Longitudinal Assessment of Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 12
- 试验地点
- 2
- 主要终点
- Intra-subject change in intact and medical food protein intake (grams/day) as reported on three-day food records at months 0, 9, and 15.
研究概览
简要总结
Conduct a prospective, longitudinal study to evaluate nutritional status in adults with phenylketonuria (PKU) before and during treatment with pegvaliase (Palynziq™).
详细描述
The investigators will compare intra-subject change in adults with PKU who follow a protein-restricted diet (protein intake less than the RDA for protein based on weight (0.8 g/kg/day) with or without a PKU medical food) within 90 days of starting pegvaliase (baseline) and 9 and 15 months later. The investigators will collect laboratory measures of nutrition status, anthropometrics, Dual-Energy X-Ray Absorptiometry (DXA) to measure body composition and bone mineral density (BMD), and indirect calorimetry to measure resting energy expenditure (REE). In addition, three-day food records will be obtained to assess protein intake and overall diet quality.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of phenylketonuria
- •Aged 18-65 years
- •Following a protein-restricted diet with or without medical food within the last 30 days
- •Routine natural protein intake from food is less than the RDA for protein based on weight (0.8 g/kg/day).
- •If not consuming animal proteins, recommended protein intake is 115% RDA due to lower bioavailability of plant proteins.
- •Within ±90 days of starting treatment with pegvaliase at time of study enrollment
排除标准
- •Unable to consent to study
- •Under age 18 or over age 65 years
- •Routine natural protein intake is greater than the RDA for protein (0.8 g/kg/d) within the last 30 days.
- •Females who are currently pregnant or lactating
结局指标
主要结局
Intra-subject change in intact and medical food protein intake (grams/day) as reported on three-day food records at months 0, 9, and 15.
时间窗: 15 months
Intra-subject difference in lean body mass (%LBM) from month 0 to 15.
时间窗: 15 months
Intra-subject change in serum markers of protein status at months 0, 9, and 15.
时间窗: 15 months
Intra-subject difference in bone mineral density from month 0 to 15.
时间窗: 15 months
次要结局
- Intra-subject change in serum concentrations of essential fatty acid nutriture at months 0, 9, and 15.(15 months)
- Intra-subject change in emotional eating, cognitive restraint, uncontrolled eating at months 0, 9, and 15.(15 months)
- Intra-subject change in BMI at months 0, 9, and 15.(15 months)
- Intra-subject change in serum markers of cardiovascular status at months 0, 9, and 15.(15 months)
- Intra-subject change in concentrations of serum markers of micronutrient status at months 0, 9, and 15.(15 months)
- Intra-subject change in food neophobia at months 0, 9, and 15.(15 months)
- Intra-subject change in Epicurean eating tendencies at months 0, 9, and 15.(15 months)
研究者
Krista Viau
Principal Investigator
Boston Children's Hospital