November 18, 2019 |
Last Verified: | November 2019 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Parkinson's disease Deferiprone |
Parkinson Disease Parkinsonian Disorders Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Nervous System Diseases Movement Disorders |
Synucleinopathies Neurodegenerative Diseases Deferiprone Iron Chelating Agents Chelating Agents Sequestering Agents Molecular Mechanisms of Pharmacological Action |