NCT02915510已完成不适用
Evaluating the Tolerance, Safety and Acceptability of PKU GMPro, a Whey Protein Derived Feed for the Dietary Management of Phenylketonuria in Children and Adults - a Pilot Trial
适应症
干预措施
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 40
- 试验地点
- 5
- 主要终点
- Blood Phenylalanine (and other amino acids)
研究概览
简要总结
This study will evaluate the safety, gastrointestinal (GI) tolerance, acceptability and compliance of a Glycomacropeptide-based protein substitute for patients with Phenylketonuria (PKU).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Supportive Care
- 盲法
- None
入排标准
- 年龄范围
- 3 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female
- •Over 3 years of age
- •Diagnosed with classical or variant type phenylketonuria
- •Have been compliant in taking at least one amino acid-based, Phe-free protein substitute, providing at least 20g protein equivalents, for at least 1 month prior to trial commencement
- •Have a prescribed daily Phe allowance
- •Written informed consent from patient, or from parent / carer if applicable
排除标准
- •Currently prescribed Sapropterin or similar tetrahydrobiopterin based medication
- •Pregnant or lactating
- •Requiring parenteral nutrition
- •Major hepatic or renal dysfunction
- •Participation in other studies within 1 month prior to entry of this study
- •Allergy to any of the study product ingredients, including milk protein or soya
- •Investigator concern around willingness/ability of patient or parent/carer to comply with protocol requirements
研究组 & 干预措施
GMP
Experimental
Single arm designed, 3 day baseline, 28day on GMP
干预措施: Glycomacropeptide-based protein substitute (Dietary Supplement)
结局指标
主要结局
Blood Phenylalanine (and other amino acids)
时间窗: 3 points throughout 28 days
Blood spot test
次要结局
- Gastro-intestinal tolerance questionaire(12/31 days)
- Product compliance questionaire(31 days)
- Product acceptability questionnaire(2/31 days)
- Patient reproted nutrient intake (3 day food diaries)(6/31 days)
- Pateint-reported appetite (visual analogue scale)(6/31 days)
- Antropometry (hieight and weight)(2/31 days)
- Safety (Adverse events reporting)(31 days)
研究者
研究点 (5)
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