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Pediatric Gastroparesis Registry (PGpR)

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ClinicalTrials.gov Identifier: NCT03680820
Recruitment Status : Not yet recruiting
First Posted : September 21, 2018
Last Update Posted : September 21, 2018
Sponsor:
Information provided by (Responsible Party):
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

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).

Condition or disease
Gastroparesis Gastroparesis-like Syndrome

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

Study Type : Observational
Estimated Enrollment : 420 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Pediatric Gastroparesis Registry: Pediatric Registry of Patients With Suspected Gastroparesis
Estimated Study Start Date : September 30, 2018
Estimated Primary Completion Date : October 30, 2022
Estimated Study Completion Date : December 30, 2022

Resource links provided by the National Library of Medicine


Group/Cohort
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



Primary Outcome Measures :
  1. Change in water load satiety volume consumed [ Time Frame: 24 weeks ]
    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).


Secondary Outcome Measures :
  1. Change in Pediatric Quality of Life (PedQL) Generic Core Scale [ Time Frame: 48 weeks ]
    Pediatric Quality of Life Inventory (PedsQL) Generic Core Scale is completed by the child, and is composed of 23 items comprising 4 dimensions: Physical Functioning, Emotional Functioning, Social Functioning, and School Functioning. Each item is answered using a 3-point scale: 0 (Not at all), 2 (Sometimes), 4 (A lot). Items are reversed scored and linearly transformed to a scale from 0 to 100, with higher scores indicating lower problems. The Total Score is calculated as the sum of all the items over the number of items answered on all the scales. The outcome is assessed by calculating the change in Total Score (48 weeks - baseline).

  2. Change in Pediatric Quality of Life (PedQL) Gastrointestinal Symptom Scale [ Time Frame: 48 weeks ]
    Pediatric Quality of Life (PedQL) Gastrointestinal Symptoms Scale is completed by the child, and is composed of 58 items comprising 10 dimensions: Stomach Pain and Hurt, Stomach Discomfort When Eating, Food and Drink Limits, Trouble Swallowing, Heart Burn and Reflux, Nausea and Vomiting, Gas and Bloating, Constipation, Blood in Poop, and Diarrhea. Each item is answered using a 3-point scale: 0 (Not at all), 2 (Sometimes), 4 (A lot). Items are reversed scored and linearly transformed to a scale from 0 to 100, with higher scores indicating lower problems. The Symptom Total Scales Score is calculated as the sum of the items answered in the 10 dimensions. The outcome is assessed by calculating the change in Total Score (48 weeks - baseline).


Biospecimen Retention:   Samples Without DNA
Plasma, serum, peripheral blood mononuclear cells (PBMC), GI mucosal biopsies (in those undergoing upper GI endoscopy), urine, and stool


Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 6 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).
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 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03680820


Contacts
Contact: Laura Miriel 410-955-4165 laura.miriel@jhu.edu

Locations
United States, Massachusetts
Boston Children's Hospital Not yet recruiting
Boston, Massachusetts, United States, 02115
Contact: Samuel Nurko, MD       Samuel.Nurko@childrens.harvard.edu   
United States, Ohio
Nationwide Children's Hospital Not yet recruiting
Columbus, Ohio, United States, 43205
Contact: Kent Williams, MD       Kent.Williams@nationwidechildrens.org   
Contact: Beverly Galliers       Beverly.Galliers@nationwidechildrens.org   
United States, Texas
Baylor College of Medicine/Texas Children's Hospital Not yet recruiting
Houston, Texas, United States, 77030
Contact: Robert J Shulman, MD    713-798-7145    rshulman@bcm.edu   
Contact: Heather Charron    713-798-0381    charron@bcm.edu   
Sponsors and Collaborators
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Investigators
Study Director: Jose Serrano, MD, PhD National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Study Director: Frank Hamilton, MD, MPH National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Study Chair: Robert J Shulman, MD Baylor College of Medicine

Additional Information:
Responsible Party: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
ClinicalTrials.gov Identifier: NCT03680820     History of Changes
Other Study ID Numbers: 8 DK PGpR
U01DK112194 ( U.S. NIH Grant/Contract )
First Posted: September 21, 2018    Key Record Dates
Last Update Posted: September 21, 2018
Last Verified: September 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Public use complete database will be submitted to the NIDDK Data Repository
Supporting Materials: Study Protocol
Informed Consent Form (ICF)
Analytic Code
Time Frame: By the end of the funding period.
Access Criteria: Application through NIDDK Data Repository; IRB approval
URL: https://repository.niddk.nih.gov/home/

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Gastroparesis
Stomach Diseases
Gastrointestinal Diseases
Digestive System Diseases
Paralysis
Neurologic Manifestations
Signs and Symptoms