Study to Evaluate Safety and Efficacy of Mirivadelgat in PH-ILD
brief summary
The goal of this clinical trial is to see if mirivadelgat will work in patients with Pulmonary Hypertension Associated with Interstitial Lung Disease (PH-ILD). It will also learn about the safety of mirivadelgat. The main question it aims to answer is if mirivadelgat will improve pulmonary vascular resistance (PVR). Pulmonary vascular resistance is a way to measure blood flow in the lungs. Researchers will compare mirivadelgat to a placebo (a look-alike capsule that contains no drug) to see if mirivadelgat works to improve the symptoms of PH-ILD. The symptoms of PH-ILD that are being looked at are exercise tolerance, heart function, and general well-being. Participants will: Take mirivadelgat or a placebo once a day for 12 weeks Visit the clinic once every 4 weeks for checkups and tests Receive phone calls every one or two weeks to check on how things are going
detailed description
The study is a phase 2, multinational, double-blind, 3-arm study to evaluate the safety and efficacy of mirivadelgat, an aldehyde dehydrogenase 2 activator, in adult subjects (aged 18 to 85 years) with PH-ILD. Subjects must have a confirmed diagnosis of ILD as defined by the American Thoracic Society (ATS), European Respiratory Society (ERS), Japanese Respiratory Society (JRS), and/or Latin American Thoracic Society (ALAT) guidelines (Raghu, 2018). The diagnosis is based on a HRCT either performed at screening or within 180 days prior to screening or a historical surgical biopsy (or other appropriate tissue sampling (e.g., cryobiopsy)) and an RHC performed at screening. Subjects who also have connective tissue disorders can comprise up to 20% of the study population.
To be eligible for the study, a subject must be willing to undergo a Right Heart Catheterization (RHC) during screening and at the Week 12 Visit (at the end of study treatment). Subjects on chronic treatment for underlying pulmonary diseases must be on a stable/optimized dose for ≥30 days prior to screening and have been receiving treatment for ≥90 days prior to screening.
The study will enroll approximately 126 subjects, assuming a drop-out rate of 20%, to obtain 99 evaluable subjects (33 evaluable subjects in each cohort).
Study visits will include a Screening Visit; Visit 2 (Study Day 1); Weeks 2, 3, 4, 6, 8,10, and 12 Visits (+/ 3 days); and a safety Follow-up Visit (+/ 3 days) after the Week 12 Visit. Visits on Weeks 2, 3, 6, and 10 will be conducted by phone calls.
official title
A Multinational Phase 2, Randomized, Double-Blinded, Placebo-Controlled, Multiple-Dose Study to Evaluate the Safety and Efficacy of Mirivadelgat, an Aldehyde Dehydrogenase 2 Activator, in Patients With Pulmonary Hypertension Associated With Interstitial Lung Disease (PH-ILD)