LaryngomalaciaNCT02700087
In Infants With Laryngomalacia, Does Acid-Blocking Medication Improve Respiratory Symptoms?
A study testing ranitidine or famotidine in people with Laryngomalacia and Acid Reflux, run by Stanford University at 1 site in United States.
Trial summary
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… Read more about this study
- Who is it for?
- Ages Up to 4 months
- Conditions the study is for
- Laryngomalacia, Acid Reflux, Stridor
- What is being tested?
- ranitidine or famotidine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2016to Feb 2017
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 4 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- age 0 - 4 months old
- stridor and flexible laryngoscopy demonstrating laryngomalacia
- airway symptom score over 4
- only has physiologic GER (I-GERQ < 16)
Who cannot join
- 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)
Trial locations and contacts
1 site in 1 country.
United States1 site
Lucile Packard Children's Hospital Stanford
Palo Alto, California, 94304
About this study
Official title: In Infants With Symptoms of Tracheomalacia or Laryngomalacia, Does Acid-Blocking Medication Improve Respiratory Symptoms? A Randomized, Controlled Trial
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.
Read the detailed description
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.
- Conditions
- Laryngomalacia, Acid Reflux, Stridor
- Being tested
- ranitidine or famotidine, Placebo
- Keywords
- Laryngomalacia
- Medical subject headings
- Laryngomalacia, Gastroesophageal Reflux, Respiratory Sounds
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ranitidine or famotidineDrug
Patients with symptomatic laryngomalacia will receive ranitidine, famotidine, or placebo medications in order to see if these medications help their symptoms to resolve.
PlaceboDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| H2 blocker versus placebo | Placebo comparator | 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. Receives: Drug: ranitidine or famotidine; Drug: Placebo |
| Treatment arm | Active comparator | 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. Receives: Drug: ranitidine or famotidine |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 10 months
Secondary outcomes (1)
Weight gain
A secondary outcome will be weight gain in percentile.
Measured: 10 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Mar 2016 (estimated)
- Study startFeb 2016
- Primary completionFeb 2017 (estimated)
- Study completionFeb 2017 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for LaryngomalaciaStudies of the same condition, open and under way.
- More trials run by Stanford UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stanford University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Douglas Sidell, Stanford University
- Investigators
- Douglas R Sidell, MD, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02700087
- Sponsor's protocol ID
- IRB-35551
- Status last verified
- Nov 2017
- First submitted
- 23 Feb 2016
- Last updated
- 17 Nov 2017
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Publications
Thompson DM. Abnormal sensorimotor integrative function of the larynx in congenital laryngomalacia: a new theory of etiology. Laryngoscope. 2007 Jun;117(6 Pt 2 Suppl 114):1-33. doi: 10.1097/MLG.0b013e31804a5750.
PubMed 17513991Olney DR, Greinwald JH Jr, Smith RJ, Bauman NM. Laryngomalacia and its treatment. Laryngoscope. 1999 Nov;109(11):1770-5. doi: 10.1097/00005537-199911000-00009.
PubMed 10569405
Study at a glance
From the registry record, last updated by the sponsor on 17 Nov 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- In Infants With Symptoms of Tracheomalacia or Laryngomalacia, Does Acid-Blocking Medication Improve Respiratory Symptoms? A Randomized, Controlled Trial
- ClinicalTrials.gov ID
- NCT02700087
- Acronym / sponsor ID
- IRB-35551
- Conditions
- Laryngomalacia, Acid Reflux, Stridor
- Keywords
- Laryngomalacia
- Study type
- Interventional
- Recruitment status
- Withdrawn
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Stanford UniversityUniversity, hospital or other
- Study start
- Feb 2016
- Primary completion
- Feb 2017 (estimated)
- Study completion
- Feb 2017 (estimated)
- First posted
- 7 Mar 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| H2 blocker versus placebo | Placebo comparator | Drug: ranitidine or famotidine, Drug: PlaceboThe 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. |
| Treatment arm | Active comparator | Drug: ranitidine or famotidinePatients 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. |
Who may qualify
- Ages
- Up to 4 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- age 0 - 4 months old
- stridor and flexible laryngoscopy demonstrating laryngomalacia
- airway symptom score over 4
- only has physiologic GER (I-GERQ < 16)
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ranitidine or famotidinePatients with symptomatic laryngomalacia will receive ranitidine, famotidine, or placebo medications in order to see if these medications help their symptoms to resolve. | Drug | H2 blocker versus placebo, Treatment arm |
| PlaceboPatients 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. | Drug | H2 blocker versus placebo |
Primary outcome
Time to normalization of airway symptoms score
Time frame: 10 months
The primary outcome measure will be the time for the ASQ score to drop to normal on ranitidine or famotidine versus placebo.
1 secondary outcome
Weight gain
Time frame: 10 month
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Nov 2017.
- Background: PubMed 17513991Thompson DM. Abnormal sensorimotor integrative function of the larynx in congenital laryngomalacia: a new theory of etiology. Laryngoscope. 2007 Jun;117(6 Pt 2 Suppl 114):1-33. doi: 10.1097/MLG.0b013e31804a5750.
- Background: PubMed 10569405Olney DR, Greinwald JH Jr, Smith RJ, Bauman NM. Laryngomalacia and its treatment. Laryngoscope. 1999 Nov;109(11):1770-5. doi: 10.1097/00005537-199911000-00009.
- Individual participant dataSharing planned: No
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Laryngomalacia, Examinations and Quality of Life in Children Before and After Treatment With Follow-up After 1 Year
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Douglas R Sidell, MD principal investigator
Stanford University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing ranitidine or famotidine in people with Laryngomalacia and Acid Reflux, run by Stanford University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 17 Nov 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Time to normalization of airway symptoms score (time frame: 10 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02700087, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Nov 2017.