Phase 2
N=125
Safety of Sildenafil in Premature Infants With Severe Bronchopulmonary Dysplasia
Bronchopulmonary Dysplasia of Newborn
Bottom Line
View on ClinicalTrials.gov: NCT04447989 ↗Enrolled (actual)
125
Serious AEs
9.0%
Results posted
Jan 2026
Primary outcome: Primary: Safety Based Upon Number of Participants With Hypotension — 1; 0; 0; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Sildenafil (Drug); Placebo (Drug)
- Age
- Pediatric
- Sex
- All
- Sponsor
- Christoph Hornik
- Primary completion
- Dec 2024
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Safety Based Upon Number of Participants With Hypotension |
1; 0; 0; 0 | — |
| SECONDARY Central Volume of Distribution (Vc) Population Pharmacokinetics (popPK) |
3.86 | — |
| SECONDARY Peripheral Volume of Distribution (Vp) Population Pharmacokinetics (popPK) |
4.49 | — |
| SECONDARY Clearance Population Pharmacokinetics (popPK) |
2.69 | — |
| SECONDARY Half-life Population Pharmacokinetics (popPK) |
8.79 | — |
| SECONDARY Peak Plasma Concentration Population Pharmacokinetics (popPK) |
130.5 | — |
Summary
This is a multicenter, randomized, placebo-controlled, sequential dose-escalating, double-masked, safety study of sildenafil in premature infants (inpatient in Neonatal Intensive Care Units (NICUs)) with severe bronchopulmonary dysplasia (BPD).
Eligibility Criteria
Inclusion Criteria
- Documented informed consent from parent or guardian, prior to study procedures
- 2 liters per minute (LPM)
- Nasal continuous positive airway pressure (NCPAP)
- Nasal intermittent positive pressure ventilation (NIPPV)
- Noninvasive neurally adjusted ventilatory assist (NAVA)
- Any other device designed to provide positive pressure through a nasal device (e.g., RAM cannula, etc.)
Exclusion Criteria
- Previous enrollment and dosing in this study, protocol number (NHLBI-2019-SIL), "Safety of Sildenafil in Premature Infants with Severe Bronchopulmonary Dysplasia (BPD)"
- Previous exposure to sildenafil within 7 days prior to randomization*
- Previous exposure to vasopressors within 24 hours prior to randomization*
- Previous exposure to inhaled nitric oxide within 24 hours prior to randomization*
- Previous exposure to milrinone within 24 hours prior to randomization*
- Evidence of pulmonary hypertension or moderate/large patent ductus arteriosus (PDA) on the most recent echocardiogram performed within 14 days prior to randomization
- Known major congenital heart defect requiring medical or surgical intervention in the neonatal period
- Known allergy to sildenafil
- Known sickle cell disease
- Aspartate aminotransferase (AST) > 225 U/L 150 U/L < 72 hours prior to randomization
- Any condition that would make the participant, in the opinion of the investigator, unsuitable for the study.
- Participant will be reassessed prior to dosing to reconfirm eligibility criteria.
Data sourced from ClinicalTrials.gov (NCT04447989). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.