Study of Parkinson's Early Stage With Deferiprone (SKY)

ApoPharma

Status and phase: Completed; Phase 2

Conditions: Parkinson's Disease

Treatments: Drug: Placebo; Drug: Deferiprone

Study type: Interventional

Funder types: Industry

Identifiers

NCT02728843

LA48-0215

Details and patient eligibility

About

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.

Full 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.

Enrollment: 140 patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

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.)

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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140 participants in 5 patient groups, including a placebo group

Deferiprone 300 mg

Experimental group

Description:

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

Treatment:

Drug: Deferiprone

Deferiprone 600 mg

Experimental group

Description:

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

Treatment:

Drug: Deferiprone

Deferiprone 900 mg

Experimental group

Description:

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

Treatment:

Drug: Deferiprone

Deferiprone 1200 mg

Experimental group

Description:

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

Treatment:

Drug: Deferiprone

Placebo

Placebo Comparator group

Description:

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

Treatment:

Drug: Placebo