Respiratory Distress Syndrome in Premature InfantPhase 2NCT01941745
Efficacy of Recombinant Human Clara Cell 10 Protein (rhCC10) Administered to Premature Neonates With Respiratory Distress Syndrome
A Phase 2 study testing Half normal saline, Low Dose rhCC10 and High dose rhCC10 in people with Respiratory Distress Syndrome in Premature Infant and Bronchopulmonary Dysplasia, run by Tufts Medical Center at 3 sites in United States.
Trial summary
Bronchopulmonary Dysplasia (BPD) is a multi-factorial disease process that is the end result of an immature, surfactant deficient lung that has been exposed to hyperoxia, mechanical ventilation and infection. Read more about this study
- Who is it for?
- Ages 24 to 29 weeks
- Conditions the study is for
- Respiratory Distress Syndrome in Premature Infant, Bronchopulmonary Dysplasia
- What is being tested?
- Half normal saline, Low Dose rhCC10 and High dose rhCC10
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 88 participants
- Where
- 3 sitesUnited States
- Study dates
- Oct 2013to 25 Aug 2017
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 24 to 29 weeks
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age less than or equal to 24 hours;
- Birth weight 600 - 1250 grams;
- Gestational age 24-29 weeks (not less than 24 weeks); at birth based on best estimate using obstetrical sonography (first or second trimester), solid dating criteria, or Ballard examination;
- Birth weight appropriate for gestational age;
- 5 minute Apgar score >5;
- Diagnosis of neonatal RDS based on clinical and radiographic criteria;
Show all 9Show fewer
- Requiring intubation and mechanical ventilation for treatment of RDS;
- Received at least one dose of surfactant (prophylaxis or rescue); and
- Written informed consent is obtained from at least one of the infant's parents or legal guardians (see section 6.2) prior to enrollment of the subject. The parent(s) or legal guardian(s) must agree to all study-related procedures and evaluations.
Who cannot join
- 5 minute Apgar score of ≤ 5;
- Major congenital anomaly (chromosomal, renal, cardiac, hepatic, neurologic, or pulmonary malformations; minor anomalies such as cleft lip/palate are permitted);
- Evidence of severe neonatal depression (as defined by cord blood acid-base balance (pH) ≤ 7.00 and/or an Apgar score of < 4 at 10 minutes);
- Evidence of congenital infection;
- Requires a major surgical procedure prior to administration of Study drug
- Enrollment in any other study involving administration of another investigational drug;
Show all 12Show fewer
- Any condition which could preclude receiving study drug or performing any study-related procedures;
- Use of postnatal corticosteroids prior to administration of r-hCC10, except as specified in the protocol;
- Use of inhaled nitric oxide prior to administration of r-hCC10;
- Mother is known to be seropositive for HIV (per maternal medical records);
- Parent or guardian is unable or unwilling to complete the study diary;
- Parent or guardian is unable to bring the infant back to the study center for follow-up evaluations.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Tufts Medical Center
Boston, Massachusetts, 02111
Brigham and Women's Hospital
Boston, Massachusetts, 02115
Baystate Medical Center
Springfield, Massachusetts, 01199
About this study
Official title: Efficacy of Recombinant Human Clara Cell Protein (rhCC10) Administered to Premature Neonates With Respiratory Distress Syndrome
Bronchopulmonary Dysplasia (BPD) is a multi-factorial disease process that is the end result of an immature, surfactant deficient lung that has been exposed to hyperoxia, mechanical ventilation and infection. These conditions initiate an inflammatory response characterized by elevated inflammatory cell infiltrates and proinflammatory cytokines that lead to the development of significant acute and chronic lung injury.
The study drug, rhCC10, is a recombinant version of natural human CC10 protein. Native CC10 is produced primarily by non-ciliated respiratory epithelial cells, called Clara cells and is the most abundant protein in the mucosal fluids in normal healthy lungs.
Read more
The purpose of this study is to evaluate the pharmacokinetics, safety, tolerability and anti-inflammatory effects of a single intratracheal (IT) dose of rhCC10 to intubated premature infants receiving positive pressure ventilation for treatment of respiratory distress syndrome (RDS) to prevent long term respiratory complications referred to as bronchopulmonary dysplasia, and, more recently, as Chronic Pulmonary Insufficiency of Prematurity (CPIP; asthma, cough, wheezing, multiple respiratory infections).
CC10 regulates inflammatory responses and protects the structural integrity of pulmonary tissue while preserving pulmonary mechanical function during various insults (eg. viral infection, bacterial endotoxin, ozone, allergens, hyperoxia). Together these properties suggest that administration of rhCC10 may help to facilitate development of normal airway epithelia and prevent the inflammation that leads to CPIP in these infants.
This study is funded by the FDA Office of Orphan Product Development (OOPD).
Read the detailed description
Recombinant human CC10 protein (rhCC10) is a novel therapeutic agent used to prevent the development of chronic respiratory morbidity (CPIP; repeated respiratory infections, asthma, re-hospitalizations) in preterm infants. Native CC10 is a natural anti-inflammatory and immunomodulatory factor produced by Clara Cells in the lung and is the most abundant protein in respiratory mucosa. Animal data demonstrate that a single intratracheal dose of rhCC10 administered shortly after birth reduces lung inflammation (important biomarkers linked to lung injury in preterm infants), promotes normal lung development, preserves lung architecture, improves pulmonary function, suppresses the response to endotoxin and enhances resistance to pulmonary infections. In preterm infants who die or develop lung inflammation and subsequent bronchopulmonary dysplasia (BPD), both the concentration and activity of CC10 are significantly reduced indicating that CC10 is essential for preventing lung injury and promoting normal lung development. In a small phase I study, rhCC10 significantly decreased several indices of pulmonary inflammation in the lungs of premature infants who were at risk of developing BPD and associated CPIP. The drug appeared to be safe, well-tolerated, and reduce risk of re-hospitalization due to respiratory illness for 9-10 months after a single intratracheal dose at the time of birth (0/11 rhCC10-treated infants vs. 3/6 placebo-treated). This supports the protective role of rhCC10 against damage from hyperoxia, mechanical ventilation, inflammation, and infection in the immature lung. A more normal airway epithelium will produce significantly more endogenous CC10, with both factors contributing to enhanced resistance to infections, less asthma, and improved long-term respiratory outcome. We propose to conduct a Phase 2 clinical trial to evaluate rhCC10 in extremely premature infants (<29 weeks gestation) for the prevention of BPD and CPIP. This will be a randomized, double-blind, placebo-controlled dose escalation study in 88 premature infants. A single intratracheal dose of study drug (rhCC10 or placebo) will be administered to preterm infants receiving surfactant and mechanical ventilation for treatment of RDS. Infants will be followed to evaluate safety, pharmacokinetics, and short and long term efficacy of this approach. Safety will be evaluated through serious adverse event (SAE) and adverse event monitoring and by Bayley neurodevelopmental assessments at 18 months corrected gestational age (CGA). Efficacy measurements will include the primary combined endpoint of alive without evidence of CPIP through 12 months CGA (defined by parental diaries and pulmonary questionnaires) comparing rhCC10 treated to placebo controls. The availability of a therapy which prevents lung injury, promotes lung development, and prevents serious respiratory infections and asthma in high risk preterm infants would be a highly significant advancement in care.
- Being tested
- Half normal saline, Low Dose rhCC10, High dose rhCC10
- Medical subject headings
- Bronchopulmonary Dysplasia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Half normal salineDrug
2 ml/kg
Low Dose rhCC10Drug
1.5 mg/kg study drug (rhCC10)
High dose rhCC10Drug
5 mg/kg in 2 ml/kg
| Study group | Role | What they receive |
|---|---|---|
| half normal saline | Placebo comparator | Single dose of half normal saline at 2 ml/kg given intratracheally times one dose Receives: Drug: Half normal saline |
| Low Dose rhCC10 | Experimental | 1.5 mg/kg study drug (rhCC10)in 2 ml/kg given intratracheally times one dose Receives: Drug: Low Dose rhCC10 |
| High dose rhCC10 | Experimental | 5 mg/kg of rhCC10 given in 2 ml/kg and administered intratracheally times one dose Receives: Drug: High dose rhCC10 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Survival Without Evidence of Chronic Pulmonary Insufficiency of Prematurity (CPIP) at 12 Months Corrected Gestational Age (CGA)
Number of events of survived participants without one or more of the CPIP components defined below:
- Medical/ER visits (CPIP-DV): At least one non-routine medical visit for respiratory causes.
- Respiratory re-hospitalizations (CPIP-RH): One or more re-hospitalizations for respiratory causes.
- Respiratory Symptoms (CPIP-SS): Evidence of respiratory symptoms (e.g. coughing and wheezing) or use of respiratory medications by parental diaries or pulmonary questionnaires.
- Respiratory Medications (CPIP-RM): Administration of respiratory medications (including oxygen).
CPIP is defined as the presence of one or more parent-reported outcomes at 12 months CGA, validated by Respiratory diaries (presence of wheezing, coughing, and/or respiratory medication use ≥2 days per week for 3 consecutive weeks), and Pulmonary questionnaires (decrease in respiratory illness requiring medications, unscheduled medical visits and/or ER or hospital admissions).
Measured: 12 Months Corrected Gestational Age (*no imputation for missing data)
Secondary outcomes (3)
Long Term Efficacy - Survival Without Evidence of Chronic Pulmonary Insufficiency of Prematurity (CPIP) at 6 Months Corrected Gestational Age (CGA)
Number of events of survived participants, graded as not having 1, 2, 3, or 4 of the CPIP components defined below:
Medical/ER visits (CPIP-DV): ≥1 non-routine medical visit(s) for respiratory causes.
Respiratory re-hospitalizations (CPIP-RH): ≥1 re-hospitalization(s) for respiratory causes Respiratory Symptoms (CPIP-SS): Parent-reported evidence of respiratory symptoms (e.g. coughing and wheezing) or use of respiratory medications Respiratory Medications (CPIP-RM): Administration of respiratory medications (including oxygen)
A participant graded without 4 CPIP components is considered in better health than a participant graded without 1 CPIP component. CPIP components were parent-validated via respiratory diaries (wheezing, coughing, and/or respiratory medication use ≥2 days/wk for 3 consecutive wks), and pulmonary questionnaires (decrease in respiratory illness requiring medications, unscheduled medical visits and/or ER or hospital admissions).
Measured: 6 months Corrected Gestational Age
Safety and Efficacy - Number of Participants With Adverse Events
The safety of the study drug was assessed by accounting the number of participants with Adverse Events (AEs) in the treatment and placebo groups.
Measured: Adverse events are monitored through 36 wks post-menstrual age (PMA)
Short Term Efficacy - Number of Neonates With Oxygen Requirement at 36 Weeks Post Menstrual Age
Short term efficacy evaluations involve number of neonates with oxygen requirement at 36 weeks post menstrual age.
Measured: 36 weeks post-menstrual age
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Sep 2013 (estimated)
- Study startOct 2013
- Primary completion25 Aug 2017
- Study completion25 Aug 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Respiratory Distress Syndrome in Premature InfantStudies of the same condition, open and under way.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 88 participants (actual)
Sponsor and investigators
- Lead sponsor
- Tufts Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- Brigham and Women's Hospital, Therabron Therapeutics, Inc., Baystate Medical Center, Poznan University of Medical Sciences, SP ZOZ Szpital Uniwersytecki w Krakowie Oddizat Neonatologii, Instytut Centrum Zdrowia Matki Polki Klinika Neonatologii
- Responsible party
- Sponsor
- Investigators
- Jonathan Davis, MD, Principal investigator, Tufts Medical CenterRichard Parad, MD/MPH, Principal investigator, Brigham and Women's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01941745
- Sponsor's protocol ID
- rhCC10 Study 2013
- Other IDs
- Grant #3899
- Status last verified
- Aug 2019
- First submitted
- 29 Aug 2013
- Last updated
- 10 Sep 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 10 Sep 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy of Recombinant Human Clara Cell Protein (rhCC10) Administered to Premature Neonates With Respiratory Distress Syndrome
- ClinicalTrials.gov ID
- NCT01941745
- Acronym / sponsor ID
- rhCC10 Study 2013
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 88 participants (actual)
- Lead sponsor
- Tufts Medical CenterUniversity, hospital or other
- Collaborators
- Brigham and Women's Hospital, Therabron Therapeutics, Inc., Baystate Medical Center, Poznan University of Medical Sciences, SP ZOZ Szpital Uniwersytecki w Krakowie Oddizat Neonatologii, Instytut Centrum Zdrowia Matki Polki Klinika Neonatologii
- Study start
- Oct 2013 (actual)
- Primary completion
- 25 Aug 2017 (actual)
- Study completion
- 25 Aug 2017 (actual)
- First posted
- 13 Sep 2013 (estimated)
- Results
- Posted 10 Sep 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Bronchopulmonary Dysplasia (BPD) is a multi-factorial disease process that is the end result of an immature, surfactant deficient lung that has been exposed to hyperoxia, mechanical ventilation and infection. These conditions initiate an inflammatory response characterized by elevated inflammatory cell infiltrates and proinflammatory cytokines that lead to the development of significant acute and chronic lung injury.
The study drug, rhCC10, is a recombinant version of natural human CC10 protein. Native CC10 is produced primarily by non-ciliated respiratory epithelial cells, called Clara cells and is the most abundant protein in the mucosal fluids in normal healthy lungs.
The purpose of this study is to evaluate the pharmacokinetics, safety, tolerability and anti-inflammatory effects of a single intratracheal (IT) dose of rhCC10 to intubated premature infants receiving positive pressure ventilation for treatment of respiratory distress syndrome (RDS) to prevent long term respiratory complications referred to as bronchopulmonary dysplasia, and, more recently, as Chronic Pulmonary Insufficiency of Prematurity (CPIP; asthma, cough, wheezing, multiple respiratory infections).
CC10 regulates inflammatory responses and protects the structural integrity of pulmonary tissue while preserving pulmonary mechanical function during various insults (eg. viral infection, bacterial endotoxin, ozone, allergens, hyperoxia). Together these properties suggest that administration of rhCC10 may help to facilitate development of normal airway epithelia and prevent the inflammation that leads to CPIP in these infants.
This study is funded by the FDA Office of Orphan Product Development (OOPD).
Detailed description
Recombinant human CC10 protein (rhCC10) is a novel therapeutic agent used to prevent the development of chronic respiratory morbidity (CPIP; repeated respiratory infections, asthma, re-hospitalizations) in preterm infants. Native CC10 is a natural anti-inflammatory and immunomodulatory factor produced by Clara Cells in the lung and is the most abundant protein in respiratory mucosa. Animal data demonstrate that a single intratracheal dose of rhCC10 administered shortly after birth reduces lung inflammation (important biomarkers linked to lung injury in preterm infants), promotes normal lung development, preserves lung architecture, improves pulmonary function, suppresses the response to endotoxin and enhances resistance to pulmonary infections. In preterm infants who die or develop lung inflammation and subsequent bronchopulmonary dysplasia (BPD), both the concentration and activity of CC10 are significantly reduced indicating that CC10 is essential for preventing lung injury and promoting normal lung development. In a small phase I study, rhCC10 significantly decreased several indices of pulmonary inflammation in the lungs of premature infants who were at risk of developing BPD and associated CPIP. The drug appeared to be safe, well-tolerated, and reduce risk of re-hospitalization due to respiratory illness for 9-10 months after a single intratracheal dose at the time of birth (0/11 rhCC10-treated infants vs. 3/6 placebo-treated). This supports the protective role of rhCC10 against damage from hyperoxia, mechanical ventilation, inflammation, and infection in the immature lung. A more normal airway epithelium will produce significantly more endogenous CC10, with both factors contributing to enhanced resistance to infections, less asthma, and improved long-term respiratory outcome. We propose to conduct a Phase 2 clinical trial to evaluate rhCC10 in extremely premature infants (<29 weeks gestation) for the prevention of BPD and CPIP. This will be a randomized, double-blind, placebo-controlled dose escalation study in 88 premature infants. A single intratracheal dose of study drug (rhCC10 or placebo) will be administered to preterm infants receiving surfactant and mechanical ventilation for treatment of RDS. Infants will be followed to evaluate safety, pharmacokinetics, and short and long term efficacy of this approach. Safety will be evaluated through serious adverse event (SAE) and adverse event monitoring and by Bayley neurodevelopmental assessments at 18 months corrected gestational age (CGA). Efficacy measurements will include the primary combined endpoint of alive without evidence of CPIP through 12 months CGA (defined by parental diaries and pulmonary questionnaires) comparing rhCC10 treated to placebo controls. The availability of a therapy which prevents lung injury, promotes lung development, and prevents serious respiratory infections and asthma in high risk preterm infants would be a highly significant advancement in care.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| half normal saline | Placebo comparator | Drug: Half normal salineSingle dose of half normal saline at 2 ml/kg given intratracheally times one dose |
| Low Dose rhCC10 | Experimental | Drug: Low Dose rhCC101.5 mg/kg study drug (rhCC10)in 2 ml/kg given intratracheally times one dose |
| High dose rhCC10 | Experimental | Drug: High dose rhCC105 mg/kg of rhCC10 given in 2 ml/kg and administered intratracheally times one dose |
Who may qualify
- Ages
- 24 to 29 weeks
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- Age less than or equal to 24 hours;
- Birth weight 600 - 1250 grams;
- Gestational age 24-29 weeks (not less than 24 weeks); at birth based on best estimate using obstetrical sonography (first or second trimester), solid dating criteria, or Ballard examination;
- Birth weight appropriate for gestational age;
- 5 minute Apgar score >5;
Key exclusion criteria (12 listed)
- 5 minute Apgar score of ≤ 5;
- Major congenital anomaly (chromosomal, renal, cardiac, hepatic, neurologic, or pulmonary malformations; minor anomalies such as cleft lip/palate are permitted);
- Evidence of severe neonatal depression (as defined by cord blood acid-base balance (pH) ≤ 7.00 and/or an Apgar score of < 4 at 10 minutes);
- Evidence of congenital infection;
- Requires a major surgical procedure prior to administration of Study drug
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 |
|---|---|---|
| Half normal saline2 ml/kg | Drug | half normal saline |
| Low Dose rhCC101.5 mg/kg study drug (rhCC10) | Drug | Low Dose rhCC10 |
| High dose rhCC105 mg/kg in 2 ml/kg | Drug | High dose rhCC10 |
Primary outcome
Survival Without Evidence of Chronic Pulmonary Insufficiency of Prematurity (CPIP) at 12 Months Corrected Gestational Age (CGA)
Time frame: 12 Months Corrected Gestational Age (*no imputation for missing data)
Number of events of survived participants without one or more of the CPIP components defined below: 1. Medical/ER visits (CPIP-DV): At least one non-routine medical visit for respiratory causes. 2. Respiratory re-hospitalizations (CPIP-RH): One or more re-hospitalizations for respiratory causes. 3. Respiratory Symptoms (CPIP-SS): Evidence of respiratory symptoms (e.g. coughing and wheezing) or use of respiratory medications by parental diaries or pulmonary questionnaires. 4. Respiratory Medications (CPIP-RM): Administration of respiratory medications (including oxygen). CPIP is defined as the presence of one or more parent-reported outcomes at 12 months CGA, validated by Respiratory diaries (presence of wheezing, coughing, and/or respiratory medication use ≥2 days per week for 3 consecutive weeks), and Pulmonary questionnaires (decrease in respiratory illness requiring medications, unscheduled medical visits and/or ER or hospital admissions).
3 secondary outcomes
Long Term Efficacy - Survival Without Evidence of Chronic Pulmonary Insufficiency of Prematurity (CPIP) at 6 Months Corrected Gestational Age (CGA)
Time frame: 6 months Corrected Gestational Age
Safety and Efficacy - Number of Participants With Adverse Events
Time frame: Adverse events are monitored through 36 wks post-menstrual age (PMA)
Short Term Efficacy - Number of Neonates With Oxygen Requirement at 36 Weeks Post Menstrual Age
Time frame: 36 weeks post-menstrual age
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Sep 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Respiratory Distress Syndrome in Premature Infant
Recruiting
REspiratory MEchanics for Delivering Individualised Exogenous Surfactant
Respiratory Distress Syndrome in Premature Infant
Not yet recruiting
The Hemodynamic Impact of High-frequency Oscillatory Ventilation and Periodic Sighs in Preterm Infants
Preterm, Respiratory Distress Syndrome in Premature Infant
Recruiting
Ventilator Pressure and Optimization of Compliance and Hemodynamics
Bronchopulmonary Dysplasia, Pulmonary Hypertension Due to Lung Diseases and Hypoxia
Active, not recruiting
Measuring Diaphragm Electrical Activity in Neonates Using a Smaller Inter-electrode Distance
Respiratory Distress Syndrome, Newborn, Infant, Premature
Not yet recruitingObservational study
Chest Ultrasound as Diagnostic and Prognostic Tool in Neonatal RDS
Respiratory Distress Syndrome, Newborn, Neonatal Respiratory Distress
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.
Jonathan Davis, MD principal investigator
Tufts Medical Center
Richard Parad, MD/MPH principal investigator
Brigham and Women's Hospital
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Half normal saline, Low Dose rhCC10 and High dose rhCC10 in people with Respiratory Distress Syndrome in Premature Infant and Bronchopulmonary Dysplasia, run by Tufts Medical Center at 3 sites 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 9 inclusion and 12 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: finished, as of the sponsor's last update on 10 Sep 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 10 Sep 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Survival Without Evidence of Chronic Pulmonary Insufficiency of Prematurity (CPIP) at 12 Months Corrected Gestational Age (CGA) (time frame: 12 Months Corrected Gestational Age (*no imputation for missing data)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 10 Sep 2019.