GpCRC Pediatric Gastroparesis Registry 2

Official title

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

Overview

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).

Detailed description

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.

Primary outcome measures

• 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™ [Time frame: Baseline, 48 weeks]

Secondary outcome measures (3)

• Presence or absence of Carnett's sign as assessed by abdominal examination [Time frame: Baseline]
• Change in volume (mL) of liquid consumed as assessed by the Water Load Satiety Test [Time frame: Baseline, 48 weeks]
• Change in Pain Catastrophizing Scale scores (PCS) [Time frame: Baseline, 48 weeks]

Eligibility criteria

Inclusion criteria

• 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

Exclusion criteria

• 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model

Cohort

Study locations

United States · 6 centers

• Massachusetts General Hospital — Boston
• Boston Children's Hospital — Boston
• Nationwide Children's Hospital — Columbus
• Texas Tech University Health Science Center — El Paso
• Baylor College of Medicine/Texas Children's Hospital — Houston
• Medical College of Wisconsin — Milwaukee

Publications

• Pasricha PJ, Grover M, Yates KP, Abell TL, Bernard CE, Koch KL, McCallum RW, Sarosiek I, Kuo B, Bulat R, Chen J, Shulman RJ, Lee L, Tonascia J, Miriel LA, Hamilton F, Farrugia G, Parkman HP; National Institute of Diabetes and Digestive and Kidney Diseases/National Institutes of Health Gastroparesis Clinical Research Consortium. Functional Dyspepsia and Gastroparesis in Tertiary Care are Interchang PMID 33548234
• Waseem S, Islam S, Kahn G, Moshiree B, Talley NJ. Spectrum of gastroparesis in children. J Pediatr Gastroenterol Nutr. 2012 Aug;55(2):166-72. doi: 10.1097/MPG.0b013e31824cf06e. PMID 22314391
• Rodriguez L, Irani K, Jiang H, Goldstein AM. Clinical presentation, response to therapy, and outcome of gastroparesis in children. J Pediatr Gastroenterol Nutr. 2012 Aug;55(2):185-90. doi: 10.1097/MPG.0b013e318248ed3f. PMID 22228004
• Rapkin AJ, Tsao JC, Turk N, Anderson M, Zeltzer LK. Relationships among self-rated tanner staging, hormones, and psychosocial factors in healthy female adolescents. J Pediatr Adolesc Gynecol. 2006 Jun;19(3):181-7. doi: 10.1016/j.jpag.2006.02.004. PMID 16731411
• Sizonenko PC, Aubert ML. Neuroendocrine changes characteristic of sexual maturation. J Neural Transm Suppl. 1986;21:159-81. PMID 3462329
• Angold A, Costello EJ. Depressive comorbidity in children and adolescents: empirical, theoretical, and methodological issues. Am J Psychiatry. 1993 Dec;150(12):1779-91. doi: 10.1176/ajp.150.12.1779. PMID 8238631
• Parkman HP, Yates K, Hasler WL, Nguyen L, Pasricha PJ, Snape WJ, Farrugia G, Koch KL, Abell TL, McCallum RW, Lee L, Unalp-Arida A, Tonascia J, Hamilton F; National Institute of Diabetes and Digestive and Kidney Diseases Gastroparesis Clinical Research Consortium. Clinical features of idiopathic gastroparesis vary with sex, body mass, symptom onset, delay in gastric emptying, and gastroparesis seve PMID 20965184
• Thapar N, Benninga MA, Crowell MD, Di Lorenzo C, Mack I, Nurko S, Saps M, Shulman RJ, Szajewska H, van Tilburg MAL, Enck P. Paediatric functional abdominal pain disorders. Nat Rev Dis Primers. 2020 Nov 5;6(1):89. doi: 10.1038/s41572-020-00222-5. PMID 33154368

Identifiers

NCT: NCT05981300 · 15-DK-PGpR2 · U01DK112194 · U01DK112193 · U01DK074035 · U24DK074008 · IRB00416909

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗