Calfactant + Budesonide for Respiratory Distress Syndrome

Not yet recruiting at 1 trial location

Overseen By Meg Baker

Age: < 18

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: University of Wisconsin, Madison

Must not be taking: Antibiotics, Corticosteroids, Indomethacin, Ibuprofen

Disqualifiers: Congenital anomalies, Pulmonary hemorrhage, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Compensation: Varies

Number of Visits: 3

Duration: 1 week

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests whether a combination of calfactant and budesonide (a corticosteroid) can safely assist extremely preterm babies with breathing problems. The primary goal is to determine if this treatment improves their breathing, measured by the respiratory severity score, while ensuring safety. Babies under 29 weeks old who require machine-assisted breathing 7-14 days after birth may qualify. Parents or guardians must provide consent in English or Spanish for their baby's participation. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group of babies, contributing to significant medical advancements.

Do I need to stop my current medications to join the trial?

The trial protocol does not specify if participants must stop taking their current medications, but it does exclude neonates who have used antibiotics within 48 hours or Indomethacin or Ibuprofen within 72 hours.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that combining budesonide with surfactant is generally safe for babies with breathing problems. Studies have found that this treatment can improve breathing without causing major harm. For example, one study found that babies receiving budesonide with surfactant had better survival rates and were less likely to develop bronchopulmonary dysplasia compared to those who did not receive this treatment.

However, another study found a higher risk of death in babies treated with budesonide alone. This indicates that while the treatment can be beneficial, risks are involved.

Overall, budesonide combined with surfactant is considered well-tolerated, but like any treatment, it can have side effects. This trial aims to provide more information about its safety in premature babies. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about using Budesonide for treating Respiratory Distress Syndrome (RDS) in premature babies because it offers a potentially more targeted approach. Unlike traditional treatments like surfactant replacement therapy, Budesonide is a corticosteroid that helps reduce inflammation in the lungs, which can be pivotal for infants struggling with breathing difficulties. Additionally, combining Budesonide with Calfactant, a type of surfactant, might enhance its effects, potentially improving lung function more effectively than existing options. This novel combination could lead to better outcomes for preterm infants requiring mechanical ventilation.

What evidence suggests that this treatment might be an effective treatment for respiratory distress syndrome?

Research has shown that using budesonide with a surfactant can improve breathing in premature babies. Some studies have found that this combination lowers the risk of developing bronchopulmonary dysplasia (BPD), a common lung issue in preterm infants. Evidence also suggests it might shorten the duration of breathing problems. However, not all studies agree, with some finding little to no effect on survival without BPD. Overall, this treatment shows promise in helping preterm babies breathe better, but results can vary. ^{3 5 6 7 8}

Who Is on the Research Team?

Dinushan Kaluarachchi, MBBS

Principal Investigator

University of Wisconsin, Madison

Are You a Good Fit for This Trial?

The Little Lungs Study is for extremely preterm infants who need mechanical ventilation at 7-14 days old. It's not specified, but typically participants must meet certain health criteria and not have conditions that could interfere with the study or pose additional risks.

Inclusion Criteria

My baby was born between 22 and 28 weeks of pregnancy.

My baby is between 7 to 14 days old.

My baby has a parent or guardian who can consent in English or Spanish.

Exclusion Criteria

Neonate: Not expected to survive for greater than 7 days at enrollment

Neonate: Major congenital anomalies

My newborn has had a lung bleed.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

1-2 weeks

Treatment

Participants receive three doses of the study drug, budesonide, for late surfactant therapy

1 week

3 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, including changes in respiratory severity score and other clinical outcomes

7 days

Long-term follow-up

Participants are monitored for long-term outcomes such as Bronchopulmonary Dysplasia (BPD) grades

up to 36 weeks post menstrual age

What Are the Treatments Tested in This Trial?

Interventions

• Budesonide

Trial Overview This trial tests if giving a combination of two drugs, Calfactant and Budesonide, can improve breathing in these infants. They'll get three doses to see if it helps their respiratory severity score without causing harm.

How Is the Trial Designed?

Group I: Premature babies: Study Intervention Experimental Treatment 2 Interventions

Preterm infants born at gestational age less than 29 0/7 weeks requiring mechanical ventilation at 7-14 days of life (targeting N=12 participants completing study to 7 days post-intervention)

Group II: Premature babies: Standard of Care Active Control 1 Intervention

Preterm infants born at gestational age less than 29 0/7 weeks requiring mechanical ventilation at 7-14 days of life (targeting N=12 participants completing study to 10 days post-randomization)

Who Is Running the Clinical Trial?

University of Wisconsin, Madison

Lead Sponsor

Trials

1,249

Recruited

3,255,000+

Meriter Foundation

Collaborator

Trials

8

Recruited

3,600+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11717239/

Efficacy of pulmonary surfactant with budesonide in premature ... Our study showed that the administration of PS with budesonide significantly improved respiratory outcomes, including the incidence of BPD, duration of ...

2. journals.plos.org journals.plos.org/plosone/article?id=10.1371/journal.pone.0312561

Efficacy of pulmonary surfactant with budesonide in premature ... Our study showed that the administration of PS with budesonide significantly improved respiratory outcomes, including the incidence of BPD, duration of ...

3. bmcpediatr.biomedcentral.com bmcpediatr.biomedcentral.com/articles/10.1186/s12887-024-04736-9

a single-blind randomized clinical trial | BMC Pediatrics Based on the results, the combination of surfactant and budesonide showed promising results in terms of reducing mortality rate and improving ...

4. jamanetwork.com jamanetwork.com/journals/jama/fullarticle/2826118

Intratracheal Budesonide Mixed With Surfactant for ... In extremely preterm infants receiving surfactant, early intratracheal budesonide may have little to no effect on survival free of bronchopulmonary dysplasia.

5. frontiersin.org frontiersin.org/journals/pediatrics/articles/10.3389/fped.2025.1518957/full

Meta-analysis of budesonide and surfactant combination ... This research, combining a meta-analysis and observational data, indicates that Budesonide-Surfactant therapy significantly reduces BPD in preterm neonates ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8717620/

Inhaled Budesonide in Neonatal Respiratory Distress ... This study evaluates the value of inhaled budesonide (BUD) administration in neonatal respiratory distress syndrome (RDS) cases especially for near-term ...

7. onlinelibrary.wiley.com onlinelibrary.wiley.com/doi/abs/10.1002/ppul.25759

Effectiveness and safety of early combined utilization ... This meta-analysis suggested that early combined utilization of budesonide and surfactant by airway might be an effective and safe clinical ...

8. nejm.org nejm.org/doi/full/10.1056/NEJMoa1708831

Long-Term Effects of Inhaled Budesonide for ... Budesonide treatment resulted in a significantly lower risk of bronchopulmonary dysplasia than placebo; however, mortality was higher in the budesonide group.