Inhaled iMatinib Pulmonary Arterial Hypertension Clinical Trial - Follow Up Long Term Extension (IMPAHCT-FUL)

Sponsor

Aerovate Therapeutics (Industry)

Overall Status

Recruiting

CT.gov ID

NCT05557942

Collaborator

(none)

462

Enrollment

Location

Arms

37.9

Anticipated Duration (Months)

12.2

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

IMPAHCT-FUL: Inhaled iMatinib Pulmonary Arterial Hypertension Clinical Trial - Follow Up Long Term Extension (LTE) Trial is a follow up study to establish the long-term safety of AV-101. The long-term effects of AV-101 on efficacy measures will also be assessed. Subjects who successfully complete the 24-week placebo-controlled parent trial (AV-101-002) will be offered the opportunity to continue into this LTE study. Subjects who enroll in the study will receive one of three active AV-101 doses until such time as the optimal dose has been selected in the parent study.

Condition or Disease	Intervention/Treatment	Phase
Pulmonary Arterial Hypertension	Drug: AV-101	Phase 2/Phase 3

Study Design

Study Type:

Interventional

Anticipated Enrollment :

462 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Primary Purpose:

Treatment

Official Title:

A Long-Term Extension, Multi-Center Safety Study of AV-101 in Subjects With Pulmonary Arterial Hypertension (PAH) Who Have Completed Study AV-101-002 (IMPAHCT-FUL)

Actual Study Start Date :

Nov 2, 2022

Anticipated Primary Completion Date :

Dec 31, 2025

Anticipated Study Completion Date :

Dec 31, 2025

Arms and Interventions

Arm	Intervention/Treatment
Experimental: low dose AV-101	Drug: AV-101 AV-101 (imatinib) administered via dry powder inhalation
Experimental: medium dose AV-101	Drug: AV-101 AV-101 (imatinib) administered via dry powder inhalation
Experimental: high dose AV-101	Drug: AV-101 AV-101 (imatinib) administered via dry powder inhalation

Arm

Intervention/Treatment

Experimental: low dose AV-101

Drug: AV-101

AV-101 (imatinib) administered via dry powder inhalation

Experimental: medium dose AV-101

Drug: AV-101

AV-101 (imatinib) administered via dry powder inhalation

Experimental: high dose AV-101

Drug: AV-101

AV-101 (imatinib) administered via dry powder inhalation

Outcome Measures

Primary Outcome Measures

Safety and tolerability of AV-101 [Through Study completion, anticipated to be approximately 3 years]
as measured by Incidence of Adverse Events

Other Outcome Measures

Change from baseline in the 6MWD [Baseline, weeks 4,12 and 24, and every 12 weeks up to study completion]
Change from baseline in NT-proBNP [Baseline, weeks 4,12 and 24, and every 12 weeks up to study completion]
Change from baseline in Right Ventricular (RV) function (e.g. TAPSE, RVFAC, RV/PA coupling, TRJV etc.) as measured by Resting Transthoracic Echocardiography [Baseline, week 24, and every 12 weeks up to study completion]
Time to Clinical Worsening [Through Study completion, anticipated to be approximately 3 years]
Clinical Worsening Events will be defined as: Death (all causes) Hospitalization for worsening PAH Initiation of parenteral prostanoids (subcutaneous or intravenous infusion) ≥ 15% decline from baseline in 6MWD accompanied with continued or worsening to WHO FC III or IV symptoms. The decline in 6MWD must be confirmed with a repeat test on a different day within 2 weeks Time to clinical worsening is the number of days between the earliest occurrence of the clinical worsening component and the date of the first dose in the parent trial.

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 75 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Key Inclusion Criteria:

To be eligible, a participant is required to be or have:

Consented to participate in the LTE and has successfully completed the placebo-controlled 24-week Study AV-101-002.

Key Exclusion Criteria:

Subjects meeting any of the following criteria:

The Investigator believes that it would not be in the best interest of the subject to be included in the LTE e.g., for clinical or social reasons.
Subjects who were not compliant with study medication in AV-101-002 as assessed by the Investigator.