Pulmonary Arterial Hypertension
Conditions
Keywords
Pulmonary Arterial Hypertension, Lungs, Pulmonary, PAH, AV-101, imatinib, IMPAHCT
Brief summary
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.
Interventions
DRUGAV-101
AV-101 (imatinib) administered via dry powder inhalation
DRUGPlacebo
Placebo administered via dry powder inhalation
Sponsors
Aerovate Therapeutics
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Phase 2b: Change from Baseline in Pulmonary Vascular Resistance (PVR) | 24 weeks |
| Phase 3: Change from Baseline in Six Minute Walk Distance (6MWD) | 24 weeks |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Phase 2b: Time to Clinical Worsening | 24 weeks | — |
| Phase 2b: Proportion of Subjects with Improvement in WHO Functional Class | 24 weeks | — |
| Phase 2b: Change from Baseline in Registry to Evaluate Early and Long-Term PAH Disease Management (REVEAL) Lite 2.0 Risk Score | 24 weeks | REVEAL Lite 2.0 risk scores range from 1 to 14; a higher score represents higher risk |
| Phase 2b: Change from Baseline in Health-Related Quality of Life emPHasis-10 Questionnaire Score | 24 weeks | emPHasis-10 questionnaire scores range from 0 - 50; a higher score represents a higher symptom burden |
| Phase 2b: Change from Baseline in N-terminal pro B-type natriuretic peptide (NT-proBNP) | 24 weeks | — |
| Phase 3: Time to Clinical Worsening | 24 weeks | — |
| Phase 3: Proportion of Subjects with Improvement in WHO Functional Class | 24 weeks | — |
| Phase 3: Change from Baseline in Registry to Evaluate Early and Long-Term PAH Disease Management (REVEAL) Lite 2.0 Risk Score | 24 weeks | REVEAL Lite 2.0 risk scores range from 1 to 14; a higher score represents higher risk |
| Phase 3: Change from Baseline in Pulmonary Arterial Hypertension-Symptoms and Impact (PAH-SYMPACT) Questionnaire Score | 24 weeks | PAH-SYMPACT is a patient reported outcome instrument developed to assess PAH symptoms and impacts |
| Phase 3: Change from Baseline in NT-proBNP | 24 weeks | — |
| Phase 2b: Change from Baseline in Six Minute Walk Distance (6MWD) | 24 weeks | — |
Countries
Argentina, Australia, Austria, Belgium, Brazil, Canada, Chile, China, Czechia, France, Germany, Greece, Israel, Italy, Latvia, Mexico, Netherlands, Poland, Portugal, Puerto Rico, Singapore, South Africa, Spain, Sweden, United Kingdom, United States
Outcome results
None listed