Bronchopulmonary DysplasiaPhase 1NCT04662151
A Clinical Safety Study of AT-100 (rhSP-D) in Preterm Neonates at High Risk for Bronchopulmonary Dysplasia (BPD)
A Phase 1 study testing AT-100 in people with Bronchopulmonary Dysplasia, run by Airway Therapeutics, Inc. at 19 sites in 2 countries.
Trial summary
The purpose of this study is to determine if an investigational drug, AT-100, can reduce the occurrence of Bronchopulmonary Dysplasia (BPD) in babies born premature, as compared to babies born premature who receive an air-sham alone. Read more about this study
- Who is it for?
- Ages 0 minutes to 96 hours
- Conditions the study is for
- Bronchopulmonary Dysplasia
- What is being tested?
- AT-100
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 37 participants
- Where
- 19 sitesin 2 countries
- Study dates
- 1 Sep 2021to 29 May 2024
- Phase
- Phase 1
- 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 0 minutes to 96 hours
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Preterm neonates born between Gestional Age (GA):
- 25 0/7 weeks to 28 6/7 weeks in the initial dose escalation cohorts.
- 23 0/7 weeks to 28 6/7 weeks in the latter cohort.
- Intubated and on mechanical ventilation.
- Receiving at least 1 dose of standard-of-care-indicated surfactant treatment (Curosurf®) after birth, and able to receive the first dose of AT-100 or air-sham within 96 hours of birth given at any time point after 15 minutes following any of the subject's Curosurf® dose(s).
- Parent or legal guardian is able to provide informed consent.
Who cannot join
- Weight at time of birth < 400 g or > 1,800 g.
- Major apparent congenital abnormalities impacting cardio and pulmonary function.
- Active DNR (Do Not Resuscitate) order in place.
- Known pulmonary air leaks (e.g. pneumothorax and pneumomediastinum) at the time of AT-100 or air-sham administration.
- History of allergy or sensitivity to any surfactant or any component of the Investigational Product (AT-100).
- AT-100 or air-sham dosing was set to occur before Data Safety Monitoring Committee recommendation to proceed to the next dose-escalation cohort.
Show all 17Show fewer
- Use of minimally invasive surfactant techniques (e.g., LISA, MIST) or INSURE or if, in the opinion of the care team, the infant is very likely too be extubated shortly after receiving Curosurf®.
- Subjects extubated and re-intubated after their Curosurf® dose(s) are eligible, so long as the subject meets Inclusion #3.
- Birth mother:
- Has known active Hepatitis B, C, or E diagnosis.
- Has a known illness or exposure that, in the judgement of the Investigator, is serious enough to induce an immune deficiency such as Human Immunodeficiency Virus (HIV) and/or is receiving chemotherapy.
- Has known active Sexually Transmitted Infection (STI).
- Has known Cytomegalovirus (CMV) active infection.
- Has known history or evidence of alcohol or drug abuse, wit the exception of marijuana/marijuana-based products/THC, based on a positive maternal or infant drug screen as evidenced by the institution's standard-of-care practice.
- Concurrent enrollment in an investigational drug, device, or treatment modulation trial that utilizes treatments outside of standard-of-care.
- Any condition or situation which, in the Investigator's judgement, puts the mother or the neonate at significant risk, could confound the trial results, or may interfere significantly with the mother's or neonate's participation in the trial.
- Symptomatic and confirmed COVID-19 infection of the mother around the time of birth.
Trial locations and contacts
19 sites in 2 countries.
19 sites in 2 countries
Spain10 sites
Airway Therapeutics Investigational Site
A Coruña, 15006
Airway Therapeutics Investigational Site
Alicante, Valencia, 03010
Airway Therapeutics Investigational Site
Barcelona, Catalonia, 08041
Airway Therapeutics Investigational Site
Cadiz, Andalusia, 11009
Airway Therapeutics Investigational Site
Lleida, 25198
Airway Therapeutics Investigational Site
Madrid, Madrid, 28007
Airway Therapeutics Investigational Site
Madrid, Madrid, 28046
Airway Therapeutics Investigational Site
Málaga, 29010
Airway Therapeutics Investigational Site
Santiago de Compostela, Galicia, 15706
Airway Therapeutics Investigational Site
Valencia, Valencia, 46026
United States9 sites
Airway Therapeutics Investigational Site
Atlanta, Georgia, 30308
Airway Therapeutics Investigational Site
Boston, Massachusetts, 02215
Airway Therapeutics Investigational Site
Durham, North Carolina, 27705
Airway Therapeutics Investigational Site
Indianapolis, Indiana, 46202
Airway Therapeutics Investigational Site
Little Rock, Arkansas, 72202
Airway Therapeutics Investigational Site
Los Angeles, California, 90017
Airway Therapeutics Investigational Site
Miami, Florida, 33143
Airway Therapeutics Investigational Site
Norfolk, Virginia, 23507
Airway Therapeutics Investigational Site
Tucson, Arizona, 85724
About this study
Official title: A Phase 1b, Randomized, Blinded, Dose-Determined Study Evaluating the Safety and Tolerability Profile of Intervention With AT-100 (rhSP-D) in Preterm Neonates at High Risk for the Development of Bronchopulmonary Dysplasia (BPD)
The purpose of this study is to determine if an investigational drug, AT-100, can reduce the occurrence of Bronchopulmonary Dysplasia (BPD) in babies born premature, as compared to babies born premature who receive an air-sham alone.
- Conditions
- Bronchopulmonary Dysplasia
- Being tested
- AT-100, Air-sham
- Keywords
- Bronchopulmonary Dysplasia, Preterm, Neonate, Mechanical ventilation, Respiratory support, recombinant human Surfactant Protein-D (rhSP-D), intratracheal, air-sham
- Medical subject headings
- Bronchopulmonary Dysplasia, Premature Birth
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AT-100Biological
Also called (rhSP-D)
reconstituted AT-100 for intratracheal administration
Air-shamProcedure or surgery
room air for intratracheal administration
| Study group | Role | What they receive |
|---|---|---|
| Phase 1b open-label AT-100 | Experimental | Once daily AT-100 via intratracheal administration for up to 2 doses (initial Phase 1b dose-escalation portion) or 7 doses (latter Phase 1b highest tolerated \& safety dose level tested portion). Receives: Biological: AT-100 |
| Phase 1b open-label air-sham | Sham comparator | Once daily air-sham via intratracheal administration for up to 2 doses (initial Phase 1b dose-escalation portion) or 7 doses (latter Phase 1b highest tolerated \& safety dose level tested portion). Receives: Procedure: Air-sham |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with treatment-related adverse events
Incidence and severity of adverse events between the two treatment groups will be compared
Measured: Adverse events will be followed up to Day 28 of life
Incidence of BPD or death
Measured: Week 36 PMA
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Dec 2020
- Study start1 Sep 2021
- Primary completion14 Aug 2023
- Study completion29 May 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (6)Papers the sponsor has linked to this study.
- Other trials for Bronchopulmonary DysplasiaStudies 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 1
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Phase 1b portion is a randomized, dual-armed, dose escalation study to establish the safest \& most tolerated AT-100 dose tested as compared to air-sham alone.
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 37 participants (actual)
Sponsor and investigators
- Lead sponsor
- Airway Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Marc O. Salzberg, MD, Study chair, Airway Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04662151
- Sponsor's protocol ID
- AT-100/001
- Status last verified
- Jul 2024
- First submitted
- 4 Dec 2020
- Last updated
- 3 Jul 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Thebaud B, Goss KN, Laughon M, Whitsett JA, Abman SH, Steinhorn RH, Aschner JL, Davis PG, McGrath-Morrow SA, Soll RF, Jobe AH. Bronchopulmonary dysplasia. Nat Rev Dis Primers. 2019 Nov 14;5(1):78. doi: 10.1038/s41572-019-0127-7.
PubMed 31727986Jensen EA, Dysart K, Gantz MG, McDonald S, Bamat NA, Keszler M, Kirpalani H, Laughon MM, Poindexter BB, Duncan AF, Yoder BA, Eichenwald EC, DeMauro SB. The Diagnosis of Bronchopulmonary Dysplasia in Very Preterm Infants. An Evidence-based Approach. Am J Respir Crit Care Med. 2019 Sep 15;200(6):751-759. doi: 10.1164/rccm.201812-2348OC.
PubMed 30995069Sorensen GL. Surfactant Protein D in Respiratory and Non-Respiratory Diseases. Front Med (Lausanne). 2018 Feb 8;5:18. doi: 10.3389/fmed.2018.00018. eCollection 2018.
PubMed 29473039Sato A, Whitsett JA, Scheule RK, Ikegami M. Surfactant protein-d inhibits lung inflammation caused by ventilation in premature newborn lambs. Am J Respir Crit Care Med. 2010 May 15;181(10):1098-105. doi: 10.1164/rccm.200912-1818OC. Epub 2010 Feb 4.
PubMed 20133924Ikegami M, Carter K, Bishop K, Yadav A, Masterjohn E, Brondyk W, Scheule RK, Whitsett JA. Intratracheal recombinant surfactant protein d prevents endotoxin shock in the newborn preterm lamb. Am J Respir Crit Care Med. 2006 Jun 15;173(12):1342-7. doi: 10.1164/rccm.200509-1485OC. Epub 2006 Mar 23.
PubMed 16556693Alonso-Ojembarrena A, Poindexter B, Aleem S, Healy H, Aguar-Carrascosa M, Moliner-Calderon E, Serrano-Martin MDM, Arroyo R, Vento M. A phase 1b randomized, multicenter, dose determination trial of zelpultide alfa (recombinant human surfactant protein D) in preterm neonates at high risk of developing bronchopulmonary dysplasia. Front Pediatr. 2025 Sep 12;13:1639573. doi: 10.3389/fped.2025.1639573. eCollection 2025.
PubMed 41018057
Study at a glance
From the registry record, last updated by the sponsor on 3 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1b, Randomized, Blinded, Dose-Determined Study Evaluating the Safety and Tolerability Profile of Intervention With AT-100 (rhSP-D) in Preterm Neonates at High Risk for the Development of Bronchopulmonary Dysplasia (BPD)
- ClinicalTrials.gov ID
- NCT04662151
- Acronym / sponsor ID
- AT-100/001
- Conditions
- Bronchopulmonary Dysplasia
- Keywords
- Bronchopulmonary Dysplasia, Preterm, Neonate, Mechanical ventilation, Respiratory support, recombinant human Surfactant Protein-D (rhSP-D), intratracheal, air-sham
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 37 participants (actual)
- Lead sponsor
- Airway Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 1 Sep 2021 (actual)
- Primary completion
- 14 Aug 2023 (actual)
- Study completion
- 29 May 2024 (actual)
- First posted
- 10 Dec 2020 (actual)
- 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.
The purpose of this study is to determine if an investigational drug, AT-100, can reduce the occurrence of Bronchopulmonary Dysplasia (BPD) in babies born premature, as compared to babies born premature who receive an air-sham alone.
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 2
- Model description
- Phase 1b portion is a randomized, dual-armed, dose escalation study to establish the safest \& most tolerated AT-100 dose tested as compared to air-sham alone.
| Arm | Type | Receives |
|---|---|---|
| Phase 1b open-label AT-100 | Experimental | Biological: AT-100Once daily AT-100 via intratracheal administration for up to 2 doses (initial Phase 1b dose-escalation portion) or 7 doses (latter Phase 1b highest tolerated \& safety dose level tested portion). |
| Phase 1b open-label air-sham | Sham comparator | Procedure: Air-shamOnce daily air-sham via intratracheal administration for up to 2 doses (initial Phase 1b dose-escalation portion) or 7 doses (latter Phase 1b highest tolerated \& safety dose level tested portion). |
Who may qualify
- Ages
- 0 minutes to 96 hours
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Preterm neonates born between Gestional Age (GA):
- Intubated and on mechanical ventilation.
- Receiving at least 1 dose of standard-of-care-indicated surfactant treatment (Curosurf®) after birth, and able to receive the first dose of AT-100 or air-sham within 96 hours of birth given at any time point after 15 minutes following any of the subject's Curosurf® dose(s).
- Parent or legal guardian is able to provide informed consent.
Key exclusion criteria (17 listed)
- Weight at time of birth < 400 g or > 1,800 g.
- Major apparent congenital abnormalities impacting cardio and pulmonary function.
- Active DNR (Do Not Resuscitate) order in place.
- Known pulmonary air leaks (e.g. pneumothorax and pneumomediastinum) at the time of AT-100 or air-sham administration.
- History of allergy or sensitivity to any surfactant or any component of the Investigational Product (AT-100).
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 |
|---|---|---|
| AT-100Also: (rhSP-D)reconstituted AT-100 for intratracheal administration | Biological | Phase 1b open-label AT-100 |
| Air-shamroom air for intratracheal administration | Procedure or surgery | Phase 1b open-label air-sham |
Primary outcome
Number of participants with treatment-related adverse events
Time frame: Adverse events will be followed up to Day 28 of life
Incidence and severity of adverse events between the two treatment groups will be compared
Incidence of BPD or death
Time frame: Week 36 PMA
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jul 2024.
- Background: PubMed 31727986Thebaud B, Goss KN, Laughon M, Whitsett JA, Abman SH, Steinhorn RH, Aschner JL, Davis PG, McGrath-Morrow SA, Soll RF, Jobe AH. Bronchopulmonary dysplasia. Nat Rev Dis Primers. 2019 Nov 14;5(1):78. doi: 10.1038/s41572-019-0127-7.
- Background: PubMed 30995069Jensen EA, Dysart K, Gantz MG, McDonald S, Bamat NA, Keszler M, Kirpalani H, Laughon MM, Poindexter BB, Duncan AF, Yoder BA, Eichenwald EC, DeMauro SB. The Diagnosis of Bronchopulmonary Dysplasia in Very Preterm Infants. An Evidence-based Approach. Am J Respir Crit Care Med. 2019 Sep 15;200(6):751-759. doi: 10.1164/rccm.201812-2348OC.
- Background: PubMed 29473039Sorensen GL. Surfactant Protein D in Respiratory and Non-Respiratory Diseases. Front Med (Lausanne). 2018 Feb 8;5:18. doi: 10.3389/fmed.2018.00018. eCollection 2018.
- Background: PubMed 20133924Sato A, Whitsett JA, Scheule RK, Ikegami M. Surfactant protein-d inhibits lung inflammation caused by ventilation in premature newborn lambs. Am J Respir Crit Care Med. 2010 May 15;181(10):1098-105. doi: 10.1164/rccm.200912-1818OC. Epub 2010 Feb 4.
- Background: PubMed 16556693Ikegami M, Carter K, Bishop K, Yadav A, Masterjohn E, Brondyk W, Scheule RK, Whitsett JA. Intratracheal recombinant surfactant protein d prevents endotoxin shock in the newborn preterm lamb. Am J Respir Crit Care Med. 2006 Jun 15;173(12):1342-7. doi: 10.1164/rccm.200509-1485OC. Epub 2006 Mar 23.
- Derived from this study: PubMed 41018057Alonso-Ojembarrena A, Poindexter B, Aleem S, Healy H, Aguar-Carrascosa M, Moliner-Calderon E, Serrano-Martin MDM, Arroyo R, Vento M. A phase 1b randomized, multicenter, dose determination trial of zelpultide alfa (recombinant human surfactant protein D) in preterm neonates at high risk of developing bronchopulmonary dysplasia. Front Pediatr. 2025 Sep 12;13:1639573. doi: 10.3389/fped.2025.1639573. eCollection 2025.
- Airway Therapeutics' corporate websiteLink provided by the sponsor.
Related trials for Bronchopulmonary Dysplasia
Recruiting
Optimization of Saturation Targets And Resuscitation Trial (OptiSTART)
Premature Infants, Bronchopulmonary Dysplasia
Recruiting
Controlled Remote Monitoring and Optimization of Oxygen Therapy in Preterm Infants
BPD - Bronchopulmonary Dysplasia, Pulmonary Hypertension
Not yet recruitingPhase 1/2
Little Lungs Study
Invasive Mechanical Ventilation, Severe Respiratory Distress Syndrome
Not yet recruiting
Strap To Reduce Apnea in Preterm Infants: A Randomized Crossover Trial
Apnea of Prematurity, Intermittent Hypoxemia
Recruiting
Ventilator Pressure and Optimization of Compliance and Hemodynamics
Bronchopulmonary Dysplasia, Pulmonary Hypertension Due to Lung Diseases and Hypoxia
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.
Marc O. Salzberg, MD study chair
Airway Therapeutics, Inc.
19 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing AT-100 in people with Bronchopulmonary Dysplasia, run by Airway Therapeutics, Inc. at 19 sites in 2 countries.
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 6 inclusion and 17 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 3 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 19 sites in Spain, 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Number of participants with treatment-related adverse events (time frame: Adverse events will be followed up to Day 28 of life). The study lists 2 primary outcomes in all. 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 NCT04662151, which also lists 6 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 3 Jul 2024.