SKY: Study of Parkinson's Early Stage With Deferiprone
Study Details
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 2 |
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.
Study Design
Arms and Interventions
| 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 |
Drug: Deferiprone
600 mg tablets
Other Names:
|
| Experimental: Deferiprone 600 mg One 600 mg tablet of deferiprone twice a day, for a total daily dosage of 1200 mg |
Drug: Deferiprone
600 mg tablets
Other Names:
|
| 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 Names:
|
| Experimental: Deferiprone 1200 mg Two 600 mg tablets of deferiprone twice a day, for a total daily dosage of 2400 mg |
Drug: Deferiprone
600 mg tablets
Other Names:
|
| 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 |
Drug: Placebo
Tablets that match the deferiprone tablets in appearance
|
Outcome Measures
Primary Outcome Measures
- Score on the Part III subscale of the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) [Nine months]
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 [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 [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 [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 [Nine months]
Change from baseline to Month 9 in overall cognitive function as assessed by MoCA score
- Pharmacodynamics measures of oxidative stress biomarkers [Nine months]
Change from baseline to Month 9 in oxidative stress
- Pharmacodynamics measures of inflammatory factor biomarkers [Nine months]
Change from baseline to Month 9 in inflammatory factor
- Time until need for rescue medication [Up to nine months]
Time elapsed until the patient is deemed to require a change or increase in antiparkinsonian medication
- Safety of deferiprone [Nine months]
Number of subjects with adverse events
- Cmax for serum deferiprone and deferiprone 3-O-glucuronide [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 [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 [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.
Eligibility Criteria
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 109/L (≥1.0 x 109/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.)
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Toronto Western Hospital | Toronto | Ontario | Canada | |
| 2 | CHU de Bordeaux, Centre Expert Parkinson | Bordeaux | France | ||
| 3 | Hôpital Henri Mondor | Creteil | France | ||
| 4 | Centre Hospitalier Régional Universitaire de Lille, Hôpital Roger Salengro | Lille | France | ||
| 5 | CHU Dupuytren | Limoges | France | ||
| 6 | Hôpital Neurologique Pierre Wertheimer | Lyon | France | ||
| 7 | CHRU de Montpellier - Hôpital Gui de Chauliac | Montpellier | France | ||
| 8 | CHU Pontchaillou | Rennes | France | ||
| 9 | CHU Charles Nicoll - Rouen | Rouen | France | ||
| 10 | Hôpitaux Universitaires de Strasbourg, Hôpital de Hautepierre | Strasbourg | France | ||
| 11 | CHU Purpan, Hôpital Pierre Paul Riquet | Toulouse | France | ||
| 12 | Heinriche-Heine Universität Düsseldorf | Dusseldorf | Germany | ||
| 13 | UKSH Campus Kiel, Neurologie | Kiel | Germany | ||
| 14 | Universitätsklinikum Gießen und Marburg GmbH | Marburg | Germany | ||
| 15 | Klinikum rechts der Isar | Munich | Germany | ||
| 16 | Royal Devon & Exeter Hospital | Exeter | Devon | United Kingdom | |
| 17 | Fairfield General Hospital | Bury | United Kingdom | ||
| 18 | Charing Cross Hospital | London | United Kingdom | ||
| 19 | Newcastle Clinical Ageing Research Unit | Newcastle Upon Tyne | United Kingdom | ||
| 20 | Derriford Hospital | Plymouth | United Kingdom |
Sponsors and Collaborators
- ApoPharma
Investigators
- Study Director: Caroline Fradette, PhD, ApoPharma Inc.
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- LA48-0215