Open-label, Randomized, 2-way Crossover, Monocentric, Controlled Study to Evaluate the Effect on Daily PHE Fluctuation of PKU GOLIKE Versus Standard of Care in Patients With Phenylketonuria
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 发起方
- 入组人数
- 21
- 试验地点
- 1
- 主要终点
- Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Day Following the Second Test Day With Standard of Care on the Following Morning, Before Any Food Intake.
研究概览
简要总结
This is an open-label, randomized, 2-way crossover, monocentric controlled study in patients (≥ 16 years old) with phenylketonuria (PKU).
The comparison will be between the test product (PKU GOLIKE, a prolonged-release amino-acids (AAs) mixture) and standard of care.
详细描述
The study will consist of a screening visit (V1), two treatment periods with two test days each and a final visit planned at the end of the second treatment period (V2). Test days will occur on two consecutive preferably non-working/school days (T1-T2 and T3-T4,) preferably over two consecutive weeks. Treatment days in the two periods will have to be the same days of the week. T1 should be within two weeks after V1. The study products will be self-administered at home. Following informed consent and verification of eligibility criteria, 20 patients with PKU will be randomized in a 1:1 ratio to one of the following two sequences: AB or BA where A=standard of care and B=PKU GOLIKE. In details:
- AB: patients will receive 3 daily self-administrations of their usual standard of care for 2 consecutive days in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in thesecond period.
- BA: patients will receive 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the first period followed by 3 daily self administrations of their usual standard of care for 2 consecutive days in the second period.
No change in the randomization sequence will be allowed.
Each patient will follow the same diet in terms of food, calories and nutrients ranges during the first days (T1 and T3) of each period and the same diet during the second days (T2 and T4) of each period, according to the age and body weight of the patient. The days before the test days each patient will follow her/his standard diet.
PKU GOLIKE /standard of care will be the only protein substitute allowed on each test day, and no sports activities will be allowed on sampling days. Five blood spots will be collected on the second test day of each period. Timing of self-administrations, meals and blood spots will be standardized for each test day. On the test days, no food will be allowed outside of the defined time windows. The first self-administration of each test day will be performed after an overnight fasting (10 -12 h) and before any food intake.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 16 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed, informed consent obtained by the patient prior to beingenrolled into the study and prior to starting any data collection. For legally minor patients, signed written consent shall be obtained also by the parents/legal guardian
- •Male or female, aged ≥16 years.
- •Patients with a registered diagnosis of PKU
- •Ability and willingness to comply with all study procedures and availability for the duration of the study.
- •Patients with mean value of blood Phe >360 µM in the previous 12 months (calculated on at least 3 samples during the previous 12 months; the last sample should be preferably obtained in the 30 days preceding inclusion in the study).
- •Patient taking free-AA and/or GMP as usual amino-acidssupplementation.
排除标准
- •Known or suspected hypersensitivity to any excipients/components of PKU GOLIKE.
- •Treatment with any drug therapy for PKU
- •Patient taking PKU GOLIKE as usual amino-acids supplementation
- •Patient taking LNAA as usual amino-acids supplementation
- •Any moderate to severe medical condition, which in the opinion of the Investigator would interfere with the study procedures or study outcome (reason to be provided)
- •Any current participation in another clinical trial involving investigational or marketed products in the 3 months prior to the inclusion in this study.
- •Pregnancy or lactation.
研究组 & 干预措施
AB where A=standard of care and B=PKU GOLIKE
Patients will receive 3 daily self-administrations of their usual standard of care for 2 consecutive days in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period.
干预措施: PKU GOLIKE (Dietary Supplement)
AB where A=standard of care and B=PKU GOLIKE
Patients will receive 3 daily self-administrations of their usual standard of care for 2 consecutive days in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period.
干预措施: Standard of Care (Dietary Supplement)
BA where B=PKU GOLIKE and A=standard of care
Patients will receive 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the first period followed by 3 daily self-administrations of their usual standard of care for 2 consecutive days in the second period.
干预措施: PKU GOLIKE (Dietary Supplement)
BA where B=PKU GOLIKE and A=standard of care
Patients will receive 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the first period followed by 3 daily self-administrations of their usual standard of care for 2 consecutive days in the second period.
干预措施: Standard of Care (Dietary Supplement)
结局指标
主要结局
Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Day Following the Second Test Day With Standard of Care on the Following Morning, Before Any Food Intake.
时间窗: at 8:00 am (±15 min) on the day following the second test day (T2; T4) with SoC
Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Day Following the Second Test Day With GOLIKE PLUS on the Following Morning, Before Any Food Intake.
时间窗: at 8:00 am (±15 min) on the day following the second test day (T2; T4) with Golike Plus
Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With Standard of Care Right Before Product Self-administration and Before Any Food Intake
时间窗: at 8:00 am(±15 min) on the second test day (T2; T4) with SoC
Measurement of blood phenylalanine (Phe) levels
Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With GOLIKE PLUS Right Before Product Self-administration and Before Any Food Intake
时间窗: at 8:00 am(±15 min) on the second test day (T2; T4) with Golike Plus
Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With Standard of Care Right Before Product Self-administration and Before Food Intake
时间窗: at 12:00 am (±15 min) on the second test day (T2; T4)with SoC
Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With GOLIKE PLUS Right Before Product Self-administration and Before Food Intake
时间窗: at 12:00 am (±15 min) on the second test day (T2; T4) with Golike Plus
Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With GOLIKE PLUS Right Before Product Self-administration and Before Any Food Intake
时间窗: at 8:00 pm (±15 min) on the second test day (T2; T4) with Golike Plus
Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With Standard of Care Before Food Intake
时间窗: At 4:00 pm (±15 min) on the second test (T2;T4) day with SoC
Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With GOLIKE PLUS Before Food Intake
时间窗: At 4:00 pm (±15 min) on the second test day (T2; T4) with Golike Plus
Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With Standard of Care Right Before Product Self-administration and Before Any Food Intake
时间窗: at 8:00 pm (±15 min) on the second test day (T2; T4) with SoC
次要结局
- Tyr Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With Standard of Care Right Before Product Self-administration and Before Any Food Intake(at 8:00 am(±15 min) on the second test day (T2; T4) with SoC)
- Tyr Blood Concentration With Dried Blood Spots (Umol/L)on the Second Test Day With GOLIKE PLUS Right Before Product Self-administration and Before Any Food Intake(at 8:00 am(±15 min)on the second test day (T2; T4) with Golike Plus)
- Tyr Blood Concentration With Dried Blood Spots (Umol/L)on the Second Test Day With Standard of Care Right Before Product Self-administration and Before Any Food Intake(at 12:00 am(±15 min) on the second test day (T2; T4) with SoC)
- Tyr Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With GOLIKE PLUS Right Before Product Self-administration and Before Any Food Intake(at 12:00 am(±15 min) on the second test day (T2; T4) with Golike Plus)
- Tyr Blood Concentration With Dried Blood Spots (Umol/L)on the Second Test Day With Standard of Care Before Food Intake(at 4:00 pm(±15 min)on the second test day (T2; T4) with SoC)
- Tyr Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With GOLIKE PLUS Before Food Intake(at 4:00 pm(±15 min)on the second test day (T2; T4) with Golike Plus)
- Tyr Blood Concentration With Dried Blood Spots (Umol/L)on the Second Test Day With Standard of Care Right Before Product Self-administration and Before Any Food Intake(at 8:00 pm(±15 min) on the second test day (T2; T4) with SoC)
- Tyr Blood Concentration With Dried Blood Spots (Umol/L)on the Second Test Day With GOLIKE PLUS Right Before Product Self-administration and Before Any Food Intake(at 8:00 pm(±15 min) on the second test day (T2; T4) with Golike Plus)
- Tyr Blood Concentration With Dried Blood Spots (Umol/L)on the Day Following the Second Test Day With Standard of Care on the Following Morning, Before Any Food Intake.(at 8:00 am(±15 min) on the day following the second test day (T2; T4) with SoC)
- Tyr Blood Concentration With Dried Blood Spots (Umol/L) on the Day Following the Second Test Day With GOLIKE PLUS on the Following Morning, Before Any Food Intake.(at 8:00 am(±15 min) on the day following the second test day (T2; T4) with Golike Plus)