RUBATO
Research type
Research Study
Full title
Prospective, multi-center, double-blind, randomized, placebo controlled, parallel group study assessing the efficacy and safety of macitentan in Fontan palliated adult and adolescent subjects
IRAS ID
222282
Contact name
Paul Clift
Contact email
Sponsor organisation
Actelion Pharmaceuticals Ltd
Eudract number
2016-003320-23
Duration of Study in the UK
2 years, 4 months, 1 days
Research summary
Research Summary
The Fontal procedure was introduced originally to treat patients with tricuspid atresia. This surgical approach has since been applied to a range of complex congenital heart malformations characterised by the presence of only one functional ventricular chamber, in patients where biventricular repair is not possible.
After successfully completed surgeries, Fontan-palliated patients are affected by a number of complications associated with considerable morbidity and mortality. Their exercise capacity (measured by peak oxygen uptake [VO2]) remains limited. Fontan-palliation surgery is mostly done by the age of 5 years, and these people do quite well but only until they reach puberty. This is because once the body starts to grow and change, the heart has to do more work.
The study will include people who are 12 years old and older. The idea is to start treatment before the strain on the heart becomes too great, causing it to begin to fail.
Currently, there are no medicinal products approved for use in the treatment of Fontan-palliated patients.
In total, 134 adults and adolescents will be randomised in a 1:1 ratio to macitentan 10 mg or placebo. Randomisation will be stratified by geographical region (America, Europe, Asia, Oceania). This study will be performed in a doubleblind fashion. The investigator and study personnel, the subjects, the clinical research associates (CRAs), Actelion personnel, and CRO personnel involved in the conduct of the study will remain blinded to the study treatment until study completion.
The study will assess the efficacy, safety, and PK of treating Fontan-palliated subjects with macitentan compared to placebo.
Macitentan 10 mg will be provided to patients as film-coated tablets debossed with ‘10’ on one side, for o.d. oral administration with or without food.
Patients taking part in the study will be given the study treatment for the first time at site, during the Randomisation Visit (week 2), after successful completion of all Screening (week 1) and Randomisation (Visit 2) assessments.
The study will last approximately 28 months from first patient, first visit to last patient, last visit.Summary of Results
This study is not in scope for a plain language summary, therefore this is not applicable.REC name
West Midlands - Edgbaston Research Ethics Committee
REC reference
17/WM/0301
Date of REC Opinion
6 Oct 2017
REC opinion
Further Information Favourable Opinion