Pediatric Gastroparesis Registry

NCT03680820 | Completed DMC

• 3 other identifiers: 8 DK PGpRU01DK112194U24DK074008
• Study Type: observational
• Target: 147
• Locations: 1 country
• Sites: 3

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).

Conditions

Gastroparesis; Gastroparesis-like Syndrome

Outcome Measures

Primary Outcomes (1)

• Change in water load satiety volume consumed

  The Water Load Satiety Test involves the participant drinking refrigerated spring water (4 degrees C) from a 1-liter container over a period of 5 minutes until feeling completely full. The total volume of water consumed (mL) over 5 minutes is the unit of measure, and the outcome is assessed by calculating the change in volume consumed (24 weeks - baseline).

  24 weeks

Secondary Outcomes (2)

• Change in Pediatric Quality of Life (PedQL) Generic Core Scale

  48 weeks

• Change in Pediatric Quality of Life (PedQL) Gastrointestinal Symptom Scale

  48 weeks

Study Arms (4)

Ages 5-9, gastroparesis

Participants 5-9 years of age at screening with documented gastroparesis (delayed gastric emptying)

Ages 5-9, gastroparesis-like syndrome

Participants 5-9 years of age at screening with cardinal symptoms of gastroparesis, but who have normal gastric emptying

Ages 10-17, gastroparesis

Participants 10-17 years of age at screening with documented gastroparesis (delayed gastric emptying)

Ages 10-17, gastroparesis-like syndrome

Participants 10-17 years of age at screening with cardinal symptoms of gastroparesis, but who have normal gastric emptying

Eligibility Criteria

• Age: 5 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• 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).

You may qualify if:

• 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:
• 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)
• 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

You may not qualify if:

• 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:
• Gastrointestinal obstruction confirmed by EGD, UGI, or abdominal CT
• Primary neurological conditions that can cause nausea and vomiting such as increased intracranial pressure, space occupying or inflammatory/infectious lesions
• 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., 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)

Sponsors & Collaborators

• Johns Hopkins Bloomberg School of Public Health: lead
• National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK): collaborator
• Baylor College of Medicine: collaborator
• Nationwide Children's Hospital: collaborator
• Boston Children's Hospital: collaborator
• Harvard Medical School (HMS and HSDM): collaborator

Study Sites (3)

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Baylor College of Medicine/Texas Children's Hospital

Houston, Texas, 77030, United States

Location

Related Links

• Study website

Biospecimen

Retention: SAMPLES WITHOUT DNA

Plasma, serum, peripheral blood mononuclear cells (PBMC), GI mucosal biopsies (in those undergoing upper GI endoscopy), urine, and stool

MeSH Terms

Conditions

Gastroparesis

Condition Hierarchy (Ancestors)

Stomach Diseases; Gastrointestinal Diseases; Digestive System Diseases; Paralysis; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• Robert J Shulman, MD

  Baylor College of Medicine

  STUDY CHAIR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 7, 2018
• First Posted: September 21, 2018
• Study Start: January 23, 2019
• Primary Completion: June 30, 2024
• Study Completion: June 30, 2024
• Last Updated: July 10, 2024

Record last verified: 2024-07

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, ICF, ANALYTIC CODE
• Time Frame: By the end of the funding period.
• Access Criteria: Application through NIDDK Data Repository; IRB approval

Locations

US (3)