Initiation of Acid Suppression Therapy Prospective Outcomes for Laryngomalacia

• ClinicalTrials.gov Identifier: NCT04614974
• Recruitment Status: Recruiting
• First Posted: November 4, 2020
• Last Update Posted: November 4, 2021
• Sponsor: Reema Padia
• Information provided by (Responsible Party): Reema Padia, University of Pittsburgh

Study Description

Brief Summary:

Laryngomalacia (LM) is the most common cause of stridor in infants. Symptoms of gastroesophageal reflux (GER) are often seen in the setting of LM; therefore, acid suppression therapy (AST) has been empirically used in the management of this disorder. The investigators recently performed a retrospective chart review assessing improvement of airway and dysphagia symptoms, weight gain, and need for surgery with AST. It was found that there was a similar improvement between LM severity groups and most patients received AST (96.6%). It is unclear if these improvements are due to AST or natural resolution of the disease. With heightened concerns of side effects related to AST in infants, particularly among those born prematurely, judicious use of these medications is needed. The investigators are now performing a prospective study looking at the outcome differences in patients with laryngomalacia who are evaluated by speech language therapy (SLP) alone versus those with SLP evaluation and acid suppression therapy (famotidine).

Condition or disease	Intervention/treatment	Phase
Laryngomalacia; Gastro Esophageal Reflux	Drug: Famotidine; Other: Speech Language Therapy	Phase 1; Phase 2

Detailed Description:

Purpose: To determine the outcome differences in patients 6 months and younger with laryngomalacia and dysphagia who are evaluated by speech language therapy (SLP) alone versus those with both SLP evaluation and acid suppression therapy (AST) (famotidine)

Hypothesis: There will be no differences in outcomes between those that had SLP alone versus those that had both SLP and AST.

Laryngomalacia (LM) is the most common cause of stridor in infants. Symptoms of gastroesophageal reflux (GER) are often seen in the setting of LM; therefore, acid suppression therapy (AST) has been empirically used in the management of this disorder. However, there is no gold standard in treating mild and moderate LM patients and therefore this study will help establish guidelines for treatment.

A medical chart review will be performed to assess airway and dysphagia symptom improvement from consult to the 3-month follow up appointment and then up to a year. The Pittsburgh Airway Symptom Score (PASS) questionnaire and the Infant Gastroesophageal Reflux Questionnaire (I-GERQ-R) will be given to families at the consult and at the 3-month follow up appointment for caregiver assessment. The physician will then perform the standard procedures at the clinic appointment. The Flexible Laryngoscopy Findings sheet will be filled out in conjunction with the clinic procedures for objective data. The surveys will then be scored to determine true GERD (score >=16) and severe laryngomalacia (LM) (RED questions on the PASS), which would further exclude these patients. There are 12 total questions on the I-GERQ-R. On the PASS, questions 1 & 2 are in the GREEN category and signifies mild LM, YELLOW signifies moderate LM, and RED is severe LM. On the PASS questionnaire, "Yes" to either #1 or #2 and nothing else is mild LM, "yes" to at least one #3-5 and none of #6-10 is moderate LM, and "yes" to any of the #6-10" indicates severe LM. There are 10 total questions on the PASS. Mild and moderate LM patients will be block randomized the day before the appointment to receive speech language therapy alone or speech language therapy with famotidine (Pepcid). Both treatments are standard of care in these patients. Speech language therapy (feeding therapy) is part of the normal clinic visit for LM patients. These patients will then be re-evaluated at their follow up appointment in 3 months (+/- 1 month). The families will take the PASS and I-GERQ-R surveys again to determine LM severity.

Study Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 160 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Intervention Model Description: Experimental, open-label randomized control trial
• Masking: None (Open Label)
• Masking Description: Open Label
• Primary Purpose: Treatment
• Official Title: Initiation of Acid Suppression Therapy Prospective Outcomes for Laryngomalacia
• Actual Study Start Date: November 18, 2020
• Estimated Primary Completion Date: December 31, 2022
• Estimated Study Completion Date: March 31, 2023

Arms and Interventions

Arm	Intervention/treatment
Experimental: Speech Language Therapy Alone; Patients in this group will receive a routine swallowing evaluation by a speech language pathologist. Patient caregivers will fill out the Infant Gastroesophageal Reflux Questionnaire (I-GERQ-R) and the Pittsburgh Airway Symptom Score (PASS) the day of the appointment and at the 3-month follow up appointment.	Other: Speech Language Therapy; Speech Language Therapy (feeding therapy) will be provided by a speech language pathologist to assess feeding and swallowing.
Active Comparator: Speech Language Therapy and Acid Suppression Therapy; Patients in this group will receive a routine swallowing evaluation by a speech language pathologist and famotidine (acid suppression therapy). Patient caregivers will fill out the Infant Gastroesophageal Reflux Questionnaire (I-GERQ-R) and the Pittsburgh Airway Symptom Score (PASS) the day of the appointment and at the 3-month follow up appointment.	Drug: Famotidine; Famotidine will be prescribed based on patients' weight. Caregivers will purchase this medication and dosage will be given to families in easy-to-understand language.; Other: Speech Language Therapy; Speech Language Therapy (feeding therapy) will be provided by a speech language pathologist to assess feeding and swallowing.

Outcome Measures

Primary Outcome Measures :

1. Airway symptom score change from consult (baseline) to 3 month follow-up appointment [ Time Frame: 3 months ]
   Score change from pre to post surveys on the Pittsburgh Airway Symptom Score (PASS) questionnaire. The PASS is on a scale from 0-10 with a higher score indicating a worse outcome. This outcome will be assessed at the consult and the 3 month follow-up appointment.
2. Dysphagia symptom score change from consult (baseline) to 3 month follow-up appointment [ Time Frame: 3 months ]
   Score change from pre to post surveys on the Pittsburgh Airway Symptom Score (PASS) questionnaire. The PASS is on a scale from 0-10 with a higher score indicating a worse outcome. This outcome will be assessed at the consult and the 3 month follow-up appointment.
3. Change in incidence of airway symptoms from consult up to 1 year assessed via electronic medical chart review [ Time Frame: 1 year ]
   Change in incidence of airway symptoms from consult up to 1 year assessed via electronic medical chart review. Notes from the Department of Otolaryngology will be reviewed which include reported symptoms from caregivers and symptoms seen upon exam. Airway symptoms included periods of apnea, chest wall retractions, cyanosis, stridor, noisy breathing, and increased respiratory rate.
4. Change in incidence of dysphagia symptoms from consult up to 1 year assessed via electronic medical chart [ Time Frame: 1 year ]
   Change in incidence of dysphagia symptoms from consult up to 1 year assessed via electronic medical chart review. Notes from the Department of Otolaryngology will be reviewed which include reported symptoms from caregivers and symptoms seen upon exam. Dysphagia symptoms included choking, coughing, gagging with feeds and/or emesis after feeds.

Secondary Outcome Measures :

1. Weight (kg) from consult up to 1 year [ Time Frame: 1 year ]
   Weight change in kilograms assessed from medical chart review from consult up to 1 year
2. Infant Gastroesophageal Reflux Questionnaire (I-GERQ-R) score change from consult (baseline) to 3 month follow-up appointment [ Time Frame: 3 months ]
   Score change from pre to post survey on the I-GERQ-R. There are 12 questions on the surveys on a scale of 0-42. A higher score indicates a worse outcome. Those with a score of >=16 on this survey at the initial consult are excluded from the study, indicating true gastroesophageal reflux disease (GERD). This outcome will be assessed at the consult and the 3 month follow-up appointment.
3. Number of participants with the need for supraglottoplasty surgery (escalation of treatment) up to 1 year [ Time Frame: 1 year ]
   The need for escalation of treatment with supraglottoplasty surgery will be assessed through medical chart review up to 1 year.
4. Number of participants with the need for acid suppression therapy medication (famotidine) from speech language therapy alone group from the day after the consult up to the 3 month follow up appointment [ Time Frame: 3 months ]
   The speech language therapy alone group will be assessed for the need for a prescription for acid suppression therapy (famotidine) from the day after the consult up to 1 year.
5. Number of participants with each type of laryngomalacia (Types 1-3) found on the Flexible Laryngoscopy procedure at the consult [ Time Frame: At initial consult ]
   All patients will be scoped with a flexible laryngoscopy at the initial consult and the type of laryngomalacia (Types 1-3) will be noted.
6. Number of participants with the need for a different acid suppression therapy medication (other than famotidine) from the day after the consult up to the 3 month follow up appointment [ Time Frame: 3 months ]
   Both groups will be assessed for the need for a different acid suppression therapy medication (other than famotidine) from the day after the consult up to the 3 month follow up appointment.

Eligibility Criteria

• Ages Eligible for Study: up to 6 Months (Child)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

• Pediatric patients ages 0 to 6 months who do need meet the criteria at the initial appointment for supraglottoplasty
• Seen in University of Pittsburgh Medical Center (UPMC) Children's Hospital of Pittsburgh (CHP) Otolaryngology Department
• Laryngomalacia without prolonged (>20 seconds) cyanosis, apnea, nor failure to thrive.

Exclusion Criteria:

• Children over the age of 6 months old will be excluded from participation.
• Premature infants (<37 weeks gestation)
• Patients with lung disease.
• Laryngomalacia with prolonged (>20 seconds) cyanosis, apnea, and failure to thrive
• Sleep induced laryngomalacia
• Patients with craniofacial abnormalities
• Patients with a syndrome
• Patients with additional airway abnormalities, seen before or at consult
• Patients with symptoms that necessitate surgery
• Patients with a prior cardiac surgery
• Patients with AST prescribed prior to the initial otolaryngology consult.

Contacts and Locations

Contacts

Contact: Jennifer L. McCoy, MA; 412-692-9879; nelsonjl2@upmc.edu

Contact: Amber D. Shaffer, PhD; 412-692-6874; shafferad@upmc.edu

Locations

United States, Pennsylvania

UPMC Children's Hospital of Pittsburgh; Recruiting

Pittsburgh, Pennsylvania, United States, 15224

Contact: Jennifer McCoy, MA 412-692-9879 nelsonjl2@upmc.edu

Contact: Amber Shaffer, PhD 412-692-6874 shafferad@upmc.edu

Sponsors and Collaborators

Reema Padia

Investigators

• Principal Investigator: Reema Padia, MD; Division of Pediatric Otolaryngology, Children's Hospital of Pittsburgh

More Information

Publications:

Rosbe KW, Kenna MA, Auerbach AD. Extraesophageal reflux in pediatric patients with upper respiratory symptoms. Arch Otolaryngol Head Neck Surg. 2003 Nov;129(11):1213-20. Review.

Hartl TT, Chadha NK. A systematic review of laryngomalacia and acid reflux. Otolaryngol Head Neck Surg. 2012 Oct;147(4):619-26. Epub 2012 Jun 27. Review.

Bibi H, Khvolis E, Shoseyov D, Ohaly M, Ben Dor D, London D, Ater D. The prevalence of gastroesophageal reflux in children with tracheomalacia and laryngomalacia. Chest. 2001 Feb;119(2):409-13.

Landry AM, Thompson DM. Laryngomalacia: disease presentation, spectrum, and management. Int J Pediatr. 2012;2012:753526. doi: 10.1155/2012/753526. Epub 2012 Feb 27.

Thompson DM. Laryngomalacia: factors that influence disease severity and outcomes of management. Curr Opin Otolaryngol Head Neck Surg. 2010 Dec;18(6):564-70. doi: 10.1097/MOO.0b013e3283405e48. Review.

• Responsible Party: Reema Padia, Assistant Professor, University of Pittsburgh
• ClinicalTrials.gov Identifier: NCT04614974
• Other Study ID Numbers: STUDY20090193
• First Posted: November 4, 2020
• Last Update Posted: November 4, 2021
• Last Verified: November 2021

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Product Manufactured in and Exported from the U.S.: No

Keywords provided by Reema Padia, University of Pittsburgh:

Laryngomalacia; Symptom improvement; Famotidine; Speech Language Therapy

Additional relevant MeSH terms:

Laryngomalacia; Gastroesophageal Reflux; Esophageal Motility Disorders; Deglutition Disorders; Esophageal Diseases; Gastrointestinal Diseases; Digestive System Diseases; Cartilage Diseases; Musculoskeletal Diseases; Laryngeal Diseases; Respiratory Tract Diseases; Otorhinolaryngologic Diseases; Musculoskeletal Abnormalities; Congenital Abnormalities; Connective Tissue Diseases; Famotidine; Anti-Ulcer Agents; Gastrointestinal Agents; Histamine H2 Antagonists; Histamine Antagonists; Histamine Agents; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Physiological Effects of Drugs