Study of Parkinson's Early Stage With Deferiprone

ApoPharma140 enrolled

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

140

Conditions

Parkinson's Disease

Interventions

DeferiproneDRUG

600 mg tablets

PlaceboDRUG

Tablets that match the deferiprone tablets in appearance

Eligibility

Sex: ALLMin age: 18 YearsMax age: 80 Years

Medical Language ↔ Plain English

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.)

Locations (20)

Toronto Western Hospital

Toronto, Ontario, Canada

CHU de Bordeaux, Centre Expert Parkinson

Bordeaux, France

Outcomes

Primary Outcomes

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.

Time frame: Nine months

Secondary Outcomes

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.

Time frame: Nine months

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.

Time frame: Nine months

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.

Time frame: Nine months

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.

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.