Initiation of Acid Suppression Therapy Prospective Outcomes for Laryngomalacia

NCT04614974 | Terminated Phase 1 Results FDA Drug

• 1 other identifier: STUDY20090193
• Study Type: interventional
• Target: 65
• Locations: 1 country
• Sites: 1

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).

Conditions

Laryngomalacia; Gastro Esophageal Reflux

Keywords

Laryngomalacia; Symptom improvement; Famotidine; Speech Language Therapy

Outcome Measures

Primary Outcomes (4)

• Airway Symptom Score Change From Consult (Baseline) to 3 Month Follow-up Appointment

  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 months

• Infant Gastroesophageal Reflux Questionnaire (I-GERQ-R) Score Change From Consult (Baseline) to 3 Month Follow-up Appointment

  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 months

• Change in Prevalence of Airway Symptoms From Consult up to 1 Year Assessed Via Electronic Medical Chart Review

  Change in prevalence 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.

  1 year

• Change in Prevalence of Dysphagia Symptoms From Consult up to 1 Year Assessed Via Electronic Medical Chart

  Change in prevalence 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.

  1 year

Secondary Outcomes (5)

• Weight (kg/Month) From Consult up to 1 Year

  1 year

• Number of Participants With the Need for Supraglottoplasty Surgery (Escalation of Treatment) up to 1 Year

  1 year

• 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 1 Year

  1 year

• Number of Participants With Each Type of Laryngomalacia (Types 1-3) Found on the Flexible Laryngoscopy Procedure at the Consult

  At initial consult

• 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

  3 months

Study Arms (2)

Speech Language Therapy Alone

EXPERIMENTAL

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 and Acid Suppression Therapy

ACTIVE COMPARATOR

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; Other: Speech Language Therapy

Interventions

Famotidine DRUG

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.

Speech Language Therapy and Acid Suppression Therapy

Speech Language Therapy OTHER

Speech Language Therapy (feeding therapy) will be provided by a speech language pathologist to assess feeding and swallowing.

Speech Language Therapy Alone; Speech Language Therapy and Acid Suppression Therapy

Eligibility Criteria

• Age: 0 Months - 6 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Reema Padia: lead

Study Sites (1)

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

Location

Related Publications (6)

• 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. doi: 10.1001/archotol.129.11.1213.

  PMID: 14623753 BACKGROUND

• Hartl TT, Chadha NK. A systematic review of laryngomalacia and acid reflux. Otolaryngol Head Neck Surg. 2012 Oct;147(4):619-26. doi: 10.1177/0194599812452833. Epub 2012 Jun 27.

  PMID: 22745201 BACKGROUND

• 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. doi: 10.1378/chest.119.2.409.

  PMID: 11171716 BACKGROUND

• 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.

  PMID: 22518182 BACKGROUND

• 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.

  PMID: 20962644 BACKGROUND

• Shaffer AD, Balogun Z, Tobey ABJ, Maguire RC, Simons JP, Dohar JE, Mccoy JL, Rushchak MV, Padia R. Acid Suppression in Mild-Moderate Laryngomalacia Without GERD: A Randomized Controlled Trial. Laryngoscope. 2025 Aug 5. doi: 10.1002/lary.32471. Online ahead of print.

  PMID: 40762274 DERIVED

MeSH Terms

Conditions

Laryngomalacia; Gastroesophageal Reflux; Communication Disorders

Interventions

Famotidine; Speech Therapy

Condition Hierarchy (Ancestors)

Cartilage Diseases; Musculoskeletal Diseases; Laryngeal Diseases; Respiratory Tract Diseases; Otorhinolaryngologic Diseases; Musculoskeletal Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Esophageal Motility Disorders; Deglutition Disorders; Esophageal Diseases; Gastrointestinal Diseases; Digestive System Diseases; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Thiazoles; Sulfur Compounds; Organic Chemicals; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Rehabilitation of Speech and Language Disorders; Rehabilitation; Aftercare; Continuity of Patient Care; Patient Care; Therapeutics

Limitations and Caveats

Early termination leading to small numbers of subjects analyzed

Results Point of Contact

• Title: Dr. Amber Shaffer
• Organization: UPMC Children's Hospital of Pittsburgh

shafferad@upmc.edu

412-692-6874

Study Officials

• Reema Padia, MD

  Division of Pediatric Otolaryngology, Children's Hospital of Pittsburgh

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Masking Details: Open Label
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Assistant Professor

Model Details: Experimental, open-label randomized control trial

Study Record Dates

• First Submitted: October 22, 2020
• First Posted: November 4, 2020
• Study Start: November 18, 2020
• Primary Completion: May 17, 2024
• Study Completion: May 17, 2024
• Last Updated: July 20, 2025
• Results First Posted: July 20, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)