Study of Parkinson's Early Stage With Deferiprone

Parkinson's Disease Clinical Trial

— SKY  

Official title:

A Dose-Ranging Study of the Efficacy, Safety, and Pharmacokinetics of Deferiprone Delayed Release Tablets in Patients With Parkinson's Disease

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02728843
• Other study ID #: LA48-0215
• Status: Completed
• Phase: Phase 2
• Start date: October 12, 2016
• Est. completion date: September 4, 2019

Study information

• Verified date: March 2024
• Source: Chiesi Canada Corp
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 140
• Est. completion date: September 4, 2019
• Est. primary completion date: August 2, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

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.)

Related Conditions & MeSH terms

• Parkinson Disease
• Parkinson's Disease

Intervention

Drug:
• Deferiprone
600 mg tablets
• Placebo
Tablets that match the deferiprone tablets in appearance

Locations

Country	Name	City	State
Canada	Toronto Western Hospital	Toronto	Ontario
France	CHU de Bordeaux, Centre Expert Parkinson	Bordeaux
France	Hôpital Henri Mondor	Creteil
France	Centre Hospitalier Régional Universitaire de Lille, Hôpital Roger Salengro	Lille
France	CHU Dupuytren	Limoges
France	Hôpital Neurologique Pierre Wertheimer	Lyon
France	CHRU de Montpellier - Hôpital Gui de Chauliac	Montpellier
France	CHU Pontchaillou	Rennes
France	CHU Charles Nicoll - Rouen	Rouen
France	Hôpitaux Universitaires de Strasbourg, Hôpital de Hautepierre	Strasbourg
France	CHU Purpan, Hôpital Pierre Paul Riquet	Toulouse
Germany	Heinriche-Heine Universität Düsseldorf	Dusseldorf
Germany	UKSH Campus Kiel, Neurologie	Kiel
Germany	Universitätsklinikum Gießen und Marburg GmbH	Marburg
Germany	Klinikum rechts der Isar	Munich
United Kingdom	Fairfield General Hospital	Bury
United Kingdom	Royal Devon & Exeter Hospital	Exeter	Devon
United Kingdom	Charing Cross Hospital	London
United Kingdom	Newcastle Clinical Ageing Research Unit	Newcastle Upon Tyne
United Kingdom	Derriford Hospital	Plymouth

Sponsors (1)

Lead Sponsor	Collaborator
ApoPharma

Countries where clinical trial is conducted

Canada,  France,  Germany,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in 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 the score for the Part III subscale (motor examination) of the MDS-UPDRS. Scores on this subscale can range from 0 (best) to 132 (worst); hence, an increase would indicate progression of the disease and a decrease would indicate improvement. The change in score was calculated as least square means.	Nine months
Secondary	Change in Total Score on the MDS-UPDRS	Change from baseline to Month 9 in total score on the MDS-UPDRS. The total score can range from 0 (best) to 260 (worst); hence, an increase would indicate progression of the disease and a decrease would indicate improvement. The change in score was calculated as least square means.	Nine months
Secondary	Change in Score on the Part I Subscale of the MDS-UPDRS	Change from baseline to Month 9 in the score for the Part I subscale (mentation, behavior, and mood) of the MDS-UPDRS. Scores on this subscale can range from 0 (best) to 52 (worst); hence, an increase would indicate progression of the disease and a decrease would indicate improvement. The change in score was calculated as least square means.	Nine months
Secondary	Change in Score on the Part II Subscale of the MDS-UPDRS	Change from baseline to Month 9 in the score for the Part II subscale (activities of daily living) of the MDS-UPDRS. Scores on this subscale can range from 0 (best) to 52 (worst); hence, an increase would indicate progression of the disease and a decrease would indicate improvement. The change in score was calculated as least square means.	Nine months
Secondary	Change in Score in the Part IV Subscale of the MDS-UPDRS	Change from baseline to Month 9 in the score for the Part IV subscale (motor complications) of the MDS-UPDRS. Scores on this subscale can range from 0 (best) to 24 (worst); hence, an increase would indicate progression of the disease and a decrease would indicate improvement. The change in score was calculated as least square means.	Nine months
Secondary	Change in the Combined Scores From Parts II and III of the MDS-UPDRS	Change from baseline to Month 9 in the combined score for the Part II subscale (motor experiences of daily living) and the Part III subscale (motor examination) of the MDS-UPDRS. The scales for these two parts range from 0 (best) to 52 (worst) and from 0 (best) to 132 (worst), respectively. Hence, the combined score can range from 0 (best) to 184 (worst), and an increase would indicate progression of the disease and a decrease would indicate improvement. The change in score is presented as least square means.	Nine months
Secondary	Change in Score on the Montreal Cognitive Assessment (MoCA) Test	Change from baseline to Month 9 in the score for overall cognitive function as assessed by the MoCA. The total score on the MoCA can range from 0 (worst) to 30 (best). Hence, a decrease in score would indicate progression of the disease and an increase would indicate improvement. The change in score is presented as least square means.	Nine months
Secondary	Safety of Deferiprone	Number of subjects with adverse events	Nine months
Secondary	Cmax for Serum Deferiprone and Deferiprone 3-O-glucuronide	Blood samples for pharmacokinetics assessments were collected at baseline, and at pre-dose and specified time points up to 12 hours post-dose at the Month 3 visit. The maximum measured serum concentration (Cmax) at the Month 3 visit was determined for deferiprone and its main metabolite, deferiprone 3-O-glucuronide.	4 hours
Secondary	Tmax for Serum Deferiprone and Deferiprone 3-O-glucuronide	Blood samples for pharmacokinetics assessments were collected at baseline, and at pre-dose and specified time points up to 12 hours post-dose at the Month 3 visit. The time to maximum observed serum concentration (Tmax) at the Month 3 visit was determined for deferiprone and its main metabolite, deferiprone 3-O-glucuronide.	4 hours
Secondary	Area Under the Curve for Serum Deferiprone and Deferiprone 3-O-glucuronide	Blood samples for pharmacokinetics assessments were collected at baseline, and at pre-dose and specified time points up to 12 hours post-dose at the Month 3 visit. The total drug exposure, AUC0-8 (area under the serum concentration time curve extrapolated to infinity) at the Month 3 visit was determined for deferiprone and its main metabolite, deferiprone 3-O-glucuronide.	4 hours