Steroids and Surfactant in Extremely Low Gestation Age Infants Dose Escalation Trial

NCT ID: NCT02907593

Last Updated: 2025-04-04

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 25 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-09-30
• Study Completion Date: 2018-04-30

Brief 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.

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)

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: PREVENTION
• Blinding Strategy: NONE

Study Groups

0.025 mg/kg Budesonide

0.025 mg/kg Budesonide in Calfactant

Group Type: EXPERIMENTAL

Budesonide in Calfactant

Intervention Type: DRUG

Budesonide in Calfactant

0.050 mg/kg Budesonide

0.050 mg/kg Budesonide in Calfactant

Group Type: EXPERIMENTAL

Budesonide in Calfactant

Intervention Type: DRUG

Budesonide in Calfactant

0.10 mg/kg Budesonide

0.10 mg/kg Budesonide in Calfactant

Group Type: EXPERIMENTAL

Budesonide in Calfactant

Intervention Type: DRUG

Budesonide in Calfactant

0.15 mg/kg Budesonide

0.15 mg/kg Budesonide in Calfactant

Group Type: EXPERIMENTAL

Budesonide in Calfactant

Intervention Type: DRUG

Budesonide in Calfactant

Interventions

Budesonide in Calfactant

Budesonide in Calfactant

Intervention Type: DRUG

Other Intervention Names

pulmicort respule; calfactant; budesonide; 0186-1986-04; infasurf

Eligibility Criteria

Inclusion Criteria

1. > 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)

2. Day of life 3-14 from the date and time of delivery, with the date of birth being DOL 0

3. Intubated and mechanically ventilated and do not anticipate extubation in next 24 hours

Exclusion Criteria

1. Serious congenital malformations or chromosomal abnormality

2. Likely to be extubated in next 24 hours

3. Clinically unstable

4. Infants who have received systemic steroids prior to dosing with study medication.

5. Infants who have received Indocin, Ibuprofen, or acetaminophen ≤ 96 hours prior to enrollment window ending

• Minimum Eligible Age: 3 Days
• Maximum Eligible Age: 14 Days
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Thrasher Research Fund

OTHER

Sponsor Role: collaborator

University of Florida

OTHER

Sponsor Role: collaborator

Florida Hospital for Children

OTHER

Sponsor Role: collaborator

University of California, San Francisco

OTHER

Sponsor Role: collaborator

Cynthia McEvoy

OTHER

Sponsor Role: lead

Responsible Party

Cynthia McEvoy

Principal Investigator, Professor of Pediatrics

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Cynthia McEvoy, MD, MCR

Role: PRINCIPAL_INVESTIGATOR

Oregon Health and Science University

Locations

University of California, San Francisco

San Francisco, California, United States

University of Florida, Jacksonville

Jacksonville, Florida, United States

Florida Hospital for Children

Orlando, Florida, United States

Oregon Health ans Science University

Portland, Oregon, United States

Vanderbilt Children's Hospital

Nashville, Tennessee, United States

Countries

United States

References

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.

Reference Type: BACKGROUND

PMID: 26351971 (View on PubMed)

Yeh 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.

Reference Type: BACKGROUND

PMID: 18426851 (View on PubMed)

Roberts 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.

Reference Type: BACKGROUND

PMID: 26416605 (View on PubMed)

Barrette 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.

Reference Type: BACKGROUND

PMID: 27281349 (View on PubMed)

McEvoy 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.

Reference Type: RESULT

PMID: 32006953 (View on PubMed)

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

SASSIE-001

Identifier Type: -

Identifier Source: org_study_id