In Infants With Laryngomalacia, Does Acid-Blocking Medication Improve Respiratory Symptoms?

Study Overview
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Brief Summary
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All neonates, ages 0 to 4 months, presenting to LPCH pediatric ENT clinic for airway difficulties or stridor will be screened for inclusion. As is consistent with an acceptable standard of medical care, these children will undergo a flexible nasal endoscopic exam to make the diagnosis of laryngomalacia, as well as be weighed and a breastfeeding history taken. If laryngomalacia is present, the study staff with then administer the Infant Gastroesophageal Reflux Questionnaire (IGERQ) and an airway symptoms questionnaire (ASQ). Those babies with an IGERQ score of less than sixteen (no more than mild reflux) and an ASQ score greater than six will be eligible for randomization. The patient will then be randomly placed in the control group (placebo) or the intervention group (ranitidine 2mg/kg every 12 hours or famotidine 0.5 mg/kg daily). Patients will stay on medication for a minimum of 6 months, or until symptoms resolve. Patients will be seen in follow up at 1, 2, 3, 4, 5, 6, 8 and 10 months. At which time I-GERQ, ASQ and weights will be taken. The primary outcome measure will be the time for the ASQ score to drop to normal on ranitidine or famotidine versus placebo. A secondary outcome will be weight gain in percentile. If the patient's I-GERQ score goes above 16 at any time in the study, the patient will be crossed over to the treatment arm and started on medical treatment.

Detailed Description
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Laryngomalacia, or a soft, floppy upper airway, is the most common cause of noisy breathing in neonates. It is caused by a combination of factors including neuromuscular weakness, cartilaginous inadequacy, and anatomic abnormalities in the voice box. The majority of affected babies' symptoms resolve by one year without intervention, but around 5% of cases require surgical intervention to treat failure to thrive or obstructive sleep apnea. Many babies with laryngomalacia are treated empirically with H2 blockers such as ranitidine or famotidine to prevent reflux of stomach acid from causing further inflammation in an already compromised upper airway. However, to date no study has been performed to show a definitive benefit of these medications. The investigators hope to determine whether H2 blockers such as ranitidine and famotidine improve airway symptoms in babies with laryngomalacia. This will have an effect on practice guideline for one of the most common problems encountered in pediatric otolaryngology. All neonates, ages 0 to 4 months, presenting to LPCH pediatric ENT clinic for airway difficulties or stridor will be screened for inclusion. As is consistent with an acceptable standard of medical care, these children will undergo a flexible nasal endoscopic exam to make the diagnosis of laryngomalacia, as well as be weighed and a breastfeeding history taken. If laryngomalacia is present, the study staff with then administer the Infant Gastroesophageal Reflux Questionnaire (IGERQ) and an airway symptoms questionnaire (ASQ). Those babies with an IGERQ score of less than sixteen (no more than mild reflux) and an ASQ score greater than six will be eligible for randomization. The patient will then be randomly placed in the control group (placebo) or the intervention group (ranitidine 2mg/kg every 12 hours or famotidine 0.5 mg/kg daily). Patients will stay on medication for a minimum of 6 months, or until symptoms resolve. Patients will be seen in follow up at 1, 2, 3, 4, 5, 6, 8 and 10 months. At which time I-GERQ, ASQ and weights will be taken. The primary outcome measure will be the time for the ASQ score to drop to normal on ranitidine or famotidine versus placebo. A secondary outcome will be weight gain in percentile. If the patient's I-GERQ score goes above 16 at any time in the study, the patient will be crossed over to the treatment arm and started on medical treatment.

Official Title
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In Infants With Symptoms of Tracheomalacia or Laryngomalacia, Does Acid-Blocking Medication Improve Respiratory Symptoms? A Randomized, Controlled Trial

Conditions
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Laryngomalacia, Acid Reflux, Stridor

Intervention / Treatment
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* Drug: ranitidine or famotidine
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: age 0 - 4 months old stridor and flexible laryngoscopy demonstrating laryngomalacia airway symptom score over 4 only has physiologic GER (I-GERQ < 16) Exclusion Criteria: requiring surgery for LGM other airway dz seen on flexible laryngoscopy history of or already on PPI therapy minimal/mild airway symptoms (airway score < 4) pathologic GERD (I-GERQ greater than 16) - These pts cannot be randomized because this is the standard score in Pediatric GI literature strongly indicating anti-reflux meds premature birth (< 36 weeks)

Ages Eligible for Study
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Maximum Age: 4 Months

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Placebo Comparator: H2 blocker versus placebo<br>The patient will then be randomly placed in the control group (placebo) or the intervention group (ranitidine 2mg/kg every 12 hours or famotidine 0.5 mg/kg daily). Patients will stay on medication for a minimum of 6 months, or until symptoms resolve. Patients will be seen in follow up at 1, 2, 3, 4, 5, 6, 8 and 10 months. At which time I-GERQ, ASQ and weights will be taken. The primary outcome measure will be the time for the ASQ score to drop to normal on ranitidine or famotidine versus placebo. | Drug: ranitidine or famotidine<br>* Patients with symptomatic laryngomalacia will receive ranitidine, famotidine, or placebo medications in order to see if these medications help their symptoms to resolve.<br>Drug: Placebo<br>* Patients with symptomatic laryngomalacia who meet inclusion criteria will be randomized to receive placebo, ranitidine or famotidine, in order to see if these medications help their symptoms to resolve.<br>|
| Active Comparator: Treatment arm<br>Patients who develop severe airway symptoms while they are in the H2 blocker versus placebo arm will then cross over to the treatment arm of the study. These patients will exit randomization and be unblinded. These patients will all be given H2 blockers, and the effects of the H2 blockers will be monitored and studied. Alternatively, patients' families may also elect to undergo surgery to treat laryngomalacia. | Drug: ranitidine or famotidine<br>* Patients with symptomatic laryngomalacia will receive ranitidine, famotidine, or placebo medications in order to see if these medications help their symptoms to resolve.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Time to normalization of airway symptoms score | The primary outcome measure will be the time for the ASQ score to drop to normal on ranitidine or famotidine versus placebo. | 10 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Weight gain | A secondary outcome will be weight gain in percentile. | 10 month | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Laryngomalacia