Diuretic Therapy for Bronchopulmonary Dysplasia
(PRIMED Trial)
Trial Summary
Will I have to stop taking my current medications?
The trial requires that participants stop taking any longer-acting diuretics at least 5 days before enrollment. If you are currently on chronic diuretics, you will need to stop those as well. However, treatment with chronic steroids for adrenal insufficiency or cardiovascular instability is allowed.
What data supports the effectiveness of the drug Furosemide for treating bronchopulmonary dysplasia?
Research shows that diuretics like Furosemide are often used in infants with bronchopulmonary dysplasia to improve lung function, although the evidence supporting their effectiveness is limited and mixed. Some studies suggest potential benefits in pulmonary outcomes, but they also highlight concerns about side effects like poor weight gain.12345
Is furosemide safe for humans?
How is the drug Furosemide unique in treating bronchopulmonary dysplasia?
What is the purpose of this trial?
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?
Research Team
Anna Maria C. Hibbs, MD, MSCE
Principal Investigator
1. Rainbow Babies and Children's Hospital
Heather Kaplan, MD, MSCE
Principal Investigator
Children's Hospital Medical Center, Cincinnati
Eligibility Criteria
This trial is for premature babies with a lung condition called bronchopulmonary dysplasia (BPD), who are expected to be hospitalized for at least 28 days, can eat enough, need extra oxygen, and were born before the 28th week of pregnancy. Babies on certain steroids or diuretics recently, those with specific blood test results or major birth defects aren't eligible.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
N-of-1 Trial
Each individual N-of-1 trial will have 2 blocks where patients crossover between furosemide and placebo to determine short-term response
Follow-up
Participants are monitored for safety and effectiveness after the N-of-1 trial
Treatment Details
Interventions
- Furosemide
- Placebo
Furosemide is already approved in European Union, United States, Canada, Japan, China for the following indications:
- Hypertension
- Edema associated with congestive heart failure
- Liver cirrhosis
- Renal disease
- Nephrotic syndrome
- Edema associated with congestive heart failure
- Liver cirrhosis
- Renal disease
- Nephrotic syndrome
- Acute pulmonary edema
- Edema associated with congestive heart failure
- Liver cirrhosis
- Renal disease
- Nephrotic syndrome
- Hypertension
- Edema associated with congestive heart failure
- Liver cirrhosis
- Renal disease
- Nephrotic syndrome
- Edema associated with congestive heart failure
- Liver cirrhosis
- Renal disease
- Nephrotic syndrome
- Hypertension
Find a Clinic Near You
Who Is Running the Clinical Trial?
Children's Hospital Medical Center, Cincinnati
Lead Sponsor
Rainbow Babies and Children's Hospital
Collaborator
Emory University
Collaborator
RTI International
Collaborator
National Heart, Lung, and Blood Institute (NHLBI)
Collaborator