A SINGLE ARM SINGLE CENTRE STUDY TO INVESTIGATE SAFETY AND EFFICACY OF SILDENAFIL IN NEAR TERM AND TERM NEWBORNS WITH PERSISTENT PULMONARY HYPERTENSION OF THE NEWBORN (PPHN)

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2009-016248-37-GB

Enrollment

Registered

2010-03-17

Start date

2010-07-15

Completion date

Unknown

Last updated

2019-12-10

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Persistent pulmonary hypertension of the newborn (PPHN) MedDRA version: 12.1 Level: LLT Classification code 10053592 Term: Newborn persistent pulmonary hypertension

Interventions

Trade Name: Revatio Product Code: UK-92,480-10 Pharmaceutical Form: Solution for infusion INN or Proposed INN: Sildenafil citrate CAS Number: 171599-83-0 Current Sponsor code: UK-92,480-10 Concentrati

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Patients must meet all of the following inclusion criteria to be eligible for enrollment into the study: 1. Evidence of signed and dated informed consent document indicating that a legally acceptable guardian has been informed of all pertinent aspects of the study. 2. =72 hours of age; and =34 weeks gestational age at screening. 3. PPHN or Hypoxic respiratory failure associated with: a. Idiopathic PPHN or b. Meconium aspiration syndrome or c. Sepsis or d. Pneumonia 4. Oxygenation Index (OI) >15 and =65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: Patients presenting with any of the following must not be included in the study: 1. Patients already receiving inhaled nitric oxide (iNO) on referral. 2. Prior or immediate need for full Cardio Pulmonary Resuscitation or Extracorporeal Membrane Oxygenation (ECMO). 3. Life threatening or lethal congenital anomaly. 4. Large left to right intracardiac or ductal shunting (diagnosed from echocardiogram on admission to GOSH). 5. Clinically significant active seizures as per clinical judgment. 6. Bleeding diathesis as per clinical judgment. 7. Participation in any other experimental studies before the current study begins or during the study participation. 8. Receipt of any prohibited concurrent medication/therapy at any time prior to screening: a. Potent cytochrome P450 3A4 inhibitors (eg, erythromycin, ketoconazole, itraconazole, and protease inhibitors). b. ritonavir or nicorandil. c. Endothelin antagonists (eg, Tracleer/bosentan). d. Nitrates or nitric oxide donors. A patient is eligible if nitroprusside was used only if it was discontinued at least 2 hours prior to study drug infusion; iNO may be used per protocol. e. Vasodilators (eg, alpha blockers, magnesium sulfate, calcium channel blockers, other PDE inhibitors, prostacyclins, etc). This excludes milrinone which is allowed during the study as concurrent therapy. f. Supplemental arginine administered for the purpose of improving NO-dependent vasodilation. Maintenance quantities in total parental nutrition (TPN) are allowed. 9. Known hereditary degenerative retinal disorders such as retinitis pigmentosa. 10. Symptoms of drug or alcohol related withdrawal. 11. In the opinion of the investigator, a patient who would be considered inappropriate for the study, for any reason: Other acute severe or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the patient inappropriate for entry into this study.

Design outcomes

Primary

Measure	Time frame
Main Objective: Efficacy: determine the efficacy of iv sildenafil in near term and term newborns with PPHN or with hypoxic respiratory failure and at risk for PPHN. The primary measure of efficacy will be the reduced need for iNO and extracorporeal membrane oxygenation ECMO). Safety: to assess the safety and tolerability of iv sildenafil in near term and term newborns with PPHN or with hypoxic respiratory failure and at risk of developing PPHN.;Secondary Objective: Efficacy: To assess the efficacy of iv sildenafil as measured by: oxygenation index; time to receipt of standard therapy (iNO or ECMO), days on mechanical ventilation, differential saturation; P/F ratio (PaO2/FiO2). Pharmacokinetics: To further characterize the PK of sildenafil in newborns that have PPHN or hypoxic respiratory failure and at risk of developing PPHN.;Primary end point(s): Efficacy - incidence of treatment failure: Need for an inhaled nitric oxide (iNO) or ECMO. Safety: the following data will be used to assess the safety of iv sildenafil in this patient population: Incidence and severity of adverse events. Laboratory parameters.	—

Measure

Time frame

Main Objective: Efficacy: determine the efficacy of iv sildenafil in near term and term newborns with PPHN or with hypoxic respiratory failure and at risk for PPHN. The primary measure of efficacy will be the reduced need for iNO and extracorporeal membrane oxygenation ECMO). Safety: to assess the safety and tolerability of iv sildenafil in near term and term newborns with PPHN or with hypoxic respiratory failure and at risk of developing PPHN.;Secondary Objective: Efficacy: To assess the efficacy of iv sildenafil as measured by: oxygenation index; time to receipt of standard therapy (iNO or ECMO), days on mechanical ventilation, differential saturation; P/F ratio (PaO2/FiO2). Pharmacokinetics: To further characterize the PK of sildenafil in newborns that have PPHN or hypoxic respiratory failure and at risk of developing PPHN.;Primary end point(s): Efficacy - incidence of treatment failure: Need for an inhaled nitric oxide (iNO) or ECMO. Safety: the following data will be used to assess the safety of iv sildenafil in this patient population: Incidence and severity of adverse events. Laboratory parameters.

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Countries

United Kingdom

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026