Pragmatic Research on Diuretic Management in Early BPD Pilot

Last updated: March 31, 2025
Sponsor: Children's Hospital Medical Center, Cincinnati
Overall Status: Active - Recruiting

Phase

4

Condition

Lung Disease

Treatment

Furosemide (plus potassium chloride)

Placebo (plus placebo electrolyte solution)

Clinical Study ID

NCT05898022
2021-0120
R34HL158586-01A1
  • Ages 2-10
  • All Genders

Study 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?

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. <28 weeks gestation at birth

  2. Post-Menstrual Age (PMA) of 29-32 weeks gestation

  3. Requiring invasive positive pressure respiratory support or NIPPV/NIMV and FiO2 ≥ 25% or requiring non-invasive positive pressure respiratory support (NCPAP≥ 5 cmH20, BiPhasic CPAP) and FiO2 ≥ 30%.

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

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

Exclusion

Exclusion Criteria:

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

  2. In infants who had electrolyte testing in the week prior to enrollment, those with aserum creatinine > 1.7 mg/dL, BUN >50 mg/dL, Na <125 mmoL/L, K ≤ 2.5 mmol/L, or Ca ≤ 6 mg/dL. Not having electrolyte testing in the week prior to enrollment is not anexclusion criterion.

  3. Current treatment with Dexamethasone or hydrocortisone for respiratory failure.Treatment with chronic steroids for history of adrenal insufficiency orcardiovascular instability is not an exclusion criterion.

  4. Treatment with any longer-acting diuretic (e.g., chlorothiazide,hydrochlorothiazide, acetazolamide) within 5 days of enrollment where exposure mayresult in carryover effects that confound the N-of-1 trial

  5. Active order for standing, regularly scheduled diuretics (e.g., chronic diuretics)

  6. Non-English speaking

  7. Current treatment with ibuprofen or indocin

Study Design

Total Participants: 30
Treatment Group(s): 2
Primary Treatment: Furosemide (plus potassium chloride)
Phase: 4
Study Start date:
August 17, 2023
Estimated Completion Date:
January 31, 2026

Connect with a study center

  • Emory University

    Atlanta, Georgia 30303
    United States

    Active - Recruiting

  • RTI International

    Durham, North Carolina 27705
    United States

    Site Not Available

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio 45229
    United States

    Active - Recruiting

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

    Cleveland, Ohio 44106
    United States

    Active - Recruiting

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