Sildenafil use in Infants with Down Syndrome During the Neonatal Period

Phase 1

• Conditions: 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: 20.1Level: PTClassification code 10067285Term: Vascular resistance pulmonary increasedSystem Organ Class: 10022891 - Investigations; Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]

• Registration Number: EUCTR2021-004600-17-IE
• Lead Sponsor: Royal College of Surgeons in Ireland

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-08-31
• Last Update Posted: 13 November 2023

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 40

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 < 40ms, (2) a PAAT:RVET < 0.25, (3) in the presence of a patent ductus arteriosus (PDA), the demonstration of bidirectional flow across the vessel or right to left flow; or (4) A left ventricular eccentricity index > 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

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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified