RUBATO OL
Research type
Research Study
Full title
Prospective, multi-center, single-arm, open-label long-term study assessing the safety, tolerability, and effectiveness of macitentan in Fontan-palliated adult and adolescent subjects.
IRAS ID
265937
Contact name
Paul Clift
Contact email
Sponsor organisation
Actelion Pharmaceuticals Ltd
Eudract number
2018-002821-45
Duration of Study in the UK
2 years, 4 months, 0 days
Research summary
Fontan circulation (also called Fontan-palliation or Fontan-palliated heart) is a surgery procedure performed in children born with certain heart abnormalities. These babies have a single ventricular chamber to pump blood throughout the entire body instead of two chambers. Fontan-palliation improves the survival of these children but complications are frequent.
The purposes of this study is to find out more about the long-term safety and effectiveness of macitentan (the study medication) in patients with Fontan-palliation. Macitentan is currently available for people with pulmonary arterial hypertension (PAH - a serious heart and lung condition that has similar characteristics to Fontan-palliation).
This study will follow-on from a currently active study, RUBATO-DB (AC-055H301). Participants in RUBATO-DB will be invited to 'roll-over' in to this study and continue receiving macitentan (or, for those participants currently receiving a placebo, they will be invited to receive macitentan for the first time). No placebo will be used in this study; all participants will receive the study medication.
This study consists of a treatment period lasting 2 years, and an End-of-Study visit 30 days following completion of treatment. Participants will be aged 12-65.
REC name
West Midlands - Coventry & Warwickshire Research Ethics Committee
REC reference
19/WM/0217
Date of REC Opinion
12 Sep 2019
REC opinion
Further Information Favourable Opinion