Phase 2/3 Study of MK-5475 in Adults with Pulmonary Arterial Hypertension
Research type
Research Study
Full title
A Phase 2/3, Multicenter, Randomized, Double-blind, Placebo-Controlled, Adaptive Design Study to Evaluate the Efficacy and Safety of MK-5475 in Adults with Pulmonary Arterial Hypertension
IRAS ID
1003596
Contact name
Dragos Vladimir Budinschi Agachi
Contact email
Sponsor organisation
Merck Sharp & Dohme Corp., a subsidiary of Merck &Co.,Inc
Eudract number
2020-001108-40
Clinicaltrials.gov Identifier
Research summary
This is a multicentre, randomised, placebo-controlled, parallel-group, double-blind Phase 2/3 study to evaluate the efficacy, safety, and tolerability of inhaled MK-5475 in participants with Pulmonary Arterial Hypertension (PAH) sponsored by Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc. (“MSD”), of Kenilworth, New Jersey, USA.
PAH defines a group of rare, progressive conditions characterised by increased vascular resistance in the pulmonary arterial circulation. Despite substantial progress in PAH management, increased awareness, and multiple PAH-specific therapies, the long-term prognosis for patients remains poor and the disease can progress to right-sided heart failure and death.
This study will be carried out in 2 parts: A Phase 2 dose selection cohort and a Phase 3 confirmatory cohort (12-week Base Period and Extension Period).
Phase 2:
Participants will take part in the Phase 2 dose selection cohort for approximately 18 weeks - Screening period of up to 4 weeks, a 12-week double-blind treatment period, and a 2-week Follow-up.
Approximately 164 male and female participants, aged 18 to 75 years with PAH will be enrolled and randomised in a 1:1:1:1 ratio, to one of the four treatment groups: MK-5475 380 micrograms (µg), MK-5475 100 µg, MK-5475 32 µg or placebo which will be orally inhaled once daily for 12 weeks.
Of the 3 MK-5475 doses studied in Phase 2, the dose with the best efficacy and safety profile in this cohort will be evaluated in the subsequent Phase 3 confirmatory cohort.Phase 3:
The Phase 3 cohort consists of a Screening period of up to 4 weeks, Base period consisting of a 12-week double-blind treatment period, Extension period where participants will continue the double-blind treatment period up to 5 years, and a 2-week Follow-up.
Approximately 286 male and female participants, aged 18 to 75 years with PAH will be randomised in a 1:1 ratio, to MK-5475 (Dose selected at the end of the Phase 2 Cohort) or placebo.REC name
North West - Greater Manchester Central Research Ethics Committee
REC reference
21/NW/0055
Date of REC Opinion
19 Apr 2021
REC opinion
Further Information Favourable Opinion