Study of Parkinson's Early Stage With Deferiprone

NCT ID: NCT02728843

Last Updated: 2024-04-10

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 140 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-10-12
• Study Completion Date: 2019-09-04

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.

Conditions

Parkinson's Disease

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Deferiprone 300 mg

One-half of a 600 mg tablet of deferiprone twice a day, for a total daily dosage of 600 mg

Group Type: EXPERIMENTAL

Deferiprone

Intervention Type: DRUG

600 mg tablets

Deferiprone 600 mg

One 600 mg tablet of deferiprone twice a day, for a total daily dosage of 1200 mg

Group Type: EXPERIMENTAL

Deferiprone

Intervention Type: DRUG

600 mg tablets

Deferiprone 900 mg

One and a half 600 mg tablets of deferiprone twice a day, for a total daily dosage of 1800 mg

Group Type: EXPERIMENTAL

Deferiprone

Intervention Type: DRUG

600 mg tablets

Deferiprone 1200 mg

Two 600 mg tablets of deferiprone twice a day, for a total daily dosage of 2400 mg

Group Type: EXPERIMENTAL

Deferiprone

Intervention Type: DRUG

600 mg tablets

Placebo

Depending on dosage cohort, either one half-tablet, one tablet, one and a half tablets, or two tablets of placebo, twice a day

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Tablets that match the deferiprone tablets in appearance

Interventions

Deferiprone

600 mg tablets

Intervention Type: DRUG

Placebo

Tablets that match the deferiprone tablets in appearance

Intervention Type: DRUG

Other Intervention Names

DFP

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

• 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.)

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

ApoPharma

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

David Devos, MD

Role: PRINCIPAL_INVESTIGATOR

Hospitalier Régional Universitaire de Lille

Locations

Toronto Western Hospital

Toronto, Ontario, Canada

CHU de Bordeaux, Centre Expert Parkinson

Bordeaux, , France

Hôpital Henri Mondor

Créteil, , 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, , France

Heinriche-Heine Universität Düsseldorf

Düsseldorf, , Germany

UKSH Campus Kiel, Neurologie

Kiel, , Germany

Universitätsklinikum Gießen und Marburg GmbH

Marburg, , Germany

Klinikum rechts der Isar

Munich, , Germany

Royal Devon & Exeter Hospital

Exeter, Devon, United Kingdom

Fairfield General Hospital

Bury, , United Kingdom

Charing Cross Hospital

London, , United Kingdom

Newcastle Clinical Ageing Research Unit

Newcastle upon Tyne, , United Kingdom

Derriford Hospital

Plymouth, , United Kingdom

Countries

Canada; France; Germany; United Kingdom

References

Devos D, Rascol O, Meissner WG, Foubert-Samier A, Lewis S, Tranchant C, Anheim M, Maltete D, Remy P, Eggert K, Pape H, Geny C, Couratier P, Carroll C, Sheridan R, Burn D, Pavese N, Raw J, Berg D, Suchowersky O, Kalia LV, Evans A, Drapier S, Danaila T, Schnitzler A, Corvol JC, Defer G, Temin NT, Fradette C, Tricta F, Moreau C; a Parkinson's disease study group. Therapeutic modalities of deferiprone in Parkinson's disease: SKY and EMBARK studies. J Parkinsons Dis. 2025 Feb;15(1):72-86. doi: 10.1177/1877718X241300295. Epub 2024 Dec 27.

Reference Type: DERIVED

PMID: 39973479 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Document Type: Informed Consent Form

View Document

Other Identifiers

LA48-0215

Identifier Type: -

Identifier Source: org_study_id