NCT04614974

Initiation of Acid Suppression Therapy Prospective Outcomes for Laryngomalacia

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 65 (actual)
• Sponsor: Reema Padia · Academic / Other
• Sex: All
• Age: 0 Months – 6 Months
• Healthy volunteers: Not accepted

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).

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. Initially, primary outcome #2 was "Dysphagia symptom score change from consult (baseline) to 3 month follow-up appointment." This is the same as the original secondary outcome measure #6 "Infant Gastroesophageal Reflux Questionnaire (I-GERQ-R) score change from consult (baseline) to 3 month follow-up appointment" (I-GERQ-R is the dysphagia symptom score used in this study). Therefore, these were consolidated to a single primary outcome measure "Infant Gastroesophageal Reflux Questionnaire (I-GERQ-R) score change from consult (baseline) to 3 month follow-up appointment." This change was made after study and data collection completion, at the time of results reporting.

Conditions

• Laryngomalacia
• Gastro Esophageal Reflux

Interventions

Type	Name	Description
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.

Timeline

• Start date: 2020-11-18
• Primary completion: 2024-05-17
• Completion: 2024-05-17
• First posted: 2020-11-04
• Last updated: 2025-07-20
• Results posted: 2025-07-20

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study