Pragmatic Research on Diuretic Management in Early Bronchopulmonary Dysplasia (PRIMED) Pilot Study
概览
- 阶段
- 4 期
- 干预措施
- Furosemide (plus potassium chloride)
- 疾病 / 适应症
- Bronchopulmonary Dysplasia
- 发起方
- Children's Hospital Medical Center, Cincinnati
- 入组人数
- 19
- 试验地点
- 4
- 主要终点
- Percent of enrolled infants who completed the full N-of-1 trial, remain on respiratory support at the conclusion of the N-of-1 trial, and were identified as a responder
- 状态
- 已完成
- 最后更新
- 3个月前
概览
简要总结
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?
研究者
Heather Kaplan
Professor of Pediatrics
Children's Hospital Medical Center, Cincinnati
入排标准
入选标准
- •\<28 weeks gestation at birth
- •Post-Menstrual Age (PMA) of 29-32 weeks gestation
- •Requiring invasive positive pressure respiratory support or NIPPV/NIMV and FiO2 ≥ 25% or requiring non-invasive positive pressure respiratory support (NCPAP≥ 5 cm H20, BiPhasic CPAP) and FiO2 ≥ 30%.
- •Receiving enteral feedings of 120 mL/kg/day or greater
- •Expected to be hospitalized for at least 28 days after enrollment
排除标准
- •Major congenital anomalies (e.g., known renal anomalies, congenital heart disease, congenital diaphragmatic hernia, or chromosomal anomalies)
- •In infants who had electrolyte testing in the week prior to enrollment, those with a serum 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 an exclusion criterion.
- •Current treatment with Dexamethasone or hydrocortisone for respiratory failure. Treatment with chronic steroids for history of adrenal insufficiency or cardiovascular instability is not an exclusion criterion.
- •Treatment with any longer-acting diuretic (e.g., chlorothiazide, hydrochlorothiazide, acetazolamide) within 5 days of enrollment where exposure may result in carryover effects that confound the N-of-1 trial
- •Active order for standing, regularly scheduled diuretics (e.g., chronic diuretics)
- •Non-English speaking
- •Current treatment with ibuprofen or indocin
研究组 & 干预措施
N-of-1 Trial
Each individual N-of-1 trial will have 2 blocks. In each block patients will crossover between furosemide (plus potassium chloride) for 4 days and placebo (plus placebo electrolyte solution) for 4 days. The total arm length (length of the N-of-1 Trial/Crossover) is 16 days. Patients may receive furosemide (plus potassium chloride) and placebo (plus placebo electrolyte solution) in one of four different treatment order sequences, however, treatment order will not be analyzed for the primary outcomes of feasibility/responder status.
干预措施: Furosemide (plus potassium chloride)
N-of-1 Trial
Each individual N-of-1 trial will have 2 blocks. In each block patients will crossover between furosemide (plus potassium chloride) for 4 days and placebo (plus placebo electrolyte solution) for 4 days. The total arm length (length of the N-of-1 Trial/Crossover) is 16 days. Patients may receive furosemide (plus potassium chloride) and placebo (plus placebo electrolyte solution) in one of four different treatment order sequences, however, treatment order will not be analyzed for the primary outcomes of feasibility/responder status.
干预措施: Placebo (plus placebo electrolyte solution)
结局指标
主要结局
Percent of enrolled infants who completed the full N-of-1 trial, remain on respiratory support at the conclusion of the N-of-1 trial, and were identified as a responder
时间窗: 23 Days
Percent of providers willing to support randomizing a responder infant
时间窗: 30 Days
次要结局
- Percent of enrolled infants completing full N-of-1 trial and identified as responder(23 days)
- Percent of enrolled infants completing full N-of-1 trial(23 days)
- Percent of enrolled infants on respiratory support at the conclusion of the N-of-1 trial(23 days)
- Percent of parents willing to randomize responder infant(30 days)
- Rate of chronic diuretic use among responders (and non-responders)(30 days)