Back to resultsStudy of Parkinson's Early Stage With Deferiprone (SKY)
Primary Purpose
Parkinson's Disease
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Deferiprone
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Parkinson's Disease focused on measuring Parkinson's disease, Deferiprone
Eligibility Criteria
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.)
Sites / Locations
- Toronto Western Hospital
- CHU de Bordeaux, Centre Expert Parkinson
- Hôpital Henri Mondor
- Centre Hospitalier Régional Universitaire de Lille, Hôpital Roger Salengro
- CHU Dupuytren
- Hôpital Neurologique Pierre Wertheimer
- CHRU de Montpellier - Hôpital Gui de Chauliac
- CHU Pontchaillou
- CHU Charles Nicoll - Rouen
- Hôpitaux Universitaires de Strasbourg, Hôpital de Hautepierre
- CHU Purpan, Hôpital Pierre Paul Riquet
- Heinriche-Heine Universität Düsseldorf
- UKSH Campus Kiel, Neurologie
- Universitätsklinikum Gießen und Marburg GmbH
- Klinikum rechts der Isar
- Royal Devon & Exeter Hospital
- Fairfield General Hospital
- Charing Cross Hospital
- Newcastle Clinical Ageing Research Unit
- Derriford Hospital
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm 5
Arm Type
Experimental
Experimental
Experimental
Experimental
Placebo Comparator
Arm Label
Deferiprone 300 mg
Deferiprone 600 mg
Deferiprone 900 mg
Deferiprone 1200 mg
Placebo
Arm Description
One-half of a 600 mg tablet of deferiprone twice a day, for a total daily dosage of 600 mg
One 600 mg tablet of deferiprone twice a day, for a total daily dosage of 1200 mg
One and a half 600 mg tablets of deferiprone twice a day, for a total daily dosage of 1800 mg
Two 600 mg tablets of deferiprone twice a day, for a total daily dosage of 2400 mg
Depending on dosage cohort, either one half-tablet, one tablet, one and a half tablets, or two tablets of placebo, twice a day
Outcomes
Primary Outcome Measures
Score on the Part III subscale of the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS)
Change from baseline to Month 9 in motor examination, as assessed by score on Part III of the MDS-UPDRS
Secondary Outcome Measures
Total score on the MDS-UPDRS
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
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
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
Change from baseline to Month 9 in overall cognitive function as assessed by MoCA score
Pharmacodynamics measures of oxidative stress biomarkers
Change from baseline to Month 9 in oxidative stress
Pharmacodynamics measures of inflammatory factor biomarkers
Change from baseline to Month 9 in inflammatory factor
Time until need for rescue medication
Time elapsed until the patient is deemed to require a change or increase in antiparkinsonian medication
Safety of deferiprone
Number of subjects with adverse events
Cmax for serum deferiprone and deferiprone 3-O-glucuronide
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 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
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.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02728843
Brief Title
Study of Parkinson's Early Stage With Deferiprone
Acronym
SKY
Official Title
A Dose-Ranging Study of the Efficacy, Safety, and Pharmacokinetics of Deferiprone Delayed Release Tablets in Patients With Parkinson's Disease
Study Type
Interventional
2. Study Status
Record Verification Date
November 2019
Overall Recruitment Status
Completed
Study Start Date
October 12, 2016 (Actual)
Primary Completion Date
August 2, 2019 (Actual)
Study Completion Date
September 4, 2019 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
ApoPharma
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The goal of this study is to evaluate the effects of deferiprone, an iron-chelating drug, in patients with Parkinson's disease. Participants will be randomized to receive one of four different dosages of deferiprone or placebo, and will take the assigned study product twice a day for nine months.
Detailed Description
This study will enroll 140 patients who have been diagnosed with Parkinson's disease within the last 3 years and are currently taking antiparkinsonian medication. There are four dosage cohorts, with patients in each cohort receiving either deferiprone tablets or placebo. At the baseline visit, participants will be randomized to a dosage cohort and to either active product or placebo within that cohort, and will take the assigned study product twice-daily for 9 months. They will come back to the study site for assessments at Months 1, 2, 3, 4, 5, 6, and 9, and will need to have their blood count checked weekly, at either the study site or a local laboratory.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Parkinson's Disease
Keywords
Parkinson's disease, Deferiprone
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
140 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Deferiprone 300 mg
Arm Type
Experimental
Arm Description
One-half of a 600 mg tablet of deferiprone twice a day, for a total daily dosage of 600 mg
Arm Title
Deferiprone 600 mg
Arm Type
Experimental
Arm Description
One 600 mg tablet of deferiprone twice a day, for a total daily dosage of 1200 mg
Arm Title
Deferiprone 900 mg
Arm Type
Experimental
Arm Description
One and a half 600 mg tablets of deferiprone twice a day, for a total daily dosage of 1800 mg
Arm Title
Deferiprone 1200 mg
Arm Type
Experimental
Arm Description
Two 600 mg tablets of deferiprone twice a day, for a total daily dosage of 2400 mg
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Depending on dosage cohort, either one half-tablet, one tablet, one and a half tablets, or two tablets of placebo, twice a day
Intervention Type
Drug
Intervention Name(s)
Deferiprone
Other Intervention Name(s)
DFP
Intervention Description
600 mg tablets
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Tablets that match the deferiprone tablets in appearance
Primary Outcome Measure Information:
Title
Score on the Part III subscale of the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS)
Description
Change from baseline to Month 9 in motor examination, as assessed by score on Part III of the MDS-UPDRS
Time Frame
Nine months
Secondary Outcome Measure Information:
Title
Total score on the MDS-UPDRS
Description
Change from baseline to Month 9 in total score on the MDS-UPDRS
Time Frame
Nine months
Title
Scores on the Part I, Part II, and Part IV subscales of the MDS-UPDRS
Description
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
Time Frame
Nine months
Title
Combined scores from Parts II and III of the MDS-UPDRS
Description
Change from baseline to Month 9 in combined scores from Parts II and III of the MDS-UPDRS
Time Frame
Nine months
Title
Score on the Montreal Cognitive Assessment (MoCA) test
Description
Change from baseline to Month 9 in overall cognitive function as assessed by MoCA score
Time Frame
Nine months
Title
Pharmacodynamics measures of oxidative stress biomarkers
Description
Change from baseline to Month 9 in oxidative stress
Time Frame
Nine months
Title
Pharmacodynamics measures of inflammatory factor biomarkers
Description
Change from baseline to Month 9 in inflammatory factor
Time Frame
Nine months
Title
Time until need for rescue medication
Description
Time elapsed until the patient is deemed to require a change or increase in antiparkinsonian medication
Time Frame
Up to nine months
Title
Safety of deferiprone
Description
Number of subjects with adverse events
Time Frame
Nine months
Title
Cmax for serum deferiprone and deferiprone 3-O-glucuronide
Description
Maximum measured serum concentration. Blood samples collected at baseline and Month 3, pre-dose and at 2 hours and 4 hours post-dose.
Time Frame
4 hours
Title
Tmax for serum deferiprone and deferiprone 3-O-glucuronide
Description
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.
Time Frame
4 hours
Title
AUC0-∞for Serum Deferiprone and Deferiprone 3-O-glucuronide
Description
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.
Time Frame
4 hours
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Caroline Fradette, PhD
Organizational Affiliation
ApoPharma Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Toronto Western Hospital
City
Toronto
State/Province
Ontario
Country
Canada
Facility Name
CHU de Bordeaux, Centre Expert Parkinson
City
Bordeaux
Country
France
Facility Name
Hôpital Henri Mondor
City
Creteil
Country
France
Facility Name
Centre Hospitalier Régional Universitaire de Lille, Hôpital Roger Salengro
City
Lille
Country
France
Facility Name
CHU Dupuytren
City
Limoges
Country
France
Facility Name
Hôpital Neurologique Pierre Wertheimer
City
Lyon
Country
France
Facility Name
CHRU de Montpellier - Hôpital Gui de Chauliac
City
Montpellier
Country
France
Facility Name
CHU Pontchaillou
City
Rennes
Country
France
Facility Name
CHU Charles Nicoll - Rouen
City
Rouen
Country
France
Facility Name
Hôpitaux Universitaires de Strasbourg, Hôpital de Hautepierre
City
Strasbourg
Country
France
Facility Name
CHU Purpan, Hôpital Pierre Paul Riquet
City
Toulouse
Country
France
Facility Name
Heinriche-Heine Universität Düsseldorf
City
Dusseldorf
Country
Germany
Facility Name
UKSH Campus Kiel, Neurologie
City
Kiel
Country
Germany
Facility Name
Universitätsklinikum Gießen und Marburg GmbH
City
Marburg
Country
Germany
Facility Name
Klinikum rechts der Isar
City
Munich
Country
Germany
Facility Name
Royal Devon & Exeter Hospital
City
Exeter
State/Province
Devon
Country
United Kingdom
Facility Name
Fairfield General Hospital
City
Bury
Country
United Kingdom
Facility Name
Charing Cross Hospital
City
London
Country
United Kingdom
Facility Name
Newcastle Clinical Ageing Research Unit
City
Newcastle Upon Tyne
Country
United Kingdom
Facility Name
Derriford Hospital
City
Plymouth
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
No
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Study of Parkinson's Early Stage With Deferiprone
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