Inhaled Imatinib Pulmonary Arterial Hypertension Clinical Trial (IMPAHCT) or A Phase 2b/3, Randomized, Controlled, 24-week Dose Ranging and Confirmatory Study of AV-101 in Patients with PAH.
- Conditions
- Pulmonary Arterial Hypertension (PAH)MedDRA version: 21.1Level: PTClassification code 10064911Term: Pulmonary arterial hypertensionSystem Organ Class: 10038738 - Respiratory, thoracic and mediastinal disordersMedDRA version: 20.0Level: LLTClassification code 10077729Term: Pulmonary arterial hypertension WHO functional class IIISystem Organ Class: 10038738 - Respiratory, thoracic and mediastinal disordersMedDRA version: 20.0Level: LLTClassification code 10077730Term: Pulmonary arterial hypertension WHO functional class IVSystem Organ Class: 10038738 - Respiratory, thoracic and mediastinal disordersMedDRA version: 20.0Level: LLTClassification code 10077740Term: Pulmonary arterial hypertension WHO functional class IISystem Organ Class: 10038738 - Respiratory, thoracic and mediastinal disordersTherapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
- Registration Number
- EUCTR2021-001910-13-IE
- Lead Sponsor
- Aerovate Therapeutics, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 462
Key Inclusion Criteria:
1. Male or female adults (between 18 and 75 years) at the Screening
Visit within 28 days prior to Day 1
2. Subjects with a diagnosis of PAH belong to one of the subgroups of the NICE classification of Group 1:
- a. I/HPAH, PAH-CTD
- b. PAH due to drugs and toxins (having been in the care of the Investigator for at least one year with no relapses of drug or toxin/chemical abuse),
- c. HIV associated or
- d. PAH due to repaired congenital heart disease (at least 1 year since repair)
*Excluding Patients with Portopulmonary Hypertension
3. World Health Organization (WHO) Functional Class II, III or IV
symptoms
4. Must be able to walk a distance of at least 100 m but no more than 475 m during the screening 6-minute walk test.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 323
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 139
Key Exclusion Criteria:
1. PH belonging to Groups 2 to 5 of the 2018 NICE classification and Group 1 diagnosed with Portopulmonary Hypertension
2. A history of LVEF = 40% on echocardiogram within 12 months of screening, or clinically significant ischemic, mitral or aortic valve disease, or constrictive heart disease in the opinion of the Investigator
3. Pregnant or breast-feeding females
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: The objective of the study is to establish an optimal dose of AV-101 based primarily upon the change in PVR (assessed by right heart catherization) and the safety and tolerability of AV-101 as evaluated in the Phase 2b Part of the study. The optimal dose will be taken into the Phase 3 Part of the study where the placebo-corrected change in 6MWD after 24 weeks of treatment will be used as the primary endpoint.;Secondary Objective: Change from baseline in NT-proBNP levels, Time to Clinical Worsening's, Multicomponent Clinical Improvements, Maintenance or Improvement of Functional Class, Change in REVEAL Lite 2.0 score and Quality of Life will be evaluated.;Primary end point(s): Primary Endpoint (Phase 2b)<br>? The placebo-corrected change from baseline in PVR at 24 weeks<br><br>Primary Endpoint (Phase 3)<br>? The placebo-corrected change in 6MWD at Week-24;Timepoint(s) of evaluation of this end point: 24 weeks
- Secondary Outcome Measures
Name Time Method Secondary end point(s): Key Secondary Endpoints for Phase 2B: <br>? Change from baseline at Week 24 in NT-proBNP<br>? Change from baseline at Week 24 in 6MWD<br>? Incidence of Clinical Worsening through 24 Weeks<br>? Improvement at Week 24 in WHO Function Class status<br>? Change from baseline at Week 24 in REVEAL Lite 2.0 Score<br>? Change from baseline at Week 24 in QoL (emPHasis-10) Questionnaire score<br><br>Key Secondary Endpoints for Phase 3:<br>? Change from baseline at Week 24 in NT-proBNP<br>? Time to Clinical Worsening through 24 weeks<br>? Improvement at Week 24 in WHO Function Class status<br>? Change from baseline at Week 24 in REVEAL Lite 2.0 Score<br>? Change from baseline at Week 24 in QoL (PAH-SYMPACT) Questionnaire score;Timepoint(s) of evaluation of this end point: Phase 2b and Phase 3 endpoints will be evaluated at Week 24 except for safety and PK. Safety will be evaluated throughout the study and PK will be evaluated at Week 4 and Week 24.