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Study of Parkinson's Early Stage With Deferiprone (SKY)

This study is currently recruiting participants.
See Contacts and Locations
Verified July 2017 by ApoPharma
Sponsor:
Information provided by (Responsible Party):
ApoPharma
ClinicalTrials.gov Identifier:
NCT02728843
First received: March 31, 2016
Last updated: July 28, 2017
Last verified: July 2017
  Purpose
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 Intervention Phase
Parkinson's Disease Drug: Deferiprone Drug: Placebo Phase 2

Study Type: Interventional
Study Design: 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

Resource links provided by NLM:


Further study details as provided by ApoPharma:

Primary Outcome Measures:
  • 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:
  • 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.


Estimated Enrollment: 140
Study Start Date: July 2016
Estimated Study Completion Date: July 2018
Estimated Primary Completion Date: December 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.
  Eligibility

Ages Eligible for Study:   18 Years to 80 Years   (Adult, Senior)
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02728843

Contacts
Contact: Caroline Fradette, PhD 416-401-7543 cfradett@apopharma.com
Contact: Fernando Tricta, MD 416-401-7332 ftricta@apopharma.com

Locations
Canada, Ontario
Toronto Western Hospital Recruiting
Toronto, Ontario, Canada
Principal Investigator: L Kalia, MD, PhD, FRCPC         
France
CHU de Bordeaux, Centre Expert Parkinson Recruiting
Bordeaux, France
Principal Investigator: W Meissner, MD, PhD         
CHRU de Caen Recruiting
Caen, France
Principal Investigator: G Defer, MD, PhD         
Hôpital Gabriel Montpied Not yet recruiting
Clermont-Ferrand, France
Principal Investigator: F. Durif, MD, PhD         
Hôpital Henri Mondor Recruiting
Creteil, France
Principal Investigator: P Remy, MD, PhD         
Centre Hospitalier Régional Universitaire de Lille, Hôpital Roger Salengro Recruiting
Lille, France
Principal Investigator: D Devos, MD, PhD         
CHU Dupuytren Recruiting
Limoges, France
Principal Investigator: F Torny, MD         
CHRU de Montpellier - Hôpital Gui de Chauliac Recruiting
Montpellier, France
Principal Investigator: C Geny, MD         
CHU Pontchaillou Not yet recruiting
Rennes, France
Principal Investigator: S Drapier, MD         
CHU Charles Nicoll - Rouen Not yet recruiting
Rouen, France
Principal Investigator: D. MALTÊTE, MD, PhD         
Hôpitaux Universitaires de Strasbourg, Hôpital de Hautepierre Recruiting
Strasbourg, France
Principal Investigator: C Tranchant, MD, PhD         
CHU Purpan, Hôpital Pierre Paul Riquet Recruiting
Toulouse, France
Principal Investigator: O Rascol, MD, PhD         
Germany
Heinriche-Heine Universität Düsseldorf Not yet recruiting
Dusseldorf, Germany
Principal Investigator: A Schnitzler, MD, PhD         
UKSH Campus Kiel, Neurologie Recruiting
Kiel, Germany
Principal Investigator: D Berg, MD, PhD         
Universitätsklinikum Gießen und Marburg GmbH Recruiting
Marburg, Germany
Principal Investigator: K Eggert, MD         
Klinikum rechts der Isar Not yet recruiting
Munich, Germany
Principal Investigator: B Haslinger, MD, PhD         
United Kingdom
Royal Devon & Exeter Hospital Recruiting
Exeter, Devon, United Kingdom
Principal Investigator: R Sheridan, MB ChB, MRCP         
Fairfield General Hospital Recruiting
Bury, United Kingdom
Principal Investigator: J Raw, MB ChB         
Charing Cross Hospital Recruiting
London, United Kingdom
Principal Investigator: S Molloy, MB BCH BAO, MRCPI, MD, FRCP         
Newcastle Clinical Ageing Research Unit Recruiting
Newcastle Upon Tyne, United Kingdom
Principal Investigator: N Pavese, MD, PhD, FRCP         
Derriford Hospital Recruiting
Plymouth, United Kingdom
Principal Investigator: C Carroll, PhD, BM Bch, MRCP         
Sponsors and Collaborators
ApoPharma
Investigators
Study Director: Caroline Fradette, PhD ApoPharma Inc.
  More Information

Responsible Party: ApoPharma
ClinicalTrials.gov Identifier: NCT02728843     History of Changes
Other Study ID Numbers: LA48-0215
Study First Received: March 31, 2016
Last Updated: July 28, 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by ApoPharma:
Parkinson's disease
Deferiprone

Additional relevant MeSH terms:
Parkinson Disease
Parkinsonian Disorders
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Movement Disorders
Neurodegenerative Diseases
Deferiprone
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action

ClinicalTrials.gov processed this record on September 19, 2017