Phase 2 study to investigate the PK of selexipag in children with PAH
Research type
Research Study
Full title
A prospective, multicenter, open-label, single-arm, Phase 2 study to investigate the safety, tolerability and pharmacokinetics of selexipag in children with pulmonary arterial hypertension.
IRAS ID
247481
Contact name
Shahin Moledina
Contact email
Sponsor organisation
Actelion Pharmaceuticals Ltd
Eudract number
2018-000145-39
Clinicaltrials.gov Identifier
Duration of Study in the UK
7 years, 0 months, 1 days
Research summary
Pulmonary arterial hypertension (PAH) is a rare and severe disease that causes narrowing of the pulmonary artery. Blood is pumped from the heart through this narrowed artery to the lungs, where it gets a fresh supply of oxygen. Because the artery is narrow in PAH it requires more effort from the heart to pump blood against the higher pressure. This can cause damage to vital organs like the heart and lungs.
There are treatments and medications approved for PAH in adults, but very few of them have been studied in children with the condition.The purpose of this study is to find the right dose of selexipag (the study medication) to be administered to children and teenagers suffering from PAH who are between 2 to and 18 years old, and to see whether the drug dose is appropriate and safe.
The study is conducted in three phases, consisting of a screening period of (up to) 6 weeks, a treatment period (including a period where medication doses are gradually increased, a maintenance period of study treatment and an end of trial visit) and a follow-up period of 30 days. Participants will be enrolled into 1 of 3 age groups: 12 years to 18 years of age, 6 years to 12 years of age and 2 years to 6 years of age. Participants may be in the study for a maximum duration of 7 years.
REC name
London - Hampstead Research Ethics Committee
REC reference
18/LO/1479
Date of REC Opinion
22 Oct 2018
REC opinion
Further Information Favourable Opinion