A Study of AV-101 (Dry Powder Inhaled Imatinib) in Patients With Pulmonary Arterial Hypertension (PAH)

Phase 3TerminatedNCT05036135

Aerovate Therapeutics202 enrolled

Overview

IMPAHCT: Inhaled iMatinib Pulmonary Arterial Hypertension Clinical Trial is a Phase 2b/Phase 3 study to evaluate the safety and efficacy of AV-101 (dry powder inhaled imatinib) in patients with Pulmonary Arterial Hypertension (PAH). The Phase 2b part of the study will assess three doses to establish an optimal dose for the Phase 3 part of the study. The Phase 2b primary endpoint will be the placebo corrected change in pulmonary vascular resistance (PVR). The Phase 3 primary endpoint will be the placebo corrected change in 6-minute walk distance (6MWD) after 24 weeks of treatment.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

202

Conditions

Pulmonary Arterial Hypertension

Interventions

Eligibility

Sex: ALLMin age: 18 YearsMax age: 75 Years

Medical Language ↔ Plain English

Key Inclusion Criteria * PAH belonging to one of the subgroups: 1. I/HPAH, PAH-CTD, 2. PAH due to drugs and/or toxins/chemicals (having been in the care of the investigator for at least one year with no relapses of drug or toxin/chemical abuse), 3. HIV associated or 4. PAH due to repaired congenital heart disease (at least 1 year since repair) * World Health Organization (WHO) Functional Class II, III or IV symptoms * Stable concomitant background therapy of at least one PAH approved medications * Able to walk a distance of at least 100 m but no more than 475 m during the Screening 6-minute walk tests. Key Exclusion Criteria * Pulmonary hypertension (PH) belonging to Groups 2 to 5 * A history of left-sided heart disease * Pregnant or breast-feeding females Additional criteria may apply, per protocol

Locations (128)

Arizona Pulmonary Specialists, Ltd.

Phoenix, Arizona, United States

University of Arizona, Department of Medicine

Tucson, Arizona, United States

UCLA/David Geffen School of Medicine-Pulmonary and Criticial Care

Los Angeles, California, United States

Kaiser Permanente Los Angeles Medical Center

Los Angeles, California, United States

Cedars-Sinai Medical Center, Pulmonary & Critical Care

Los Angeles, California, United States

Outcomes

Primary Outcomes

Phase 2b: Change from Baseline in Pulmonary Vascular Resistance (PVR)

Time frame: 24 weeks

Phase 3: Change from Baseline in Six Minute Walk Distance (6MWD)

Time frame: 24 weeks

Secondary Outcomes

Phase 2b: Change from Baseline in N-terminal pro B-type natriuretic peptide (NT-proBNP)

Time frame: 24 weeks

Phase 2b: Change from Baseline in Six Minute Walk Distance (6MWD)

Time frame: 24 weeks

Phase 2b: Time to Clinical Worsening

Time frame: 24 weeks

Phase 2b: Proportion of Subjects with Improvement in WHO Functional Class

Time frame: 24 weeks

Phase 2b: Change from Baseline in Registry to Evaluate Early and Long-Term PAH Disease Management (REVEAL) Lite 2.0 Risk Score

REVEAL Lite 2.0 risk scores range from 1 to 14; a higher score represents higher risk

Time frame: 24 weeks

Phase 2b: Change from Baseline in Health-Related Quality of Life emPHasis-10 Questionnaire Score

emPHasis-10 questionnaire scores range from 0 - 50; a higher score represents a higher symptom burden

Time frame: 24 weeks

Phase 3: Change from Baseline in NT-proBNP

Time frame: 24 weeks

Phase 3: Time to Clinical Worsening

Time frame: 24 weeks

Phase 3: Proportion of Subjects with Improvement in WHO Functional Class

Time frame: 24 weeks

Phase 3: Change from Baseline in Registry to Evaluate Early and Long-Term PAH Disease Management (REVEAL) Lite 2.0 Risk Score

REVEAL Lite 2.0 risk scores range from 1 to 14; a higher score represents higher risk

Time frame: 24 weeks

Phase 3: Change from Baseline in Pulmonary Arterial Hypertension-Symptoms and Impact (PAH-SYMPACT) Questionnaire Score

PAH-SYMPACT is a patient reported outcome instrument developed to assess PAH symptoms and impacts

Time frame: 24 weeks