Hydrocortisone for Bronchopulmonary Dysplasia

No longer recruiting at 19 trial locations

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: NICHD Neonatal Research Network

Must not be taking: Indomethacin, Ibuprofen, Corticosteroids

Disqualifiers: Major congenital anomalies, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 10 days

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests whether hydrocortisone, a type of steroid, can help premature babies needing breathing support avoid severe lung problems, known as bronchopulmonary dysplasia (BPD). Infants in the study will receive either hydrocortisone or a placebo (a harmless substance with no effect). Researchers aim to determine if this treatment improves survival chances without major lung or brain issues by the age of two. Babies born before 30 weeks who remain on a breathing machine after about two weeks may be suitable for this trial. As a Phase 3 trial, this study represents the final step before FDA approval, offering a promising opportunity to contribute to a potentially life-saving treatment.

Will I have to stop taking my current medications?

The trial does not specify if participants must stop taking their current medications, but infants who have received certain treatments like indomethacin or ibuprofen within 48 hours, or hydrocortisone recently, are excluded.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research shows that hydrocortisone is under study for its potential benefits in babies at risk for bronchopulmonary dysplasia (BPD), a lung condition. Previous studies have found that hydrocortisone may lower the risk of developing BPD in very premature babies, suggesting it might help some babies breathe better. However, the long-term effects of using hydrocortisone in these infants remain unclear.

Studies have not found major differences in important outcomes, like survival without severe BPD, when comparing hydrocortisone to a placebo. While the treatment appears promising, more information is needed to confirm its long-term safety and effectiveness.

Hydrocortisone is usually well-tolerated in other situations, and side effects have been minimal. Since this study is in the later stages of testing, the treatment has passed earlier safety checks. This is reassuring, but participants should discuss potential risks with their healthcare providers. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Most treatments for bronchopulmonary dysplasia (BPD) involve the use of corticosteroids like dexamethasone to reduce inflammation in the lungs. However, hydrocortisone is unique because it provides a potentially safer alternative, with fewer side effects, especially concerning brain development in premature infants. Unlike other steroids, hydrocortisone may offer a gentler approach that reduces the risk of neurodevelopmental issues. Researchers are excited about hydrocortisone because it might offer the benefits of inflammation reduction while minimizing harm, making it a promising option for vulnerable newborns.

What evidence suggests that hydrocortisone might be an effective treatment for bronchopulmonary dysplasia?

Research has shown that hydrocortisone, which participants in this trial may receive, can lower the risk of bronchopulmonary dysplasia (BPD) in very premature babies. One study found that babies who received a low dose of hydrocortisone early had a reduced chance of developing BPD. Another review indicated that early use of hydrocortisone decreased death rates in these babies, although it did not significantly impact BPD at 36 weeks. As a type of steroid, hydrocortisone helps reduce swelling, which may explain its effectiveness. Overall, these studies suggest that hydrocortisone can be beneficial, especially when administered early. ^{2 3 6 7 8}

Who Is on the Research Team?

Uday Devaskar, MD

Principal Investigator

University of California, Los Angeles

Pablo J Sanchez, MD

Principal Investigator

Research Institute at Nationwide Children's Hospital

Bradley Yoder, MD

Principal Investigator

University of Utah

C. Michael Cotten, MD

Principal Investigator

Duke University

David Carlton, MD

Principal Investigator

Emory University

Abhik Das, PhD

Principal Investigator

RTI International

Krisa P Van Meurs, MD

Principal Investigator

Stanford University

Edward F Bell, MD

Principal Investigator

University of Iowa

Kristi L Watterberg, MD

Principal Investigator

University of New Mexico

Jon E Tyson, MD, MPH

Principal Investigator

The University of Texas Health Science Center, Houston

Carl T D'Angio, MD

Principal Investigator

University of Rochester

Greg Sokol, MD

Principal Investigator

Indiana University

Brenda P Poindexter, MD

Principal Investigator

Children's Hospital Medical Center, Cincinnati

Abbot R Laptook, MD

Principal Investigator

Brown University, Women & Infants Hospital of Rhode Island

William Truog, MD

Principal Investigator

Children's Mercy Hospital Kansas City

Eric Eichenwald, MD

Principal Investigator

University of Pennsylvania

Michele C Walsh, MD

Principal Investigator

Case Western Reserve University, Rainbow Babies and Children's Hospital

Waldemar A Carlo, MD

Principal Investigator

University of Alabama at Birmingham

Seetha Shankaran, MD

Principal Investigator

Wayne State University

Myra Wyckoff, MD

Principal Investigator

University of Texas, Southwestern Medical Center at Dallas

Are You a Good Fit for This Trial?

This trial is for newborns who are less than 30 weeks old at gestational age, intubated and have been on mechanical ventilation for at least a week. They must be in the hospital from birth or transferred within 72 hours after birth. Babies with major congenital anomalies, those with life support limitations, prior extensive hydrocortisone use, or recent indomethacin/ibuprofen treatment cannot participate.

Inclusion Criteria

I was born at or admitted to an NRN site within 72 hours after birth.

You are using a tube to help you breathe.

My baby was born before 30 weeks of pregnancy.

Exclusion Criteria

You were born with major physical abnormalities.

Choosing to limit assistance or help.

I have taken hydrocortisone in the last week.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

A 10-day tapering course of hydrocortisone treatment for infants <30 weeks estimated gestational age at birth who remain intubated at 14 - 28 days postnatal age

10 days

Follow-up

Participants are monitored for survival without moderate/severe BPD and neurodevelopmental outcomes at 22-26 months corrected age

22-26 months

Extended follow-up

Subjects will be seen for a follow-up visit at 5-6 years corrected age to assess functional developmental and respiratory outcomes

5-6 years

What Are the Treatments Tested in This Trial?

Interventions

• Hydrocortisone
• Placebo

Trial Overview The study tests if a 10-day course of hydrocortisone can help premature infants avoid bronchopulmonary dysplasia (BPD) and improve survival without severe neurodevelopmental issues by age two. Infants will randomly receive either hydrocortisone or a placebo to compare outcomes.

How Is the Trial Designed?

Group I: Hydrocortisone Experimental Treatment 1 Intervention

hydrocortisone sodium succinate for intravenous administration (unpreserved, Solu-Cortef plain, Pfizer®, reconstituted with unpreserved normal saline to avoid exposure to the benzyl alcohol contained in preserved diluents)

Group II: Placebo Placebo Group 1 Intervention

Saline placebo

Hydrocortisone is already approved in European Union, United States, Canada for the following indications:

Approved in European Union as Hydrocortisone for:

• Adrenal insufficiency
• Allergic reactions
• Asthma
• Severe acute pancreatitis

Approved in United States as Hydrocortisone for:

• Adrenal insufficiency
• Allergic reactions
• Asthma
• Severe acute pancreatitis

Approved in Canada as Hydrocortisone for:

• Adrenal insufficiency
• Allergic reactions
• Asthma
• Severe acute pancreatitis

Who Is Running the Clinical Trial?

NICHD Neonatal Research Network

Lead Sponsor

Trials

62

Recruited

209,000+

National Center for Research Resources (NCRR)

Collaborator

Trials

540

Recruited

317,000+

National Heart, Lung, and Blood Institute (NHLBI)

Collaborator

Trials

3,987

Recruited

47,860,000+

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Collaborator

Trials

2,103

Recruited

2,760,000+

National Center for Advancing Translational Sciences (NCATS)

Collaborator

Trials

394

Recruited

404,000+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11562334/

The HYdrocortisone for Bronchopulmonary Dysplasia ... It aims to characterize the childhood functional motor, cognitive, academic, and pulmonary outcomes of this large, well-phenotyped trial cohort.

2. frontiersin.org frontiersin.org/journals/pediatrics/articles/10.3389/fped.2025.1582881/full

Early low-dose hydrocortisone is associated with a ... Early low-dose hydrocortisone is associated with a reduced risk of bronchopulmonary dysplasia in infants born at less than 26 weeks' gestational ...

3. bmcpediatr.biomedcentral.com bmcpediatr.biomedcentral.com/articles/10.1186/s12887-024-05198-9

The HYdrocortisone for Bronchopulmonary Dysplasia ... The HYBRiD Outcomes Study will be the largest and most comprehensive evaluation to date of the functional early school age outcomes of children with a history ...

4. ebneo.org ebneo.org/ebneo-review-hydrocortisone-to-improve-survival-without-bpd/

Hydrocortisone to Improve Survival without ... Per a recent Cochrane review (4), early hydrocortisone was shown to reduce mortality but had little to no effect on BPD at 36 weeks' ...

5. bmjopen.bmj.com bmjopen.bmj.com/content/12/6/e059553

study protocol for a systematic review and meta-analysis Postnatal steroids during the first few weeks of life have been demonstrated to be effective in decreasing the incidence of bronchopulmonary dysplasia (BPD).

6. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39543521/

The HYdrocortisone for Bronchopulmonary Dysplasia ... Longer-term impacts of hydrocortisone exposure and severity of BPD on functional outcomes of high-risk infants remain unknown.

7. trialsjournal.biomedcentral.com trialsjournal.biomedcentral.com/articles/10.1186/s13063-018-2505-y

Systemic Hydrocortisone To Prevent Bronchopulmonary ... The SToP-BPD study is a multicentre, double-blind, placebo-controlled hydrocortisone trial in preterm infants at risk for BPD.

8. elsevier.health elsevier.health/en-US/preview/hydrocortisone

Hydrocortisone Effect of early low-dose hydrocortisone on survival without bronchopulmonary dysplasia ... Study data found no difference in the primary outcome ...