MACiTEPH
Research type
Research Study
Full title
A prospective, randomized, double-blind, multicenter, placebo-controlled, parallel group, adaptive Phase 3 study with open-label extension to evaluate efficacy and safety of macitentan 75 mg in inoperable or persistent/recurrent chronic thromboembolic pulmonary hypertension.
IRAS ID
279163
Contact name
Joanna Pepke-Zaba
Contact email
Sponsor organisation
ACTELION Pharmacteuticals Ltd.
Eudract number
2019-004131-24
Clinicaltrials.gov Identifier
Duration of Study in the UK
5 years, 5 months, 3 days
Research summary
Chronic thromboembolic pulmonary hypertension (CTEPH) is one of the leading causes of severe pulmonary hypertension (PH). It is a rare, progressive pulmonary vascular disease that if left untreated, leads to progressively increasing pulmonary vascular resistance (PVR) and eventually right ventricle failure and death.
Macitentan 75 mg is expected to provide significant clinical benefit to CTEPH patients, including those treated with background PH-specific therapy, except ERAs and intravenous, subcutaneous, or inhaled prostacyclins/prostacyclin analogs. Macitentan offers a new mode of action for the treatment of inoperable and persistent/recurrent CTEPH and addresses an important unmet medical need for an alternative treatment in this indication.
This study will assess the effect of macitentan 75 mg on exercise capacity and is expected to confirm the role of macitentan as a first-line, as well as add-on treatment for CTEPH.
At least 144 participants will be randomized in a 1:1 ratio to macitentan 75 mg or placebo and the study will comprise of the following periods:
- Screening (up to 60 days)
- Double blind (DB) treatment period (52 weeks) which includes an uptitration and maintenance phase.
- Post-treatment observation phase (up to 52 weeks) for patients who prematurely discontinue DB treatment but do not withdraw consent.
- Open-Label extension period (104 weeks) which starts after the last patient has completed the double blind treatment period.
- Safety follow up period (30 days) which starts on the day of the last dose of study intervention.The primary endpoint will be to evaluate the effect of macitentan 75 mg versus
placebo on exercise capacity at Week 28 in participants.The entire study is expected to last approximately 6 years and will be conducted at NHS specialist PH centres in the United Kingdom.
REC name
South Central - Berkshire Research Ethics Committee
REC reference
20/SC/0388
Date of REC Opinion
23 Nov 2020
REC opinion
Favourable Opinion