Clinical trial NCT02728843: Study of Parkinson's Early Stage With Deferiprone
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
Overall status: COMPLETED
Phase: PHASE2
Conditions: Parkinson's Disease
Keywords: Parkinson's disease
Deferiprone
Lead sponsor: Chiesi Canada Corp
Sponsor class: INDUSTRY
First posted: 2016-04-05
Enrollment: 140
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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.)

Primary outcomes:
measure: Change in Score on the Part III Subscale of the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS)
timeFrame: Nine months
Secondary outcomes:
measure: Change in Total Score on the MDS-UPDRS
timeFrame: Nine months
measure: Change in Score on the Part I Subscale of the MDS-UPDRS
timeFrame: Nine months
measure: Change in Score on the Part II Subscale of the MDS-UPDRS
timeFrame: Nine months
measure: Change in Score in the Part IV Subscale of the MDS-UPDRS
timeFrame: Nine months
measure: Change in the Combined Scores From Parts II and III of the MDS-UPDRS
timeFrame: Nine months
measure: Change in Score on the Montreal Cognitive Assessment (MoCA) Test
timeFrame: Nine months
measure: Safety of Deferiprone
timeFrame: Nine months
measure: Cmax for Serum Deferiprone and Deferiprone 3-O-glucuronide
timeFrame: 4 hours
measure: Tmax for Serum Deferiprone and Deferiprone 3-O-glucuronide
timeFrame: 4 hours
measure: Area Under the Curve for Serum Deferiprone and Deferiprone 3-O-glucuronide
timeFrame: 4 hours
Other outcomes:
Locations:
city: Toronto
country: Canada
facility: Toronto Western Hospital
state: Ontario
city: Bordeaux
country: France
facility: CHU de Bordeaux, Centre Expert Parkinson
city: Creteil
country: France
facility: Hôpital Henri Mondor
city: Lille
country: France
facility: Centre Hospitalier Régional Universitaire de Lille, Hôpital Roger Salengro
city: Limoges
country: France
facility: CHU Dupuytren
city: Lyon
country: France
facility: Hôpital Neurologique Pierre Wertheimer
city: Montpellier
country: France
facility: CHRU de Montpellier - Hôpital Gui de Chauliac
city: Rennes
country: France
facility: CHU Pontchaillou
city: Rouen
country: France
facility: CHU Charles Nicoll - Rouen
city: Strasbourg
country: France
facility: Hôpitaux Universitaires de Strasbourg, Hôpital de Hautepierre
city: Toulouse
country: France
facility: CHU Purpan, Hôpital Pierre Paul Riquet
city: Dusseldorf
country: Germany
facility: Heinriche-Heine Universität Düsseldorf
city: Kiel
country: Germany
facility: UKSH Campus Kiel, Neurologie
city: Marburg
country: Germany
facility: Universitätsklinikum Gießen und Marburg GmbH
city: Munich
country: Germany
facility: Klinikum rechts der Isar
city: Exeter
country: United Kingdom
facility: Royal Devon & Exeter Hospital
state: Devon
city: Bury
country: United Kingdom
facility: Fairfield General Hospital
city: London
country: United Kingdom
facility: Charing Cross Hospital
city: Newcastle Upon Tyne
country: United Kingdom
facility: Newcastle Clinical Ageing Research Unit
city: Plymouth
country: United Kingdom
facility: Derriford Hospital
Study officials:
affiliation: Hospitalier Régional Universitaire de Lille
name: David Devos, MD
role: PRINCIPAL_INVESTIGATOR
References: