Safety & efficacy of sildenafil in newborns with PPHN Version 1

• Research type

  Research Study

• Full title

  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)

• IRAS ID

  47613

• Contact name

  Christine Pierce

• Eudract number

  2009-016248-37

• ISRCTN Number

  Not Sent

• Research summary

  Persistent pulmonary hypertension of the newborn (PPHN) may be precipitated by meconium aspiration, sepsis or hypoxia, and is usually diagnosed as hypoxic respiratory failure that is not responsive to 100% oxygen. Currently the main treatment for PPHN is inhaled nitric oxide (iNO). Extracorporeal membrane oxygenation (ECMO) is a supportive therapy also used in PPHN unresponsive to iNO. These treatments are difficult to administer and only available at specialist units, and are expensive. Sildenafil is a prescription drug licensed for use in adults with pulmonary arterial hypertension, however, there is increasing evidence that sildenafil may be useful in the treatment of PPHN. Sildenafil treatment for PPHN would have the potential benefits, over existing treatments, of being simple to administer, cost effective and potentially available in all hospitals. This study will investigate whether sildenafil is a safe and effective treatment for PPHN. Newborn babies diagnosed with PPHN and being transferred to the neonatal intensive care unit at Great Ormond Street hospital (GOSH) will be able to take part in this study. All patients will receive intravenous sildenafil treatment for up to a maximum of 14 days. There is no placebo in this study. Study data will be compared to historical controls from the GOSH database. Patients will receive all the normal care to treat PPHN and standard treatments for PPHN (iNO and ECMO) will also be available and administered if required. Study data will be collected from the procedures conducted during the normal care of the patients the only additional procedures will be blood samples which will be used to measure the amount of sildenafil in the blood.

• REC name

  London - Bloomsbury Research Ethics Committee

• REC reference

  10/H0713/36

• Date of REC Opinion

  15 Jul 2010

• REC opinion

  Further Information Favourable Opinion