Use of Intrapyloric Botulinum Injections in Children

This study aims to evaluate the effect of intrapyloric botulinum toxin in children with feeding disorders.

Study Overview

• Status: Unknown
• Conditions: Vomiting; Gastroparesis; Feeding Disorders

Detailed Description

Chronic vomiting and feeding difficulties are common in young children and have a negative physical and psychosocial impact for patients and families. Currently there is no straightforward treatment algorithm for these issues, and management often involves multiple medication trials and procedures. Intrapyloric botulinum toxin injection has been proposed as a treatment for nausea and vomiting in adults, but there is minimal prior research on use in children and no prior research on use in children with feeding disorders. The aims of this study are: (1) to determine the efficacy of intrapyloric botulinum toxin injection for reducing gastrointestinal symptoms in children, (2) to determine the efficacy of intrapyloric botulinum injections for improving feeding outcomes in children, and (3) to define predictors of response to intrapyloric botulinum toxin injection.

• Study Type: Observational
• Enrollment (Anticipated): 100

Contacts and Locations

• Study Contact: Name: Suzanna Hirsch, MD; Phone Number: 617-355-6058; Email: suzanna.hirsch@childrens.harvard.edu
• Study Contact Backup: Name: Rachel Rosen, MD, MPH; Email: rachel.rosen@childrens.harvard.edu

Study Locations

• United States
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Recruiting
      • Boston Children's Hospital
      • Contact: Suzanna Hirsch, MD; Email: suzanna.hirsch@childrens.harvard.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 month to 18 years (Child, Adult)
• Accepts Healthy Volunteers: N/A
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Patients from our tertiary care center who are undergoing upper endoscopy with or without intrapyloric botulinum injections.

Description

Inclusion Criteria:

• Patients with chronic nausea, vomiting, or feeding intolerance undergoing upper endoscopy with or without intrapyloric botulinum injections.
• Age between 30 days and 18 years.

Exclusion Criteria:

• Significant uncontrolled inflammation on upper or lower endoscopy
• Known inflammatory bowel disease
• Uncontrolled thyroid disease
• Current opioid use

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort
Botulinum; We will follow for symptom improvement over time patients with nausea, vomiting, feeding intolerance, or other signs of gastric dysfunction who were determined by their gastroenterologist to require upper endoscopy with intrapyloric botulinum injections and if clinically applicable endoscopic functional luminal imaging probe (EndoFLIP). The dosage will be determined by the patient's physician.
Control; We will follow for symptom improvement over time patients with nausea, vomiting, feeding intolerance, or other signs of gastric dysfunction who were determined by their gastroenterologist to require upper endoscopy without intrapyloric botulinum injections.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Degree of improvement in quality of life	Quality of life will be measured with the Pediatric Quality of Life Inventory (PedsQL). This 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). Scores range 0 to 100, with lower scores indicating worse quality of life. The outcome will be assessed by calculating the change in score from baseline to 4 weeks.	4 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Degree of symptomatic improvement	Symptoms will be measured with the Gastroparesis Cardinal Symptom Index (GCSI). The GCSI is based on three subscales: post-prandial fullness/early satiety (4 items); nausea/vomiting (3 items), and bloating (2 items). A six-point Likert response scale, ranging from 0 (none) to 5 (very severe) is used to rate severity of each symptom. Scores range from 0 to 45, with higher scores indicating worse symptoms. This outcome will be assess by calculating the change in score from baseline to 4 weeks.	4 weeks

Collaborators and Investigators

• Sponsor: Boston Children's Hospital
• Investigators: Principal Investigator: Rachel Rosen, MD, MPH, Boston Children's Hospital

Study record dates

Study Major Dates

• Study Start (Actual): March 10, 2020
• Primary Completion (Anticipated): May 1, 2021
• Study Completion (Anticipated): May 1, 2021

Study Registration Dates

• First Submitted: January 23, 2020
• First Submitted That Met QC Criteria: January 30, 2020
• First Posted (Actual): January 31, 2020

Study Record Updates

• Last Update Posted (Actual): December 8, 2020
• Last Update Submitted That Met QC Criteria: December 4, 2020
• Last Verified: December 1, 2020

More Information

Terms related to this study

• Keywords: Botulinum Toxin
• Additional Relevant MeSH Terms: Mental Disorders; Digestive System Diseases; Neurologic Manifestations; Signs and Symptoms, Digestive; Gastrointestinal Diseases; Stomach Diseases; Paralysis; Vomiting; Feeding and Eating Disorders; Gastroparesis
• Other Study ID Numbers: P00033019

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No