to assess the impact of oral sildenafil administration on echocardiography measurements of pulmonary vascular resistance in neonates with Down Syndrome without Congenital Heart Disease. MedDRA version: 20.1 Level: PT Classification code 10067285 Term: Vascular resistance pulmonary increased System Organ Class: 10022891 - Investigations
Conditions
Interventions
Trade Name: Revatio 10 mg/ml powder for oral suspension
Pharmaceutical Form: Powder for oral suspension
INN or Proposed INN: SILDENAFIL CITRATE
Other descriptive name: SILDENAFIL CITRATE
Concentration
Sponsors
Royal College of Surgeons in Ireland
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Antenatal or postnatal diagnosis of Down Syndrome in babies born from a singleton pregnancy at =35 weeks gestation. 2. Babies with a diagnosis of pulmonary hypertension, defined as two or more of the following echocardiography markers being present: (1) A PAAT 1.8. 3. Must live within 60 minutes by car or 50km of hospital Are the trial subjects under 18? yes Number of subjects for this age range: 40 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Lack of parental consent 2. Congenital heart disease other than a Patent Ductus Arteriosus 3. Other major congenital anomaly (such as gastrointestinal issues such as duodenal atresia, or renal or liver issues). 4. The presence of heart block or pre-excitation on a rhythm strip 5. Hypotension 6. Abnormal liver function tests at screening 7. Treatment with any of the following drugs: nitric oxide donors, nitrates, guanyl cyclase inhibitors (e.g. riociguat), potent CYP 3A4 inhibitors (e.g. ketoconazole, itraconazole, ritonavir), alpha-blockers, vitamin K antagonists or bosentan. 8. History of a persistent bleeding disorder 9. Close family history of hereditary degenerative retinal disorders (such as retinitis pigmentosa) or sickle cell disease 10. Participation in another clinical trial of an investigational medicinal product within 30 days 11. Presence of any other illness or condition that renders the patient unsuitable for trial participation in the opinion of the investigator 12. Allergy or hypersensitivity to any of the components of Sildenafil 13. Any reason in the opinion of the investigator that the parent(s) would be unable to administer the investigational medicinal product to the subject
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective is to assess the impact of oral sildenafil administration on echocardiography measurements of pulmonary vascular resistance in neonates with Down Syndrome without Congenital Heart Disease.;Secondary Objective: The secondary objectives are to assess the safety of sildenafil administration in this population.;Primary end point(s): The primary outcome to be assessed is measurement of pulmonary vascular resistance using surrogate echocardiography markers performed at 2 months of age to include: •Pulmonary artery acceleration time indexed to heart rate •Left ventricular eccentricity index •Tricuspid regurgitant jet velocity (if present) ;Timepoint(s) of evaluation of this end point: 2 months of age | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary endpoints all assessed at 2 months of age: • Full blood count including platelet counts at 3 months of age • Full renal profile including urea and creatinine • Full liver profile • Incidence of hypotension • Incidence of arrhythmias ;Timepoint(s) of evaluation of this end point: 2 months of age | — |
Countries
Ireland
Contacts
Public Contactmandy jackson
Royal College of Surgeons in Ireland
Outcome results
None listed