Pediatric Gastroparesis Registry

In the Pediatric Gastroparesis Registry (PGpR), we will collect detailed epidemiological,
clinical, psychological, and patient outcome data with the goal of classifying patients with
gastroparesis and gastroparesis-like syndromes into pathophysiologically defined phenotypes.

The Primary Objective is to create a national prospective registry of children and
adolescents with gastroparesis and gastroparesis-like syndrome (symptoms of gastroparesis but
normal gastric emptying) to include:

1. Demographic, clinical, psychological, nutritional characteristics, physiological
measures, and serial assessments of symptoms over 3 years while receiving clinical care;

2. Establish a Biorepository of plasma, serum, peripheral blood mononuclear cells (PBMC),
GI mucosal biopsies (in those undergoing upper GI endoscopy), urine and stool collected
from the children and adolescents in this registry which will allow for future analyses
such as cytokines, bacterial DNA and microbiome to investigate the etiology and
pathogenesis of gastroparesis in children.

Secondary Objectives:

1. To determine what factors, if any, play a role in the outcomes of children who do not
improve within two years of diagnosis

2. To assess the spectrum of symptom severity of the cardinal gastroparesis symptoms:
nausea, vomiting, early satiety, postprandial fullness (and upper abdominal pain, if
present) in children with gastroparesis

3. To assess several areas of gastric motility in pediatric patients with symptoms of
gastroparesis (e.g., intragastric meal distribution, global gastric emptying)

4. To capture the clinical symptomatic course (outcome) of pediatric patients followed in
the registry

5. To determine whether gastroparesis symptoms are correlated with gastric emptying in a
pediatric population

6. To characterize abdominal pain in patients with gastroparesis and gastroparesis-like
syndrome by using questionnaires, water load satiety testing and quantitative sensory
testing (QST)

7. Determine the prevalence of hypermobility spectrum disorders in pediatric patients with
gastroparesis

8. To determine if the volume of water consumed during the water load satiety test is an
indirect measure of gastric accommodation

9. To determine if the water load satiety test is associated with an increased severity of
gastroparesis symptoms (fullness, bloating, abdominal pain, etc).

10. To compare the intragastric meal distribution during scintigraphy to symptoms of early
satiety, postprandial fullness in patients with symptoms of gastroparesis

11. Define immune cell profiles, cytokine induction and epigenetic changes in diabetic and
idiopathic gastroparesis

Inclusion Criteria:

- Cardinal symptoms of gastroparesis of at least 12 weeks duration. Cardinal symptoms of
gastroparesis are the constellation of some combination of: nausea, vomiting, early
satiety, postprandial fullness; symptoms sometimes may be accompanied by upper
abdominal pain

- An etiology of either diabetic, idiopathic, or post-fundoplication gastroparesis or
gastroparesis-like disorder (symptoms of gastroparesis but normal gastric emptying;
see below)

- Gastric emptying scintigraphy (GES) of solids using the 4-hour Egg Beaters® protocol
(or equivalent generic liquid egg white meal) within the last 6 months with either:

1. Abnormal gastric emptying rate defined as an abnormal 2-hour (>60% retention)
and/or 4-hour (>10% retention) result based on a 4-hour scintigraphic gastric
emptying study. (This group will comprise ~75% of patients in the registry)

2. Patients with a normal gastric emptying rate, but who have symptoms of
gastroparesis. (This group will comprise ~25% of patients in the registry)

- Age at least 5 years, and under 18 years at initial screening visit

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, or a history of
bipolar disorder (because of inability to complete the gastroparesis symptom
questionnaires: 24-hour recall and gastroparesis cardinal symptom questionnaire (the
latter to be completed during the scintigraphy if possible)

- 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)

- Presence of any other condition that could cause delayed gastric emptying including:

1. Gastrointestinal obstruction confirmed by EGD, UGI, or abdominal CT

2. Primary neurological conditions that can cause nausea and vomiting such as
increased intracranial pressure, space occupying or inflammatory/infectious
lesions

3. Acute or chronic renal failure (abnormal creatinine for age) and/or on
hemodialysis or peritoneal dialysis

4. Acute liver failure

5. Advanced liver disease (features of portal hypertension)

- Clinically significant congenital heart disease (i.e., vagal injury during cardiac
repair)

- History of esophageal, gastric or bowel surgery excepting prior fundoplication

- Metabolic disease including mitochondrial disease and inborn errors of metabolism

- Chronic lung disease (including cystic fibrosis)

- A serious chronic medical condition (e.g., cystic fibrosis)

- Use of medications that can affect motility during the gastric emptying study

- Any other condition, which in the opinion of the investigator, could explain the
symptoms or interfere with study requirements

- Inability to obtain informed consent/assent