Study of Parkinson's Early Stage With Deferiprone

NCT02728843 | Completed Phase 2 Results DMC

• 1 other identifier: LA48-0215
• Study Type: interventional
• Target: 140
• Locations: 4 countries
• Sites: 20

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.

Conditions

Parkinson's Disease

Keywords

Parkinson's disease; Deferiprone

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (10)

• Change in Total Score on the MDS-UPDRS

  Nine months

• Change in Score on the Part I Subscale of the MDS-UPDRS

  Nine months

• Change in Score on the Part II Subscale of the MDS-UPDRS

  Nine months

• Change in Score in the Part IV Subscale of the MDS-UPDRS

  Nine months

• Change in the Combined Scores From Parts II and III of the MDS-UPDRS

  Nine months

Study Arms (5)

Deferiprone 300 mg

EXPERIMENTAL

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

Drug: Deferiprone

Deferiprone 600 mg

EXPERIMENTAL

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

Drug: Deferiprone

Deferiprone 900 mg

EXPERIMENTAL

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

Drug: Deferiprone

Deferiprone 1200 mg

EXPERIMENTAL

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

Drug: Deferiprone

Placebo

PLACEBO COMPARATOR

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

Interventions

Deferiprone DRUG

600 mg tablets

Also known as: DFP

Deferiprone 1200 mg; Deferiprone 300 mg; Deferiprone 600 mg; Deferiprone 900 mg

Placebo DRUG

Tablets that match the deferiprone tablets in appearance

Placebo

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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 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.)

Sponsors & Collaborators

• ApoPharma: lead

Study Sites (20)

Toronto Western Hospital

Toronto, Ontario, Canada

Location

CHU de Bordeaux, Centre Expert Parkinson

Bordeaux, France

Location

Hôpital Henri Mondor

Créteil, France

Location

Centre Hospitalier Régional Universitaire de Lille, Hôpital Roger Salengro

Lille, France

Location

CHU Dupuytren

Limoges, France

Location

Hôpital Neurologique Pierre Wertheimer

Lyon, France

Location

CHRU de Montpellier - Hôpital Gui de Chauliac

Montpellier, France

Location

CHU Pontchaillou

Rennes, France

Location

CHU Charles Nicoll - Rouen

Rouen, France

Location

Hôpitaux Universitaires de Strasbourg, Hôpital de Hautepierre

Strasbourg, France

Location

CHU Purpan, Hôpital Pierre Paul Riquet

Toulouse, France

Location

Heinriche-Heine Universität Düsseldorf

Düsseldorf, Germany

Location

UKSH Campus Kiel, Neurologie

Kiel, Germany

Location

Universitätsklinikum Gießen und Marburg GmbH

Marburg, Germany

Location

Klinikum rechts der Isar

Munich, Germany

Location

Royal Devon & Exeter Hospital

Exeter, Devon, United Kingdom

Location

Fairfield General Hospital

Bury, United Kingdom

Location

Charing Cross Hospital

London, United Kingdom

Location

Newcastle Clinical Ageing Research Unit

Newcastle upon Tyne, United Kingdom

Location

Derriford Hospital

Plymouth, United Kingdom

Location

Related Publications (1)

• 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.

  PMID: 39973479 DERIVED

MeSH Terms

Conditions

Parkinson Disease

Interventions

Deferiprone; Isoflurophate

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Intervention Hierarchy (Ancestors)

Pyridones; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Organofluorophosphonates; Organophosphonates; Organophosphorus Compounds; Organic Chemicals

Results Point of Contact

• Title: Caroline Fradette, PhD
• Organization: Chiesi Canada Corp.

c.fradette@chiesi.com

1-800-854-3534

Study Officials

• David Devos, MD

  Hospitalier Régional Universitaire de Lille

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: The placebo tablets had the same appearance as the deferiprone tablets, and within each dosing cohort, all participants took the same number of tablets at each dose.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 31, 2016
• First Posted: April 5, 2016
• Study Start: October 12, 2016
• Primary Completion: August 2, 2019
• Study Completion: September 4, 2019
• Last Updated: April 10, 2024
• Results First Posted: April 10, 2024

Record last verified: 2024-03

Data Sharing

• IPD Sharing: Will not share

Locations

DE (4); GB (5); CA (1); FR (10)