PGpR2: GpCRC Pediatric Gastroparesis Registry 2

Sponsor

Johns Hopkins Bloomberg School of Public Health (Other)

Overall Status

Not yet recruiting

CT.gov ID

NCT05981300

Collaborator

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) (NIH), Texas Tech University Health Sciences Center, El Paso (Other), Baylor College of Medicine (Other), Massachusetts General Hospital (Other), Medical College of Wisconsin (Other)

216

Enrollment

Locations

Anticipated Duration (Months)

Patients Per Site

0.8

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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).

Condition or Disease	Intervention/Treatment	Phase
Gastroparesis Gastroparesis-like Syndrome

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.

Study Design

Study Type:

Observational [Patient Registry]

Anticipated Enrollment :

216 participants

Observational Model:

Cohort

Time Perspective:

Prospective

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

Anticipated Study Start Date :

Oct 1, 2023

Anticipated Primary Completion Date :

Aug 1, 2026

Anticipated Study Completion Date :

Jun 1, 2027

Arms and Interventions

Arm	Intervention/Treatment
Ages 8-25 with delayed gastric emptying Participants aged 8-25 with delayed gastric emptying of solids based on gastric emptying scintigraphy
Ages 8-25 with normal gastric emptying Participants aged 8-25 with normal gastric emptying of solids based on gastric emptying scintigraphy

Outcome Measures

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™ [Baseline, 48 weeks]
The PedsQL™ GI Symptoms Scales questionnaire has 65 response items covering 10 dimensions, with each item offered as a better-to-worse 5-point Likert scale choice: 0=never, 1=almost never, 2=sometimes, 3=often, 4=almost always. The item responses are transformed to a worse-to-better order and rescaled to 0-100: 0=almost always, 25= often, 50=sometimes, 75=almost never, 100= never, with higher scores indicating better health-related quality of life (HRQOL) and fewer problems or symptoms.he per-participant PedsQL™ GI Symptoms Scales total score is the sum of the 65 transformed and rescaled item responses. The primary outcome measure is the arithmetic mean of the 65 transformed item responses and is repeated at baseline and 48 weeks for each participant.

Secondary Outcome Measures

Presence or absence of Carnett's sign as assessed by abdominal examination [Baseline]
Presence or absence Carnett's sign for abdominal wall pain at baseline. Carnett's sign is a finding on clinical examination in which (acute) abdominal pain remains unchanged or increases when the muscles of the abdominal wall are tensed. As part of the abdominal examination, the patient is asked to lift the head and shoulders from the examination table to tense the abdominal muscles. An alternative is to ask the patient to raise both legs with straight knees. A positive test indicates the increased likelihood that the abdominal wall and not the abdominal cavity is the source of the pain (for example, due to rectus sheath hematoma instead of appendicitis). A negative Carnett's sign is said to occur when the abdominal pain decreases when the patient is asked to lift the head; this points to an intra-abdominal cause of the pain.
Change in volume (mL) of liquid consumed as assessed by the Water Load Satiety Test [Baseline, 48 weeks]
Change from baseline to 48 weeks in amount of liquid (mL) consumed during the Water Load Satiety Test (WLST) Lower volumes are signs of impaired accommodation.
Change in Pain Catastrophizing Scale scores (PCS) [Baseline, 48 weeks]
The outcome is assessed using change in the total score at 48-weeks minus the baseline score. A negative change indicates reduced pain catastrophizing. PCS total score is computed by summing responses to all 13 items. PCS total scores range from 0 - 52.

Eligibility Criteria

Criteria

Ages Eligible for Study:

8 Years to 25 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Massachusetts General Hospital	Boston	Massachusetts	United States	02114
2	Boston Children's Hospital	Boston	Massachusetts	United States	02115
3	Nationwide Children's Hospital	Columbus	Ohio	United States	43205
4	Texas Tech University Health Science Center	El Paso	Texas	United States	79905
5	Baylor College of Medicine/Texas Children's Hospital	Houston	Texas	United States	77030
6	Medical College of Wisconsin	Milwaukee	Wisconsin	United States	53226

Sponsors and Collaborators

Johns Hopkins Bloomberg School of Public Health
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Texas Tech University Health Sciences Center, El Paso
Baylor College of Medicine
Massachusetts General Hospital
Medical College of Wisconsin

Investigators

Study Chair: Robert J Shulman, MD, Baylor College of Medicine

Study Documents (Full-Text)

None provided.

More Information

Publications

Responsible Party:

Johns Hopkins Bloomberg School of Public Health

ClinicalTrials.gov Identifier:

NCT05981300

Other Study ID Numbers:

15-DK-PGpR2
U01DK112194
U01DK112193
U01DK074035
U24DK074008

First Posted:

Aug 8, 2023

Last Update Posted:

Aug 8, 2023

Last Verified:

Aug 1, 2023

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Keywords provided by Johns Hopkins Bloomberg School of Public Health

Functional Disorder of Gastrointestinal Tract

Gastro-Intestinal Disorder

Additional relevant MeSH terms:

Gastroparesis

Study Results

No Results Posted as of Aug 8, 2023