A Phase 2, Randomized, Double-Blind, Multi-Dose, Placebo-Controlled Study to Evaluate the Efficacy and Safety of LAM-001 in Adults With Pulmonary Hypertension Associated With Interstitial Lung Disease (PH-ILD)
OrphAI Therapeutics
Summary
A Phase 2, Randomized, Double-Blind, Multi-Dose, Placebo-Controlled Study to Evaluate the Efficacy and Safety of LAM-001 in Adults with Pulmonary Hypertension Associated with Interstitial Lung Disease (PH-ILD).
Description
A Two-Part Phase 2 study to Assess LAM-001 (Inhaled Sirolimus) for the Treatment of Pulmonary Hypertension - Part A was a Single-Arm, Open-Label, Exploratory Study in Group 1 and Group 3 PH Patients (Completed) and Part B is a Phase 2, Randomized, Double-Blind, Multi-Dose, Placebo-Controlled Study to Evaluate the Efficacy and Safety of LAM-001 in Adults with Pulmonary Hypertension Associated with Interstitial Lung Disease (PH-ILD). In Part B, approximately 75 participants will receive standard of care plus LAM- 001 (inhaled sirolimus) 100 mcg, 200 mcg or placebo by oral inhalation once daily…
Eligibility
- Age range
- 18–80 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Age 18-80 years (\>70 y/o requires medical monitor approval) 2. Diagnosis of PH-ILD as defined by CT imaging within 1 year of screening that demonstrates diffuse parenchymal lung disease or abnormal PFTs (see IC #3) associated with one of the following: 1. Idiopathic interstitial pneumonia (IIP) including: * Idiopathic pulmonary fibrosis (IPF) * Idiopathic nonspecific interstitial pneumonia * Respiratory bronchiolitis-associated interstitial lung disease (RB-ILD) * Unclassifiable idiopathic interstitial pneumonia 2. Chronic hypersensitivi…
Interventions
- DrugPlacebo
Matching placebo administered via dry powder inhalation
- DrugLAM-001
LAM-001 administered via dry powder inhaler
Locations (3)
- University of ArizonaTucson, Arizona
- Yale New Haven HospitalNew Haven, Connecticut
- Brigham and Women's HospitalBoston, Massachusetts