GastroparesisPGpRNCT03680820
Pediatric Gastroparesis Registry
An observational study of Gastroparesis and Gastroparesis-like Syndrome, run by Johns Hopkins Bloomberg School of Public Health at 3 sites in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 5 to 17 years
- Conditions the study is for
- Gastroparesis, Gastroparesis-like Syndrome
- How many people
- 147 participants
- Where
- 3 sitesUnited States
- Study dates
- 23 Jan 2019to 30 Jun 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 17 years
- All sexes
- No healthy volunteers
- Child
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).
Who may be able to join
- 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
Who cannot join
- 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:
Show all 19Show fewer
- 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)
- 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
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Boston Children's Hospital
Boston, Massachusetts, 02115
Nationwide Children's Hospital
Columbus, Ohio, 43205
Baylor College of Medicine/Texas Children's Hospital
Houston, Texas, 77030
About this study
Official title: Pediatric Gastroparesis Registry: Pediatric Registry of Patients With Suspected Gastroparesis
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).
Read the 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:
- Demographic, clinical, psychological, nutritional characteristics, physiological measures, and serial assessments of symptoms over 3 years while receiving clinical care;
- 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:
- To determine what factors, if any, play a role in the outcomes of children who do not improve within two years of diagnosis
- 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
- To assess several areas of gastric motility in pediatric patients with symptoms of gastroparesis (e.g., intragastric meal distribution, global gastric emptying)
- To capture the clinical symptomatic course (outcome) of pediatric patients followed in the registry
- To determine whether gastroparesis symptoms are correlated with gastric emptying in a pediatric population
- To characterize abdominal pain in patients with gastroparesis and gastroparesis-like syndrome by using questionnaires, water load satiety testing and quantitative sensory testing (QST)
- Determine the prevalence of hypermobility spectrum disorders in pediatric patients with gastroparesis
- To determine if the volume of water consumed during the water load satiety test is an indirect measure of gastric accommodation
- To determine if the water load satiety test is associated with an increased severity of gastroparesis symptoms (fullness, bloating, abdominal pain, etc).
- To compare the intragastric meal distribution during scintigraphy to symptoms of early satiety, postprandial fullness in patients with symptoms of gastroparesis
- Define immune cell profiles, cytokine induction and epigenetic changes in diabetic and idiopathic gastroparesis
- Conditions
- Gastroparesis, Gastroparesis-like Syndrome
- Medical subject headings
- Gastroparesis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 24 weeks
Secondary outcomes (2)
Change in Pediatric Quality of Life (PedQL) Generic Core Scale
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).
Measured: 48 weeks
Change in Pediatric Quality of Life (PedQL) Gastrointestinal Symptom Scale
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).
Measured: 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Sep 2018
- Study start23 Jan 2019
- Primary completion30 Jun 2024
- Study completion30 Jun 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for GastroparesisStudies of the same condition, open and under way.
- More trials run by Johns Hopkins Bloomberg School of Public HealthThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 147 participants (actual)
Sponsor and investigators
- Lead sponsor
- Johns Hopkins Bloomberg School of Public Health
- Sponsor type
- University, hospital or other
- Collaborators
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), Baylor College of Medicine, Nationwide Children's Hospital, Boston Children's Hospital, Harvard Medical School (HMS and HSDM)
- Responsible party
- Sponsor
- Investigators
- Robert J Shulman, MD, Study chair, Baylor College of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03680820
- Sponsor's protocol ID
- 8 DK PGpR
- Other IDs
- U01DK112194, U24DK074008
- Acronym
- PGpR
- Status last verified
- Jul 2024
- First submitted
- 7 Sep 2018
- Last updated
- 10 Jul 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Public use complete database will be submitted to the NIDDK Data Repository
Study at a glance
From the registry record, last updated by the sponsor on 10 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pediatric Gastroparesis Registry: Pediatric Registry of Patients With Suspected Gastroparesis
- ClinicalTrials.gov ID
- NCT03680820
- Acronym / sponsor ID
- PGpR / 8 DK PGpR
- Conditions
- Gastroparesis, Gastroparesis-like Syndrome
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 147 participants (actual)
- Lead sponsor
- Johns Hopkins Bloomberg School of Public HealthUniversity, hospital or other
- Collaborators
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), Baylor College of Medicine, Nationwide Children's Hospital, Boston Children's Hospital, Harvard Medical School (HMS and HSDM)
- Study start
- 23 Jan 2019 (actual)
- Primary completion
- 30 Jun 2024 (actual)
- Study completion
- 30 Jun 2024 (actual)
- First posted
- 21 Sep 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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:
- Demographic, clinical, psychological, nutritional characteristics, physiological measures, and serial assessments of symptoms over 3 years while receiving clinical care;
- 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:
- To determine what factors, if any, play a role in the outcomes of children who do not improve within two years of diagnosis
- 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
- To assess several areas of gastric motility in pediatric patients with symptoms of gastroparesis (e.g., intragastric meal distribution, global gastric emptying)
- To capture the clinical symptomatic course (outcome) of pediatric patients followed in the registry
- To determine whether gastroparesis symptoms are correlated with gastric emptying in a pediatric population
- To characterize abdominal pain in patients with gastroparesis and gastroparesis-like syndrome by using questionnaires, water load satiety testing and quantitative sensory testing (QST)
- Determine the prevalence of hypermobility spectrum disorders in pediatric patients with gastroparesis
- To determine if the volume of water consumed during the water load satiety test is an indirect measure of gastric accommodation
- To determine if the water load satiety test is associated with an increased severity of gastroparesis symptoms (fullness, bloating, abdominal pain, etc).
- To compare the intragastric meal distribution during scintigraphy to symptoms of early satiety, postprandial fullness in patients with symptoms of gastroparesis
- Define immune cell profiles, cytokine induction and epigenetic changes in diabetic and idiopathic gastroparesis
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| 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 |
Who may qualify
- Ages
- 5 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- 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).
Key inclusion criteria (6 listed)
- 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:
- Age at least 5 years, and under 18 years at initial screening visit
Key exclusion criteria (19 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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).
2 secondary outcomes
Change in Pediatric Quality of Life (PedQL) Generic Core Scale
Time frame: 48 weeks
Change in Pediatric Quality of Life (PedQL) Gastrointestinal Symptom Scale
Time frame: 48 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jul 2024.
- Study websiteLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Public use complete database will be submitted to the NIDDK Data Repository
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Robert J Shulman, MD study chair
Baylor College of Medicine
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Gastroparesis and Gastroparesis-like Syndrome, run by Johns Hopkins Bloomberg School of Public Health at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in water load satiety volume consumed (time frame: 24 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Jul 2024.