A Dose-Ranging Study of the Efficacy, Safety, and Pharmacokinetics of Deferiprone Delayed Release Tablets in Patients With Parkinson's Disease
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- ApoPharma
- 入组人数
- 140
- 试验地点
- 20
- 主要终点
- Change in Score on the Part III Subscale of the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS)
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
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.
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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.)
研究组 & 干预措施
Deferiprone 300 mg
One-half of a 600 mg tablet of deferiprone twice a day, for a total daily dosage of 600 mg
干预措施: Deferiprone (Drug)
Deferiprone 600 mg
One 600 mg tablet of deferiprone twice a day, for a total daily dosage of 1200 mg
干预措施: Deferiprone (Drug)
Deferiprone 900 mg
One and a half 600 mg tablets of deferiprone twice a day, for a total daily dosage of 1800 mg
干预措施: Deferiprone (Drug)
Deferiprone 1200 mg
Two 600 mg tablets of deferiprone twice a day, for a total daily dosage of 2400 mg
干预措施: Deferiprone (Drug)
Placebo
Depending on dosage cohort, either one half-tablet, one tablet, one and a half tablets, or two tablets of placebo, twice a day
干预措施: Placebo (Drug)
结局指标
主要结局
Change in Score on the Part III Subscale of the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS)
时间窗: Nine months
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.
次要结局
- Change in Score on the Part II Subscale of the MDS-UPDRS(Nine months)
- Change in the Combined Scores From Parts II and III of the MDS-UPDRS(Nine months)
- Change in Score in the Part IV Subscale of the MDS-UPDRS(Nine months)
- Cmax for Serum Deferiprone and Deferiprone 3-O-glucuronide(4 hours)
- 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 Montreal Cognitive Assessment (MoCA) Test(Nine months)
- Tmax for Serum Deferiprone and Deferiprone 3-O-glucuronide(4 hours)
- Safety of Deferiprone(Nine months)
- Area Under the Curve for Serum Deferiprone and Deferiprone 3-O-glucuronide(4 hours)