GpCRC Pediatric Gastroparesis Registry 2: Characterization and Clinical Course of Symptoms and Gastric Emptying in Pediatric, Adolescent, and Young Adult Participants With Symptoms of Gastroparesis
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 216
- 试验地点
- 12
- 主要终点
- Change in mean symptom severity of gastrointestinal symptoms using the change in total score from the Pediatric Quality of Life Inventory (PedsQL) Gastrointestinal Symptoms Scales™
研究概览
简要总结
The objective of the Pediatric Gastroparesis Registry 2 is to create a national prospective registry of children, adolescents, and young adults with gastroparesis and gastroparesis-like syndrome (symptoms of gastroparesis but normal gastric emptying).
详细描述
An observational study to investigate the natural history and clinical course of children, adolescents, and young adults with symptoms of gastroparesis (e.g., nausea, vomiting, abdominal pain, bloating, distention) with either delayed or normal gastric emptying. To better understand these disorders, this registry will capture demographic, clinical, physiological, questionnaire, and patient outcome data to characterize the participants and their clinical course.
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
- 8 Years 至 25 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Provision of signed and dated informed consent form and assent, as age appropriate.
- •Stated willingness to comply with all study procedures and availability for the duration of the study
- •8 to 25 years of age at the time of enrollment
- •Symptoms of Gp of at least 12 weeks duration: constellation of some combination of:
- •nausea, vomiting, early satiety, postprandial fullness; symptoms sometimes may be accompanied by upper abdominal pain
- •Have undergone a gastric emptying scintigraphic study of solids using 4-hour Egg Beaters protocol (or equivalent generic liquid egg white meal) within the last 12 months who fall into one of the two categories:
- •Delayed gastric emptying-defined as an abnormal 2-hour (>60% retention) and/or 4- hour (>10% retention) result based on a 4-hour scintigraphic gastric emptying study
- •Gastric emptying that is not delayed but who have symptoms of Gp (designated in this study as GLS) or have previously participated in the Pediatric Gastroparesis Registry (PGpR) study
- •An etiology of either diabetic or idiopathic Gp or GLS
排除标准
- •Inability to comply with or complete the scintigraphic gastric emptying test (including allergy to eggs)
- •Pregnancy
- •Autism spectrum disorder, significant developmental delay, psychosis (because of inability to complete questionnaires)
- •Use of narcotic analgesics greater than three days per week.
- •Presence of conditions associated with GI mucosal disease (e.g., inflammatory bowel disease, known eosinophilic gastroenteritis or eosinophilic esophagitis, gastric ulcer, peptic ulcer, celiac disease)
- •Presence of any other condition that could case delayed gastric emptying
- •Gastrointestinal construction confirmed by upper endoscopy (EGD), upper gastrointestinal series (UGI), or abdominal CT
- •Primary neurological conditions that can cause nausea and vomiting such as increased intracranial pressure, space occupying or inflammatory/infectious legions
- •Acute or chronic renal failure (abnormal creatinine for age) and /or on hemodialysis or peritoneal dialysis
- •Acute liver failure
- •Advanced liver disease (features of portal hypertension)
- •Clinically significant congenital heart disease (i.e., vaginal injury during cardiac repair)
- •History of esophageal, gastric or bowel surgery.
- •Metabolic disease including mitochondrial disease and inborn errors of metabolism
- •Chronic lung disease (including cystic fibrosis)
- •A serious chronic medical condition (e.g., inflammatory bowel disease)
- •Use of medications that can affect motility during the gastric emptying study
- •Any other condition, which in the option of the investigator, could explain the symptoms or could interfere with study requirements.
研究组 & 干预措施
Ages 8-25 with normal gastric emptying
Participants aged 8-25 with normal gastric emptying of solids based on gastric emptying scintigraphy
Ages 8-25 with delayed gastric emptying
Participants aged 8-25 with delayed gastric emptying of solids based on gastric emptying scintigraphy
结局指标
主要结局
Change in mean symptom severity of gastrointestinal symptoms using the change in total score from the Pediatric Quality of Life Inventory (PedsQL) Gastrointestinal Symptoms Scales™
时间窗: Baseline, 48 weeks
The PedsQL™ GI Symptoms Scales questionnaire has 65 response items covering 10 dimensions, with each item offered as a better-to-worse 5-point Likert scale choice: 0=never, 1=almost never, 2=sometimes, 3=often, 4=almost always. The item responses are transformed to a worse-to-better order and rescaled to 0-100: 0=almost always, 25= often, 50=sometimes, 75=almost never, 100= never, with higher scores indicating better health-related quality of life (HRQOL) and fewer problems or symptoms.he per-participant PedsQL™ GI Symptoms Scales total score is the sum of the 65 transformed and rescaled item responses. The primary outcome measure is the arithmetic mean of the 65 transformed item responses and is repeated at baseline and 48 weeks for each participant.
次要结局
- Change in Pain Catastrophizing Scale scores (PCS)(Baseline, 48 weeks)
- Change in volume (mL) of liquid consumed as assessed by the Water Load Satiety Test(Baseline, 48 weeks)
- Presence or absence of Carnett's sign as assessed by abdominal examination(Baseline)