Bronchopulmonary Dysplasia (BPD)Phase 1/2SASSIENCT02907593
Steroids and Surfactant in Extremely Low Gestation Age Infants Dose Escalation Trial
A Phase 1/2 study testing Budesonide in Calfactant in people with Bronchopulmonary Dysplasia (BPD), run by Cynthia McEvoy at 5 sites in United States.
Trial summary
This is a phase I/II open-label study to determine the lowest, safe, effective dose of budesonide given with calfactant as the vehicle. Read more about this study
- Who is it for?
- Ages 3 to 14 days
- Conditions the study is for
- Bronchopulmonary Dysplasia (BPD)
- What is being tested?
- Budesonide in Calfactant
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 5 sitesUnited States
- Study dates
- Sep 2016to Apr 2018
- Phase
- Phase 1/2
- 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 3 to 14 days
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- > 23 0/7 and < 27 6/7 weeks of gestational age based on center's best estimate of due date (using earliest obstetrical ultrasound, last menstrual period, examination, and other pertinent available information)
- Day of life 3-14 from the date and time of delivery, with the date of birth being DOL 0
- Intubated and mechanically ventilated and do not anticipate extubation in next 24 hours
Who cannot join
- Serious congenital malformations or chromosomal abnormality
- Likely to be extubated in next 24 hours
- Clinically unstable
- Infants who have received systemic steroids prior to dosing with study medication.
- Infants who have received Indocin, Ibuprofen, or acetaminophen ≤ 96 hours prior to enrollment window ending
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
University of Florida, Jacksonville
Jacksonville, Florida, 32209
Vanderbilt Children's Hospital
Nashville, Tennessee, 37232
Florida Hospital for Children
Orlando, Florida, 32803
Oregon Health ans Science University
Portland, Oregon, 97239
University of California, San Francisco
San Francisco, California, 94143
About this study
Official title: Steroids and Surfactant in Extremely Low Gestation Age Infants Pilot Dose Escalation Trial
This is a phase I/II open-label study to determine the lowest, safe, effective dose of budesonide given with calfactant as the vehicle.
Read the detailed description
This is a phase I/II open-label study to determine the lowest safe, effective dose of budesonide given with calfactant as the vehicle; the investigators will perform an unblinded dose escalation study. The investigators will administer four dosing levels of budesonide suspended in calfactant beginning with 0.025 mg/kg of budesonide administered to 8 extremely low gestational age newborns (ELGANs) who are intubated at 3-10 days of age. Daily doses (at the same dosage) will be administered to infants who remain intubated for a potential of 5 total doses in each patient. Subsequent groups of 8 infants each will receive 0.05 mg/kg, 0.10 mg/kg, and 0.15 mg/kg of budesonide in calfactant (up to 5 total doses in each patient). A total of up to 32 infants will be enrolled in the trial. The investigators will evaluate the clinical, laboratory and safety data from each group of treated infants to 28 days of age before moving to the next dosing level of budesonide.
- Conditions
- Bronchopulmonary Dysplasia (BPD)
- Being tested
- Budesonide in Calfactant
- Keywords
- Bronchopulmonary dysplasia, Pulmonary, Surfactant, Steroids, Extremely low gestational age
- Medical subject headings
- Bronchopulmonary Dysplasia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Budesonide in CalfactantDrug
Also called pulmicort respule, calfactant, budesonide, 0186-1986-04
Budesonide in Calfactant
| Study group | Role | What they receive |
|---|---|---|
| 0.025 mg/kg Budesonide | Experimental | 0.025 mg/kg Budesonide in Calfactant Receives: Drug: Budesonide in Calfactant |
| 0.050 mg/kg Budesonide | Experimental | 0.050 mg/kg Budesonide in Calfactant Receives: Drug: Budesonide in Calfactant |
| 0.10 mg/kg Budesonide | Experimental | 0.10 mg/kg Budesonide in Calfactant Receives: Drug: Budesonide in Calfactant |
| 0.15 mg/kg Budesonide | Experimental | 0.15 mg/kg Budesonide in Calfactant Receives: Drug: Budesonide in Calfactant |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of ELGANs With Clinical and Anti-inflammatory Efficacy of Escalating Doses of Budesonide Determined by Monitoring Respiratory Severity Score (RSS)
Dose escalation will be defined as effective at the dose level in which 5 of 8 infants achieve the following:
- Extubation within 72 hours of first dose or after < 3 doses-without re-intubation before 28 days of age
- RSS on nasal continuous positive airway pressure (NCPAP) < 1.5 or on nasal cannula Fi02 <25% at < 2L/min at 28 days of age, persisting for at least 72 hours.
- Cumulative supplemental oxygen < 4.2 from time of enrollment to 28 days of age 4
- No respiratory support at 28 days, including no supplemental oxygen by nasal cannula
AND a > 50% suppression of the tracheal aspirate interleukin-8 (IL-8) or CCL2 (chemokine ligand 2) at 24-72 hours (or prior to extubation if occurs at <24 hours after dosing) after the initial dose in 5 of 8 of the infants (this may be a different combination of 5 patients than those who met the above criteria).
Measured: 28 days of life for each dosing group
The Clinical and Anti-inflammatory Efficacy of Escalating Doses of Budesonide Suspended in Calfactant and Given Into the Lungs of ELGANs by Monitoring Tracheal Aspirate Cytokine Levels.
Number of ELGANS with a clinical AND Cytokine response: a > 50% suppression of the tracheal aspirate interleukin-8 or chemokine ligand 2 at 24-72 hours (or prior to extubation if occurs at <24 hours after dosing) after the initial dose in 5 of 8 of the infants
Measured: 28 days of life for each dosing group
Secondary outcomes (5)
Adverse Events/Subject Safety
Safety assessments will include the subject's vital signs, clinical laboratory testing, morbidities associated with prematurity, morbidities associated with administration of budesonide in calfactant, and Adverse Events (AE)s. Clinical parameters/ AEs of interest are those potentially consistent with elevated glucocorticoid levels and will be specifically evaluated. A Data and Safety Monitoring Board (DSMB) will be established to review safety data. All of the data will be reviewed to monitor subject safety.
Measured: Through 28 days of life
Serial Budesonide Levels, Peak
The research team will obtain dried blood spot samples at 15 minutes, and 1 and 4 hours after the first dose of budesonide in calfactant for budesonide levels. Trough concentrations (Cmin) will be drawn before potential subsequent daily doses.
Measured: At 15 minutes, 1 and 4 hours after first dose and prior to each additional dose
Serial Budesonide Levels, T 1/2
The research team will obtain dried blood spot samples at 15 minutes, and 1 and 4 hours after the first dose of budesonide in calfactant for budesonide levels. Trough concentrations (Cmin) will be drawn before potential subsequent daily doses.
Measured: At 15 minutes, 1 and 4 hours after first dose and prior to each additional dose
Serial Budesonide Levels, AUC
The research team will obtain dried blood spot samples at 15 minutes, and 1 and 4 hours after the first dose of budesonide in calfactant for budesonide levels. Trough concentrations (Cmin) will be drawn before potential subsequent daily doses.
Measured: At 15 minutes, 1 and 4 hours after first dose and prior to each additional dose
Respiratory Severity Score: Mean Airway Pressure and Oxygen Requirement at 28 Days of Age
Clinical respiratory status (RSS and oxygen requirement) around dosing and at 28 days of age will be compared between dosing groups and also compared to the matched historical control patients from the "Trial of Late Surfactant:(TOLSURF) study.
The respiratory severity score (RSS) is a simplified severity score consisting of the mean airway pressure (MAP) multiplied by the fraction of inspired oxygen (FiO2). This score ranges from 0 to 12, with a higher score indicating more severe disease.
Measured: 28 days of life for each dosing group
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Sep 2016 (estimated)
- Study startSep 2016
- Primary completionApr 2018
- Study completionApr 2018
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.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Bronchopulmonary Dysplasia (BPD)Studies 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 1/2
- Primary purpose
- Prevention
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cynthia McEvoy
- Sponsor type
- University, hospital or other
- Collaborators
- Thrasher Research Fund, University of Florida, Florida Hospital for Children, University of California, San Francisco
- Responsible party
- Sponsor-investigator, Cynthia McEvoy, Oregon Health and Science University
- Investigators
- Cynthia McEvoy, MD, MCR, Principal investigator, Oregon Health and Science University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02907593
- Sponsor's protocol ID
- SASSIE-001
- Acronym
- SASSIE
- Status last verified
- Mar 2025
- First submitted
- 2 Aug 2016
- Last updated
- 4 Apr 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Yeh TF, Chen CM, Wu SY, Husan Z, Li TC, Hsieh WS, Tsai CH, Lin HC. Intratracheal Administration of Budesonide/Surfactant to Prevent Bronchopulmonary Dysplasia. Am J Respir Crit Care Med. 2016 Jan 1;193(1):86-95. doi: 10.1164/rccm.201505-0861OC.
PubMed 26351971Yeh TF, Lin HC, Chang CH, Wu TS, Su BH, Li TC, Pyati S, Tsai CH. Early intratracheal instillation of budesonide using surfactant as a vehicle to prevent chronic lung disease in preterm infants: a pilot study. Pediatrics. 2008 May;121(5):e1310-8. doi: 10.1542/peds.2007-1973. Epub 2008 Apr 21.
PubMed 18426851Roberts JK, Stockmann C, Dahl MJ, Albertine KH, Egan E, Lin Z, Reilly CA, Ballard PL, Ballard RA, Ward RM. Pharmacokinetics of Budesonide Administered with Surfactant in Premature Lambs: Implications for Neonatal Clinical Trials. Curr Clin Pharmacol. 2016;11(1):53-61. doi: 10.2174/1574884710666150929100210.
PubMed 26416605Barrette AM, Roberts JK, Chapin C, Egan EA, Segal MR, Oses-Prieto JA, Chand S, Burlingame AL, Ballard PL. Antiinflammatory Effects of Budesonide in Human Fetal Lung. Am J Respir Cell Mol Biol. 2016 Nov;55(5):623-632. doi: 10.1165/rcmb.2016-0068OC.
PubMed 27281349McEvoy CT, Ballard PL, Ward RM, Rower JE, Wadhawan R, Hudak ML, Weitkamp JH, Harris J, Asselin J, Chapin C, Ballard RA. Dose-escalation trial of budesonide in surfactant for prevention of bronchopulmonary dysplasia in extremely low gestational age high-risk newborns (SASSIE). Pediatr Res. 2020 Oct;88(4):629-636. doi: 10.1038/s41390-020-0792-y. Epub 2020 Feb 1.
PubMed 32006953
Study at a glance
From the registry record, last updated by the sponsor on 4 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Steroids and Surfactant in Extremely Low Gestation Age Infants Pilot Dose Escalation Trial
- ClinicalTrials.gov ID
- NCT02907593
- Acronym / sponsor ID
- SASSIE / SASSIE-001
- Conditions
- Bronchopulmonary Dysplasia (BPD)
- Keywords
- Bronchopulmonary dysplasia, Pulmonary, Surfactant, Steroids, Extremely low gestational age
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Cynthia McEvoyUniversity, hospital or other
- Collaborators
- Thrasher Research Fund, University of Florida, Florida Hospital for Children, University of California, San Francisco
- Study start
- Sep 2016
- Primary completion
- Apr 2018 (actual)
- Study completion
- Apr 2018 (actual)
- First posted
- 20 Sep 2016 (estimated)
- Results
- Posted 4 Apr 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a phase I/II open-label study to determine the lowest, safe, effective dose of budesonide given with calfactant as the vehicle.
Detailed description
This is a phase I/II open-label study to determine the lowest safe, effective dose of budesonide given with calfactant as the vehicle; the investigators will perform an unblinded dose escalation study. The investigators will administer four dosing levels of budesonide suspended in calfactant beginning with 0.025 mg/kg of budesonide administered to 8 extremely low gestational age newborns (ELGANs) who are intubated at 3-10 days of age. Daily doses (at the same dosage) will be administered to infants who remain intubated for a potential of 5 total doses in each patient. Subsequent groups of 8 infants each will receive 0.05 mg/kg, 0.10 mg/kg, and 0.15 mg/kg of budesonide in calfactant (up to 5 total doses in each patient). A total of up to 32 infants will be enrolled in the trial. The investigators will evaluate the clinical, laboratory and safety data from each group of treated infants to 28 days of age before moving to the next dosing level of budesonide.
Study design
- Primary purpose
- Prevention
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| 0.025 mg/kg Budesonide | Experimental | Drug: Budesonide in Calfactant0.025 mg/kg Budesonide in Calfactant |
| 0.050 mg/kg Budesonide | Experimental | Drug: Budesonide in Calfactant0.050 mg/kg Budesonide in Calfactant |
| 0.10 mg/kg Budesonide | Experimental | Drug: Budesonide in Calfactant0.10 mg/kg Budesonide in Calfactant |
| 0.15 mg/kg Budesonide | Experimental | Drug: Budesonide in Calfactant0.15 mg/kg Budesonide in Calfactant |
Who may qualify
- Ages
- 3 to 14 days
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- > 23 0/7 and < 27 6/7 weeks of gestational age based on center's best estimate of due date (using earliest obstetrical ultrasound, last menstrual period, examination, and other pertinent available information)
- Day of life 3-14 from the date and time of delivery, with the date of birth being DOL 0
- Intubated and mechanically ventilated and do not anticipate extubation in next 24 hours
Key exclusion criteria (5 listed)
- Serious congenital malformations or chromosomal abnormality
- Likely to be extubated in next 24 hours
- Clinically unstable
- Infants who have received systemic steroids prior to dosing with study medication.
- Infants who have received Indocin, Ibuprofen, or acetaminophen ≤ 96 hours prior to enrollment window ending
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 |
|---|---|---|
| Budesonide in CalfactantAlso: pulmicort respule, calfactant, budesonide, 0186-1986-04Budesonide in Calfactant | Drug | 0.025 mg/kg Budesonide, 0.050 mg/kg Budesonide, 0.10 mg/kg Budesonide, 0.15 mg/kg Budesonide |
Primary outcome
Number of ELGANs With Clinical and Anti-inflammatory Efficacy of Escalating Doses of Budesonide Determined by Monitoring Respiratory Severity Score (RSS)
Time frame: 28 days of life for each dosing group
Dose escalation will be defined as effective at the dose level in which 5 of 8 infants achieve the following: 1. Extubation within 72 hours of first dose or after < 3 doses-without re-intubation before 28 days of age 2. RSS on nasal continuous positive airway pressure (NCPAP) < 1.5 or on nasal cannula Fi02 <25% at < 2L/min at 28 days of age, persisting for at least 72 hours. 3. Cumulative supplemental oxygen < 4.2 from time of enrollment to 28 days of age 4 4. No respiratory support at 28 days, including no supplemental oxygen by nasal cannula AND a > 50% suppression of the tracheal aspirate interleukin-8 (IL-8) or CCL2 (chemokine ligand 2) at 24-72 hours (or prior to extubation if occurs at <24 hours after dosing) after the initial dose in 5 of 8 of the infants (this may be a different combination of 5 patients than those who met the above criteria).
The Clinical and Anti-inflammatory Efficacy of Escalating Doses of Budesonide Suspended in Calfactant and Given Into the Lungs of ELGANs by Monitoring Tracheal Aspirate Cytokine Levels.
Time frame: 28 days of life for each dosing group
Number of ELGANS with a clinical AND Cytokine response: a > 50% suppression of the tracheal aspirate interleukin-8 or chemokine ligand 2 at 24-72 hours (or prior to extubation if occurs at <24 hours after dosing) after the initial dose in 5 of 8 of the infants
5 secondary outcomes
Adverse Events/Subject Safety
Time frame: Through 28 days of life
Serial Budesonide Levels, Peak
Time frame: At 15 minutes, 1 and 4 hours after first dose and prior to each additional dose
Serial Budesonide Levels, T 1/2
Time frame: At 15 minutes, 1 and 4 hours after first dose and prior to each additional dose
Serial Budesonide Levels, AUC
Time frame: At 15 minutes, 1 and 4 hours after first dose and prior to each additional dose
Respiratory Severity Score: Mean Airway Pressure and Oxygen Requirement at 28 Days of Age
Time frame: 28 days of life for each dosing group
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Apr 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 26351971Yeh TF, Chen CM, Wu SY, Husan Z, Li TC, Hsieh WS, Tsai CH, Lin HC. Intratracheal Administration of Budesonide/Surfactant to Prevent Bronchopulmonary Dysplasia. Am J Respir Crit Care Med. 2016 Jan 1;193(1):86-95. doi: 10.1164/rccm.201505-0861OC.
- Background: PubMed 18426851Yeh TF, Lin HC, Chang CH, Wu TS, Su BH, Li TC, Pyati S, Tsai CH. Early intratracheal instillation of budesonide using surfactant as a vehicle to prevent chronic lung disease in preterm infants: a pilot study. Pediatrics. 2008 May;121(5):e1310-8. doi: 10.1542/peds.2007-1973. Epub 2008 Apr 21.
- Background: PubMed 26416605Roberts JK, Stockmann C, Dahl MJ, Albertine KH, Egan E, Lin Z, Reilly CA, Ballard PL, Ballard RA, Ward RM. Pharmacokinetics of Budesonide Administered with Surfactant in Premature Lambs: Implications for Neonatal Clinical Trials. Curr Clin Pharmacol. 2016;11(1):53-61. doi: 10.2174/1574884710666150929100210.
- Background: PubMed 27281349Barrette AM, Roberts JK, Chapin C, Egan EA, Segal MR, Oses-Prieto JA, Chand S, Burlingame AL, Ballard PL. Antiinflammatory Effects of Budesonide in Human Fetal Lung. Am J Respir Cell Mol Biol. 2016 Nov;55(5):623-632. doi: 10.1165/rcmb.2016-0068OC.
- Results publication: PubMed 32006953McEvoy CT, Ballard PL, Ward RM, Rower JE, Wadhawan R, Hudak ML, Weitkamp JH, Harris J, Asselin J, Chapin C, Ballard RA. Dose-escalation trial of budesonide in surfactant for prevention of bronchopulmonary dysplasia in extremely low gestational age high-risk newborns (SASSIE). Pediatr Res. 2020 Oct;88(4):629-636. doi: 10.1038/s41390-020-0792-y. Epub 2020 Feb 1.
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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.
Cynthia McEvoy, MD, MCR principal investigator
Oregon Health and Science University
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Budesonide in Calfactant in people with Bronchopulmonary Dysplasia (BPD), run by Cynthia McEvoy at 5 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 3 inclusion and 5 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: stopped early, as of the sponsor's last update on 4 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined 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.
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 4 Apr 2025). 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?
Number of ELGANs With Clinical and Anti-inflammatory Efficacy of Escalating Doses of Budesonide Determined by Monitoring Respiratory Severity Score (RSS) (time frame: 28 days of life for each dosing group). 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 NCT02907593, which also lists 5 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 4 Apr 2025.