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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
Sponsor:
Information provided by (Responsible Party):
ApoPharma

Study Description
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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
Parkinson's Disease Drug: Deferiprone Drug: Placebo 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
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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

Resource links provided by the National Library of Medicine

Drug Information available for: Deferiprone

Arms and Interventions
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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 Name: DFP
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 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
 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
 Drug: Placebo
Tablets that match the deferiprone tablets in appearance


Outcome Measures
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Primary Outcome Measures :
  1. 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


Secondary Outcome Measures :
  1. Total score on the MDS-UPDRS [ Time Frame: Nine months ]
    Change from baseline to Month 9 in total score on the MDS-UPDRS

  2. 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

  3. 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

  4. 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

  5. Pharmacodynamics measures of oxidative stress biomarkers [ Time Frame: Nine months ]
    Change from baseline to Month 9 in oxidative stress

  6. Pharmacodynamics measures of inflammatory factor biomarkers [ Time Frame: Nine months ]
    Change from baseline to Month 9 in inflammatory factor

  7. 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

  8. Safety of deferiprone [ Time Frame: Nine months ]
    Number of subjects with adverse events

  9. 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.

  10. 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.

  11. 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.


Eligibility Criteria
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Information from the National Library of Medicine

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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
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.)
Contacts and Locations
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Information from the National Library of Medicine

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


Locations
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Sponsors and Collaborators
ApoPharma
Investigators
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Study Director: Caroline Fradette, PhD ApoPharma Inc.
More Information
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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
Keywords provided by ApoPharma:
Parkinson's disease
Deferiprone
Additional relevant MeSH terms:
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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