Trial Outcomes & Findings for Pragmatic Research on Diuretic Management in Early BPD Pilot (NCT NCT05898022)
NCT ID: NCT05898022
Last Updated: 2026-07-14
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE4
Target enrollment
19 participants
Primary outcome timeframe
23 Days
Results posted on
2026-07-14
Participant Flow
Infant screening and enrollment took placed from 08/2023 to 08/2025 at 3 academic centers in the U.S. and their affiliate neonatal intensive care units.
Infant screening occurred in continuous fashion once infant reached 29 weeks post-menstrual age; infants with high likelihood of eligibility could be consented and, then, assigned to the study arm/group only if meeting eligibility criteria.
Participant milestones
| Measure |
ABAB: Infants
Infants randomized to ABAB Sequence (Furosemide + Potassium Chloride, followed by Placebo Diuretic + Electrolyte, followed by Furosemide + Potassium Chloride, followed by Placebo Diuretic + Electrolyte). Furosemide dosing was 2 mg/kg given and potassium chloride dosing was 2 mEq/kg given enterally. The treatment sequence consisted of two treatment blocks with two treatment periods per block. Initially each treatment period was 1 week (2 day washout). Due to lower-than-expected rates of enrollment and N-of-1 trial completion and the intent to explore feasibility barriers as part of the pilot study, after enrolling the first 14 patients, period length was shortened to 4 days (1 day washout).
|
ABBA: Infants
Infants randomized to ABBA Sequence (Furosemide + Potassium Chloride, followed by Placebo Diuretic + Electrolyte, followed by Placebo Diuretic + Electrolyte, followed by Furosemide + Potassium Chloride). Furosemide dosing was 2 mg/kg given and potassium chloride dosing was 2 mEq/kg given enterally. The treatment sequence consisted of two treatment blocks with two treatment periods per block. Initially each treatment period was 1 week (2 day washout). Due to lower-than-expected rates of enrollment and N-of-1 trial completion and the intent to explore feasibility barriers as part of the pilot study, after enrolling the first 14 patients, period length was shortened to 4 days (1 day washout).
|
BAAB: Infants
Infants randomized to BAAB Sequence (Placebo Diuretic + Electrolyte, followed by Furosemide + Potassium Chloride, followed by Furosemide + Potassium Chloride, followed by Placebo Diuretic + Electrolyte). Furosemide dosing was 2 mg/kg given and potassium chloride dosing was 2 mEq/kg given enterally. The treatment sequence consisted of two treatment blocks with two treatment periods per block. Initially each treatment period was 1 week (2 day washout). Due to lower-than-expected rates of enrollment and N-of-1 trial completion and the intent to explore feasibility barriers as part of the pilot study, after enrolling the first 14 patients, period length was shortened to 4 days (1 day washout).
|
BABA: Infants
Infants randomized to BABA Sequence (Placebo Diuretic + Electrolyte, followed by Furosemide + Potassium Chloride, followed by Placebo Diuretic + Electrolyte, followed by Furosemide + Potassium Chloride). Furosemide dosing was 2 mg/kg given and potassium chloride dosing was 2 mEq/kg given enterally. The treatment sequence consisted of two treatment blocks with two treatment periods per block. Initially each treatment period was 1 week (2 day washout). Due to lower-than-expected rates of enrollment and N-of-1 trial completion and the intent to explore feasibility barriers as part of the pilot study, after enrolling the first 14 patients, period length was shortened to 4 days (1 day washout).
|
|---|---|---|---|---|
|
Period 1
STARTED
|
4
|
5
|
5
|
5
|
|
Period 1
COMPLETED
|
3
|
4
|
5
|
5
|
|
Period 1
NOT COMPLETED
|
1
|
1
|
0
|
0
|
|
Period 2
STARTED
|
3
|
4
|
5
|
5
|
|
Period 2
COMPLETED
|
1
|
4
|
4
|
3
|
|
Period 2
NOT COMPLETED
|
2
|
0
|
1
|
2
|
|
Period 3
STARTED
|
1
|
4
|
4
|
3
|
|
Period 3
COMPLETED
|
1
|
4
|
3
|
3
|
|
Period 3
NOT COMPLETED
|
0
|
0
|
1
|
0
|
|
Period 4
STARTED
|
1
|
3
|
3
|
3
|
|
Period 4
COMPLETED
|
1
|
2
|
3
|
3
|
|
Period 4
NOT COMPLETED
|
0
|
1
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Pragmatic Research on Diuretic Management in Early BPD Pilot
Baseline characteristics by cohort
| Measure |
N-of-1 Trial Series: Infants
n=19 Participants
All patients were enrolled in an individual N-of-1 trial with 2 blocks. In each block patients crossover between active treatment and placebo. Participants were analyzed as a single arm (a set of N-of-1 trials), with results reported accordingly.
|
|---|---|
|
Age, Continuous
|
35 days
n=9 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=9 Participants
|
|
Sex: Female, Male
Male
|
13 Participants
n=9 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=9 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=9 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=9 Participants
|
|
Race (NIH/OMB)
Black or African American
|
8 Participants
n=9 Participants
|
|
Race (NIH/OMB)
White
|
11 Participants
n=9 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=9 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=9 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=9 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
19 Participants
n=9 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=9 Participants
|
PRIMARY outcome
Timeframe: 23 DaysOutcome measures
| Measure |
N-of-1 Trial Series: Infants
n=19 Participants
All patients were enrolled in an individual N-of-1 trial with 2 blocks. In each block patients crossover between active treatment and placebo
|
|---|---|
|
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
|
4 Participants
|
PRIMARY outcome
Timeframe: 30 DaysPopulation: Infants with analyzable N-of-1 trial results classified as responders
Outcome measures
| Measure |
N-of-1 Trial Series: Infants
n=4 Participants
All patients were enrolled in an individual N-of-1 trial with 2 blocks. In each block patients crossover between active treatment and placebo
|
|---|---|
|
Percent of Providers Willing to Support Randomizing a Responder Infant
|
4 Participants
|
SECONDARY outcome
Timeframe: 23 daysOutcome measures
| Measure |
N-of-1 Trial Series: Infants
n=19 Participants
All patients were enrolled in an individual N-of-1 trial with 2 blocks. In each block patients crossover between active treatment and placebo
|
|---|---|
|
Percent of Enrolled Infants Completing Full N-of-1 Trial and Identified as Responder
|
4 Participants
|
SECONDARY outcome
Timeframe: 23 daysOutcome measures
| Measure |
N-of-1 Trial Series: Infants
n=19 Participants
All patients were enrolled in an individual N-of-1 trial with 2 blocks. In each block patients crossover between active treatment and placebo
|
|---|---|
|
Percent of Enrolled Infants Completing Full N-of-1 Trial
|
10 Participants
|
SECONDARY outcome
Timeframe: 23 daysPopulation: Infants who completed a full N-of-1 trial
Outcome measures
| Measure |
N-of-1 Trial Series: Infants
n=10 Participants
All patients were enrolled in an individual N-of-1 trial with 2 blocks. In each block patients crossover between active treatment and placebo
|
|---|---|
|
Percent of Enrolled Infants on Respiratory Support at the Conclusion of the N-of-1 Trial
|
10 Participants
|
SECONDARY outcome
Timeframe: 30 daysPopulation: Infants with analyzable N-of-1 trial data classified as responders
Outcome measures
| Measure |
N-of-1 Trial Series: Infants
n=4 Participants
All patients were enrolled in an individual N-of-1 trial with 2 blocks. In each block patients crossover between active treatment and placebo
|
|---|---|
|
Rate of Chronic Diuretic Use Among Responders
|
3 Participants
|
SECONDARY outcome
Timeframe: 30 daysPopulation: Infants with analyzable N-of-1 trial data classified as non-responders
Outcome measures
| Measure |
N-of-1 Trial Series: Infants
n=7 Participants
All patients were enrolled in an individual N-of-1 trial with 2 blocks. In each block patients crossover between active treatment and placebo
|
|---|---|
|
Rate of Chronic Diuretic Use Among Non-responders
|
6 Participants
|
SECONDARY outcome
Timeframe: 30 daysPopulation: Infants with analyzable N-of-1 trial results classified as responders
Outcome measures
| Measure |
N-of-1 Trial Series: Infants
n=4 Participants
All patients were enrolled in an individual N-of-1 trial with 2 blocks. In each block patients crossover between active treatment and placebo
|
|---|---|
|
Percent of Parents Willing to Randomize Responder Infant
|
4 Participants
|
Adverse Events
N-of-1 Trial Series
Serious events: 1 serious events
Other events: 18 other events
Deaths: 0 deaths
Furosemide Periods
Serious events: 1 serious events
Other events: 14 other events
Deaths: 0 deaths
Placebo Periods
Serious events: 0 serious events
Other events: 12 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
N-of-1 Trial Series
n=19 participants at risk
Count of Adverse Events per Patient Occurring During All Treatment Periods. All patients were enrolled in an individual N-of-1 trial with 2 blocks. In each block patients crossover between active treatment (Furosemide + Potassium Chloride) and placebo. Participants were analyzed as a single arm (a set of N-of-1 trials), with results reported accordingly.
|
Furosemide Periods
n=18 participants at risk
Count of Adverse Events per Patient Occurring During Treatment with Furosemide. All patients were enrolled in an individual N-of-1 trial with 2 blocks. In each block patients crossover between active treatment (Furosemide + Potassium Chloride) and placebo. Participants were analyzed as a single arm (a set of N-of-1 trials), with results reported accordingly.
|
Placebo Periods
n=16 participants at risk
Count of Adverse Events per Patient Occurring During Treatment with Placebo. All patients were enrolled in an individual N-of-1 trial with 2 blocks. In each block patients crossover between active treatment (Furosemide + Potassium Chloride) and placebo. Participants were analyzed as a single arm (a set of N-of-1 trials), with results reported accordingly.
|
|---|---|---|---|
|
Gastrointestinal disorders
Necrotizing Enterocolitis
|
5.3%
1/19 • 23 days
Data on adverse events was collected only from enrolled infants. Adverse event data was not collected from providers and parents completing surveys.
|
5.6%
1/18 • 23 days
Data on adverse events was collected only from enrolled infants. Adverse event data was not collected from providers and parents completing surveys.
|
0.00%
0/16 • 23 days
Data on adverse events was collected only from enrolled infants. Adverse event data was not collected from providers and parents completing surveys.
|
Other adverse events
| Measure |
N-of-1 Trial Series
n=19 participants at risk
Count of Adverse Events per Patient Occurring During All Treatment Periods. All patients were enrolled in an individual N-of-1 trial with 2 blocks. In each block patients crossover between active treatment (Furosemide + Potassium Chloride) and placebo. Participants were analyzed as a single arm (a set of N-of-1 trials), with results reported accordingly.
|
Furosemide Periods
n=18 participants at risk
Count of Adverse Events per Patient Occurring During Treatment with Furosemide. All patients were enrolled in an individual N-of-1 trial with 2 blocks. In each block patients crossover between active treatment (Furosemide + Potassium Chloride) and placebo. Participants were analyzed as a single arm (a set of N-of-1 trials), with results reported accordingly.
|
Placebo Periods
n=16 participants at risk
Count of Adverse Events per Patient Occurring During Treatment with Placebo. All patients were enrolled in an individual N-of-1 trial with 2 blocks. In each block patients crossover between active treatment (Furosemide + Potassium Chloride) and placebo. Participants were analyzed as a single arm (a set of N-of-1 trials), with results reported accordingly.
|
|---|---|---|---|
|
Infections and infestations
Sepsis (presumed)
|
5.3%
1/19 • 23 days
Data on adverse events was collected only from enrolled infants. Adverse event data was not collected from providers and parents completing surveys.
|
5.6%
1/18 • 23 days
Data on adverse events was collected only from enrolled infants. Adverse event data was not collected from providers and parents completing surveys.
|
0.00%
0/16 • 23 days
Data on adverse events was collected only from enrolled infants. Adverse event data was not collected from providers and parents completing surveys.
|
|
Infections and infestations
Tracheitis
|
5.3%
1/19 • 23 days
Data on adverse events was collected only from enrolled infants. Adverse event data was not collected from providers and parents completing surveys.
|
0.00%
0/18 • 23 days
Data on adverse events was collected only from enrolled infants. Adverse event data was not collected from providers and parents completing surveys.
|
6.2%
1/16 • 23 days
Data on adverse events was collected only from enrolled infants. Adverse event data was not collected from providers and parents completing surveys.
|
|
Infections and infestations
Urinary Tract Infection
|
21.1%
4/19 • 23 days
Data on adverse events was collected only from enrolled infants. Adverse event data was not collected from providers and parents completing surveys.
|
11.1%
2/18 • 23 days
Data on adverse events was collected only from enrolled infants. Adverse event data was not collected from providers and parents completing surveys.
|
12.5%
2/16 • 23 days
Data on adverse events was collected only from enrolled infants. Adverse event data was not collected from providers and parents completing surveys.
|
|
Metabolism and nutrition disorders
Electrolyte Abnormality
|
63.2%
12/19 • 23 days
Data on adverse events was collected only from enrolled infants. Adverse event data was not collected from providers and parents completing surveys.
|
50.0%
9/18 • 23 days
Data on adverse events was collected only from enrolled infants. Adverse event data was not collected from providers and parents completing surveys.
|
37.5%
6/16 • 23 days
Data on adverse events was collected only from enrolled infants. Adverse event data was not collected from providers and parents completing surveys.
|
|
General disorders
Other
|
26.3%
5/19 • 23 days
Data on adverse events was collected only from enrolled infants. Adverse event data was not collected from providers and parents completing surveys.
|
22.2%
4/18 • 23 days
Data on adverse events was collected only from enrolled infants. Adverse event data was not collected from providers and parents completing surveys.
|
18.8%
3/16 • 23 days
Data on adverse events was collected only from enrolled infants. Adverse event data was not collected from providers and parents completing surveys.
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
5.3%
1/19 • 23 days
Data on adverse events was collected only from enrolled infants. Adverse event data was not collected from providers and parents completing surveys.
|
0.00%
0/18 • 23 days
Data on adverse events was collected only from enrolled infants. Adverse event data was not collected from providers and parents completing surveys.
|
6.2%
1/16 • 23 days
Data on adverse events was collected only from enrolled infants. Adverse event data was not collected from providers and parents completing surveys.
|
|
Infections and infestations
Pneumonia
|
5.3%
1/19 • 23 days
Data on adverse events was collected only from enrolled infants. Adverse event data was not collected from providers and parents completing surveys.
|
5.6%
1/18 • 23 days
Data on adverse events was collected only from enrolled infants. Adverse event data was not collected from providers and parents completing surveys.
|
0.00%
0/16 • 23 days
Data on adverse events was collected only from enrolled infants. Adverse event data was not collected from providers and parents completing surveys.
|
|
Metabolism and nutrition disorders
Dehydration
|
5.3%
1/19 • 23 days
Data on adverse events was collected only from enrolled infants. Adverse event data was not collected from providers and parents completing surveys.
|
0.00%
0/18 • 23 days
Data on adverse events was collected only from enrolled infants. Adverse event data was not collected from providers and parents completing surveys.
|
6.2%
1/16 • 23 days
Data on adverse events was collected only from enrolled infants. Adverse event data was not collected from providers and parents completing surveys.
|
|
Infections and infestations
Sepsis (culture positive)
|
5.3%
1/19 • 23 days
Data on adverse events was collected only from enrolled infants. Adverse event data was not collected from providers and parents completing surveys.
|
0.00%
0/18 • 23 days
Data on adverse events was collected only from enrolled infants. Adverse event data was not collected from providers and parents completing surveys.
|
6.2%
1/16 • 23 days
Data on adverse events was collected only from enrolled infants. Adverse event data was not collected from providers and parents completing surveys.
|
Additional Information
Heather Kaplan, MD, MSCE
Cincinnati Children's Hospital Medical Center
Phone: 5138030478
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place