Hydrocortisone for BPD

NCT ID: NCT01353313

Last Updated: 2025-02-27

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

800 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-08-11

Study Completion Date

2024-09-12

Brief Summary

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The Hydrocortisone and Extubation study will test the safety and efficacy of a 10 day course of hydrocortisone for infants who are less than 30 weeks estimated gestational age and who are intubated at 14-28 days of life. Infants will be randomized to receive hydrocortisone or placebo. This study will determine if hydrocortisone improves infants'survival without moderate or severe BPD and will be associated with improvement in survival without moderate or severe neurodevelopmental impairment at 22 - 26 months corrected age.

Detailed Description

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Bronchopulmonary dysplasia (BPD) remains a leading morbidity of the extremely preterm infant, and prolonged mechanical ventilation is associated with increased risk for BPD. Dexamethasone has been used previously to facilitate extubation and decrease the incidence of BPD; however, due to adverse effects on neurodevelopmental outcomes, the use of this drug has decreased. One cohort study suggests that hydrocortisone (HC) may facilitate extubation. HC has thus far not been associated with adverse neurodevelopmental outcomes in either cohort studies or randomized controlled trials. A recent meta-analysis of postnatal corticosteroid therapy begun after the first week of life suggested that "late therapy may reduce neonatal mortality without significantly increasing the risk of adverse long-term neurodevelopmental outcomes," although the methodological quality of some of the follow-up was acknowledged to be limited.

This is a randomized controlled trial to study the efficacy and safety of 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. Based on previous Network data these criteria define a population with a risk of death or BPD at 36 weeks postmenstrual age of approximately 65 - 75%. The primary outcome for this study will incorporate both (1) survival without moderate to severe BPD by Network physiologic definition and (2) survival without moderate or severe NDI at 18 - 22 months corrected age. Therefore, the results of this study will be reported only when follow-up data are available unless (1) the trial is stopped early by the DSMC because of strong evidence of benefit or harm, or (2) at the time all subjects have completed treatment the DCC finds a substantial survival benefit favoring hydrocortisone (p\<0.001). Individual study assignment will remain masked until the follow-up is completed. Secondary outcomes will include short term measures such as respiratory morbidities and growth at 36 weeks postmenstrual age and long term measures including growth and other outcomes at 22 - 26 months corrected age.

Secondary studies include:

1. Effect of Hydrocortisone on the Cardiac mass of Premature Intubated Infants - will determine left ventricular mass index at 36 weeks postmenstrual age (or prior to discharge/transfer if after 34 weeks) in infants enrolled in the hydrocortisone for BPD RCT, and compare HC-treated infants to placebo-treated infants. It will similarly assess and compare the incidence of pulmonary hypertension in these patients.
2. 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 at early school age. In a subset of five Neonatal Research Network Clinical Centers, impulse oscillometry (IOS), which is the optimal direct measure of lung capacity and function, will be performed to validate the 6-minute walk test and International Study of Asthma and Allergies in Childhood (ISAAC) questionnaire as functional measures of pulmonary status. Also at these five Centers, the six minute walk test, ISAAAC questionnaire, and IOS will be administered as part of (1) the Healthy Lungs sub-study, which will recruit 120 TOP 5 study participants who had minimal lung disease when they were infants to define normative ranges in healthy, preterm-born children, and (2) the Healthy Lungs Two sub-study, which will recruit 120 healthy, term-born children without history of lung disease to characterize functional and mechanical respiratory outcomes at 5-7 years of age.

Conditions

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Infant, Newborn Infant, Small for Gestational Age Infant, Very Low Birth Weight Infant, Premature Bronchopulmonary Dysplasia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

PREVENTION

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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Placebo

Saline placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Saline placebo to be administered either intravenously or orally if no intravenous line is available, at the same dose, and tapered as follows:

4mg/kg/day ¸ q 6 hours x 2 days, then 2mg/kg/day ¸ q 6 hours x 3 days; then

1mg/kg/day ¸ q 12 hours x 3 days; then 0.5mg/kg/d as a single dose x 2 days

Hydrocortisone

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 Type EXPERIMENTAL

Hydrocortisone

Intervention Type DRUG

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), to be administered either intravenously or orally if no intravenous line is available at the same dose, and tapered as follows:

4mg/kg/day ¸ q 6 hours x 2 days, then 2mg/kg/day ¸ q 6 hours x 3 days; then

1mg/kg/day ¸ q 12 hours x 3 days; then 0.5mg/kg/d as a single dose x 2 days

Interventions

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Hydrocortisone

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), to be administered either intravenously or orally if no intravenous line is available at the same dose, and tapered as follows:

4mg/kg/day ¸ q 6 hours x 2 days, then 2mg/kg/day ¸ q 6 hours x 3 days; then

1mg/kg/day ¸ q 12 hours x 3 days; then 0.5mg/kg/d as a single dose x 2 days

Intervention Type DRUG

Placebo

Saline placebo to be administered either intravenously or orally if no intravenous line is available, at the same dose, and tapered as follows:

4mg/kg/day ¸ q 6 hours x 2 days, then 2mg/kg/day ¸ q 6 hours x 3 days; then

1mg/kg/day ¸ q 12 hours x 3 days; then 0.5mg/kg/d as a single dose x 2 days

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* infants \<30 weeks estimated gestational age
* inborn at an NRN site or were admitted to an NRN site before 72 hours postnatal age
* have received at least 7days of mechanical ventilation;
* are receiving mechanical ventilation through an endotracheal tube .

Exclusion Criteria

* Major congenital anomalies
* Decision to limit support
* Indomethacin or ibuprofen treatment within 48 hours of study drug
* Previous corticosteroid treatment for BPD
* Received hydrocortisone for 14 or more cumulative days
* Received hydrocortisone within 7 days of study entry
Maximum Eligible Age

30 Weeks

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Center for Research Resources (NCRR)

NIH

Sponsor Role collaborator

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

NIH

Sponsor Role collaborator

National Heart, Lung, and Blood Institute (NHLBI)

NIH

Sponsor Role collaborator

National Center for Advancing Translational Sciences (NCATS)

NIH

Sponsor Role collaborator

NICHD Neonatal Research Network

NETWORK

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Michele C Walsh, MD

Role: PRINCIPAL_INVESTIGATOR

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

Seetha Shankaran, MD

Role: PRINCIPAL_INVESTIGATOR

Wayne State University

Abbot R Laptook, MD

Role: PRINCIPAL_INVESTIGATOR

Brown University, Women & Infants Hospital of Rhode Island

C. Michael Cotten, MD

Role: PRINCIPAL_INVESTIGATOR

Duke University

David Carlton, MD

Role: PRINCIPAL_INVESTIGATOR

Emory University

Greg Sokol, MD

Role: PRINCIPAL_INVESTIGATOR

Indiana University

Abhik Das, PhD

Role: PRINCIPAL_INVESTIGATOR

RTI International

Krisa P Van Meurs, MD

Role: PRINCIPAL_INVESTIGATOR

Stanford University

Brenda P Poindexter, MD

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital Medical Center, Cincinnati

Waldemar A Carlo, MD

Role: PRINCIPAL_INVESTIGATOR

University of Alabama at Birmingham

Edward F Bell, MD

Role: PRINCIPAL_INVESTIGATOR

University of Iowa

Kristi L Watterberg, MD

Role: STUDY_CHAIR

University of New Mexico

Myra Wyckoff, MD

Role: PRINCIPAL_INVESTIGATOR

University of Texas, Southwestern Medical Center at Dallas

Jon E Tyson, MD, MPH

Role: PRINCIPAL_INVESTIGATOR

The University of Texas Health Science Center, Houston

Eric Eichenwald, MD

Role: PRINCIPAL_INVESTIGATOR

University of Pennsylvania

Carl T D'Angio, MD

Role: PRINCIPAL_INVESTIGATOR

University of Rochester

Uday Devaskar, MD

Role: PRINCIPAL_INVESTIGATOR

University of California, Los Angeles

Pablo J Sanchez, MD

Role: PRINCIPAL_INVESTIGATOR

Research Institute at Nationwide Children's Hospital

William Truog, MD

Role: PRINCIPAL_INVESTIGATOR

Children's Mercy Hospital Kansas City

Bradley Yoder, MD

Role: PRINCIPAL_INVESTIGATOR

University of Utah

Locations

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University of Alabama at Birmingham

Birmingham, Alabama, United States

Site Status

University of California - Los Angeles

Los Angeles, California, United States

Site Status

Stanford University

Palo Alto, California, United States

Site Status

Emory University

Atlanta, Georgia, United States

Site Status

Indiana University

Indianapolis, Indiana, United States

Site Status

University of Iowa

Iowa City, Iowa, United States

Site Status

Wayne State University

Detroit, Michigan, United States

Site Status

Children's Mercy Hospital

Kansas City, Missouri, United States

Site Status

University of New Mexico

Albuquerque, New Mexico, United States

Site Status

University of Rochester

Rochester, New York, United States

Site Status

RTI International

Durham, North Carolina, United States

Site Status

Duke University

Durham, North Carolina, United States

Site Status

Cincinnati Children's Medical Center

Cincinnati, Ohio, United States

Site Status

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

Cleveland, Ohio, United States

Site Status

Research Institute at Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status

Univeristy of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status

Brown University, Women & Infants Hospital of Rhode Island

Providence, Rhode Island, United States

Site Status

University of Texas Southwestern Medical Center at Dallas

Dallas, Texas, United States

Site Status

University of Texas Health Science Center at Houston

Houston, Texas, United States

Site Status

University of Utah

Salt Lake City, Utah, United States

Site Status

Countries

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United States

References

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DeMauro SB, Kirpalani H, Ziolkowski K, Hintz S, Watterberg K, Lowe J, Shankaran S, Chawla S, Vohr B, Msall M, D'Angio C, Yoder BA, Lai K, Winter S, Colaizy T, Merhar S, Bann CM, Trotta M, Newman J, Natarajan A, Das A. The HYdrocortisone for Bronchopulmonary Dysplasia Respiratory and Developmental (HYBRiD) outcomes study: protocol for a longitudinal cohort study. BMC Pediatr. 2024 Nov 14;24(1):737. doi: 10.1186/s12887-024-05198-9.

Reference Type DERIVED
PMID: 39543521 (View on PubMed)

Gentle SJ, Rysavy MA, Li L, Laughon MM, Patel RM, Jensen EA, Hintz S, Ambalavanan N, Carlo WA, Watterberg K; National Institute of Child Health and Human Development Neonatal Research Network. Heterogeneity of Treatment Effects of Hydrocortisone by Risk of Bronchopulmonary Dysplasia or Death Among Extremely Preterm Infants in the National Institute of Child Health and Human Development Neonatal Research Network Trial: A Secondary Analysis of a Randomized Clinical Trial. JAMA Netw Open. 2023 May 1;6(5):e2315315. doi: 10.1001/jamanetworkopen.2023.15315.

Reference Type DERIVED
PMID: 37256621 (View on PubMed)

Watterberg KL, Walsh MC, Li L, Chawla S, D'Angio CT, Goldberg RN, Hintz SR, Laughon MM, Yoder BA, Kennedy KA, McDavid GE, Backstrom-Lacy C, Das A, Crawford MM, Keszler M, Sokol GM, Poindexter BB, Ambalavanan N, Hibbs AM, Truog WE, Schmidt B, Wyckoff MH, Khan AM, Garg M, Chess PR, Reynolds AM, Moallem M, Bell EF, Meyer LR, Patel RM, Van Meurs KP, Cotten CM, McGowan EC, Hines AC, Merhar S, Peralta-Carcelen M, Wilson-Costello DE, Kilbride HW, DeMauro SB, Heyne RJ, Mosquera RA, Natarajan G, Purdy IB, Lowe JR, Maitre NL, Harmon HM, Hogden LA, Adams-Chapman I, Winter S, Malcolm WF, Higgins RD; Eunice Kennedy Shriver NICHD Neonatal Research Network. Hydrocortisone to Improve Survival without Bronchopulmonary Dysplasia. N Engl J Med. 2022 Mar 24;386(12):1121-1131. doi: 10.1056/NEJMoa2114897.

Reference Type DERIVED
PMID: 35320643 (View on PubMed)

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Document Type: Informed Consent Form

View Document

Related Links

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http://neonatal.rti.org/

NICHD Neonatal Research Network site

Other Identifiers

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U10HD034216

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U10HD027904

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U10HD021364

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U10HD027853

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U10HD040689

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U10HD040492

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U10HD027851

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U10HD021373

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U10HD027856

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U10HD053109

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U10HD036790

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U10HD027880

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U10HD053089

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U10HD021385

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U10HD068244

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U10HD068263

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U10HD068270

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U10HD068278

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U10HD068284

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U24HL137729

Identifier Type: NIH

Identifier Source: secondary_id

View Link

1UG3HL137872

Identifier Type: NIH

Identifier Source: secondary_id

View Link

NICHD-NRN-0045

Identifier Type: -

Identifier Source: org_study_id

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