Clinical trial NCT03680820: Pediatric Gastroparesis Registry
Official title: Pediatric Gastroparesis Registry: Pediatric Registry of Patients With Suspected Gastroparesis
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Gastroparesis
Gastroparesis-like Syndrome
Lead sponsor: Johns Hopkins Bloomberg School of Public Health
Sponsor class: OTHER
Collaborators: name: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
name: Baylor College of Medicine
name: Nationwide Children's Hospital
name: Boston Children's Hospital
name: Harvard Medical School (HMS and HSDM)
First posted: 2018-09-21
Enrollment: 147
Sex: ALL
Minimum age (years): 5
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Children 5-9 yrs. of age \[n=210\] and 10-17 yrs. of age \[n=210\] suspected of having gastroparesis at the time they are undergoing gastric scintigraphy or who have undergone scintigraphy in the past 12 months with documented gastroparesis or gastroparesis-like syndrome (symptoms of gastroparesis but normal gastric emptying; 25% of the proposed study number 105 out of 420).

Brief summary: The objective of the Pediatric Gastroparesis Registry is to create a national prospective registry of children and adolescents with gastroparesis and gastroparesis-like syndrome (symptoms of gastroparesis but normal gastric emptying).

Detailed description: 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

Eligibility criteria: 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 12 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

Primary outcomes:
measure: Change in water load satiety volume consumed
timeFrame: 24 weeks
Secondary outcomes:
measure: Change in Pediatric Quality of Life (PedQL) Generic Core Scale
timeFrame: 48 weeks
measure: Change in Pediatric Quality of Life (PedQL) Gastrointestinal Symptom Scale
timeFrame: 48 weeks
Other outcomes:
Locations:
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Houston
country: United States
facility: Baylor College of Medicine/Texas Children's Hospital
state: Texas
Study officials:
affiliation: Baylor College of Medicine
name: Robert J Shulman, MD
role: STUDY_CHAIR
References: