A Phase II, Randomized, Double-Blind, Safety and Efficacy Study of Tiprelestat Versus Placebo When Added to Standard of Care for the Treatment of Pulmonary Arterial Hypertension (PAH)

Stanford University

Summary

The primary objective of this study is to compare the efficacy, safety, and tolerability of tiprelestat plus Standard of Care (SOC) compared with placebo plus SOC in patients with World Health Organization (WHO) functional class II-IV pulmonary arterial hypertension (PAH).

Eligibility

Age range: 18–75 years
Sex: All
Healthy volunteers: No

Detailed criteria

Inclusion Criteria: 1. Adults age 18 to 75 years. 2. Willingness to give written informed consent prior to any study-related procedures being performed and to be able to adhere to the study restrictions and examination schedule. 3. Diagnosis of WHO Group I PAH. 4. WHO functional class II - IV despite optimized treatment SOC, with 1 or more modalities including phosphodiesterase 5 (PDE5) inhibitor, soluble guanylate cyclase stimulator (sGCS), endothelin receptor antagonist (ERA), and/or a prostacyclin analogue or receptor agonist (SC/inhaled/PO) (see #5), as well as Sotatercept (see #6). 5. On…

Interventions

Drug
Tiprelestat (5 mg)
5 mg of tiprelestat in 1 mL saline administered as a daily subcutaneous injection for 168 days.
Drug
Placebo (1 mL 0.9% saline solution)
Matching 1 mL 0.9% saline solution administered as a daily subcutaneous injection for 168 days.
Drug
Tiprelestat (10 mg)
10 mg of tiprelestat in 1 mL saline administered as a daily subcutaneous injection for 168 days.

Locations (10)

The University of Arizona / Banner University Medical Center
Tucson, Arizona
University of California, San Francisco
San Francisco, California
Stanford University
Stanford, California
University of Colorado (UCD) Anschutz
Aurora, Colorado
Harvard University / Brigham and Women's Hospital
Boston, Massachusetts
Duke University
Durham, North Carolina