A Phase 2 Study of a Vitamin Metabolite for PKAN
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 77
- 试验地点
- 2
- 主要终点
- Number of Treatment-emergent Adverse Events Assessed Using CTCAE v4.0
研究概览
简要总结
The purpose of this study is to learn more about how people with the condition pantothenate kinase-associated neurodegeneration (PKAN) respond to a specialized study product. We are hoping to find out if the study product is safe, what effects-good and bad-the study product causes, and whether the study product changes certain measures of disease in PKAN.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Other
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
Masking was used for the initial 6-month randomized, double-blind phase, placebo-controlled.
入排标准
- 年龄范围
- 3 Months 至 89 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Has a diagnosis of PKAN confirmed by: a) genetic testing confirming 2 pathogenic or likely pathogenic mutations, or (b) typical findings on exam and brain MR imaging with only one pathogenic mutation +/- a second likely pathogenic or VOUS in PANK2, or (c) typical findings on exam and brain MR imaging with a single likely pathogenic or VOUS in PANK2, or (d) be a symptomatic sibling of a proband subject meeting a, b or c.
- •Be between 3 months old and 89 years old.
- •Be able to take study product by mouth or feeding tube.
- •Be willing and able to complete study procedures / telephone visits / blood draws independently, OR have a caregiver / parent willing and able to assist with these tasks.
- •Be enrolled or willing to enroll in the PKANready natural history study (eIRB 10832).
- •Be resident in North America (US or Canada) for the duration of the trial.
排除标准
- •Have had exposure to a putative PANK2 bypass therapeutic agent in the 30 days prior to screening.
- •Be concurrently enrolled in another interventional clinical trial.
- •Have concurrent medical or other condition expected to preclude completion of study procedures of confound the assessment of clinical and laboratory measures of safety.
结局指标
主要结局
Number of Treatment-emergent Adverse Events Assessed Using CTCAE v4.0
时间窗: 6 months following first dose in double-blind phase
Safety will be measured using the number of treatment-emergent adverse events (both AE and SAE) by treatment arm. Counts are adjusted for number of prescription medications at baseline as a measure of baseline disease severity.
Number of Treatment-emergent Clinically Significant Laboratory Abnormalities on Complete Blood Count.
时间窗: 6-month randomized, double-blind, placebo-controlled phase
Safety will be assessed by measuring the number of treatment-emergent clinically significant laboratory abnormalities on Complete Blood Count. Clinical significance will be determined by Medical Safety Monitor and Clinical Investigators.
Number of Treatment-emergent Clinically Significant Laboratory Abnormalities on Comprehensive Metabolic Profile.
时间窗: 6-month randomized, double-blind, placebo-controlled phase
Safety will be assessed by measuring the number of treatment-emergent clinically significant laboratory abnormalities on Comprehensive Metabolic Profile by collection month. Clinical significance will be determined by Medical Safety Monitor and Clinical Investigators.
Number of Participants Retained in Each Arm.
时间窗: 6 month randomized, double-blind, placebo-controlled period
Tolerability will be assessed by measuring the number of participants retained in each arm over the course of the study.
Mean Percent of Study Product Consumed.
时间窗: 6-month randomized, double-blind, placebo-controlled phase
Tolerability will be assessed by adherence to the study product regimen arm at each follow-up time point.
次要结局
- CoASY mRNA Expression(Up to 6 months after first dose)
研究者
Susan J. Hayflick
Professor
Oregon Health and Science University