Pragmatic Research on Diuretic Management in Early BPD Pilot

• Status: Not yet recruiting
• Trial end date: 2025-09-30
• Target enrollment: 0
• Participant gender: All

Summary

Babies who are born prematurely often develop a chronic lung disease called bronchopulmonary dysplasia (BPD). BPD puts babies at higher risk for problems with growth and development. Diuretics, such as furosemide, are frequently used in the management of early BPD). Many clinicians use informal trials of therapy to see if a baby responds to diuretics in the short-term before starting chronic diuretic therapy. Despite frequent use of diuretics, it is unclear how many babies truly respond to therapy and if there are long-term benefits of diuretic treatment. Designing research studies to figure this out has been challenging. The Pragmatic Research on Diuretic Management in Early BPD (PRIMED) study is a feasibility pilot study to help us get information to design a larger trial of diuretic management for BPD. Key questions this study will answer include: (1) Can we use an N-of-1 trial to determine whether a particular baby responds to furosemide? In an N-of-1 trial, a baby is switched between furosemide and placebo to compare that particular infant's response on and off diuretics. It is a more rigorous approach to the informal trials of therapy that are often conducted in clinical care. We hope to learn how many babies have a short-term response to furosemide ("responders"); (2) how many babies will still be on respiratory support at the end of the N-of-1 trial? This will help us determine how many patients would be eligible to randomize to chronic diuretic therapy in the second phase of the larger trail, and (3) if a baby is identified as a short-term responder, how many parents and physicians would be willing to randomize the baby to chronic diuretics (3 months) versus placebo in the longer trial?

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Children's Hospital Medical Center, Cincinnati
• Collaborators: Emory University; National Heart, Lung, and Blood Institute (NHLBI); Rainbow Babies and Children's Hospital; RTI International
• Treatments: Furosemide

Criteria

Inclusion Criteria:

1. <28 weeks gestation at birth

2. Post-Menstrual Age (PMA) of 30-32 weeks gestation

3. Requiring positive pressure respiratory support (NCPAP≥ 5 cm H20, NIPPV/BiPhasic CPAP, or mechanical ventilation)

4. Receiving FiO2 ≥ 30%

5. Receiving enteral feedings of 120 mL/kg/day or greater

6. Expected to be hospitalized for at least 28 days after enrollment

Exclusion Criteria:

1. Major congenital anomalies (e.g., known renal anomalies, congenital heart disease, congenital diaphragmatic hernia, or chromosomal anomalies)

2. Serum creatinine > 1.7 mg/dL, BUN >50 mg/dL, Na <125 mmoL/L, K ≤ 2.5 mmol/L, or Ca ≤ 6 mg/dL in week prior to enrollment

3. Treatment with a time-limited course of Dexamethasone or hydrocortisone within 10 days of enrollment. Exposure to a time-limited course dexamethasone or hydrocortisone for respiratory failure within 10 days of enrollment may result in carryover effects that confound the N-of-1 trial. Treatment with chronic steroids for adrenal insufficiency is not an exclusion criteria.

4. Treatment with any diuretics within 7 days of enrollment. Exposure to diuretics within 7 days of enrollment may result in carryover effects that confound the N-of-1 trial.

5. Non-English speaking