IMPAHCT
Research type
Research Study
Full title
IMPAHCT: A Phase 2b/3, Randomized, Double-Blind, Placebo-Controlled, 24-Week Dose Ranging and Confirmatory Study to Evaluate the Safety and Efficacy of AV-101 in Patients with Pulmonary Arterial Hypertension (PAH).
IRAS ID
299052
Contact name
Alistair Colin Church
Contact email
Sponsor organisation
Aerovate Therapeutics, Inc.
Eudract number
2021-001910-13
Duration of Study in the UK
3 years, 0 months, days
Research summary
PAH includes a group of rare, chronic cardiopulmonary diseases with various etiologies that share the common pathologic features of inappropriate cell growth resulting in the increased resistance to blood flow through the pulmonary vasculature. WHO classification Group 1 is characterised by the presence of pre-capillary pulmonary hypertension (mPAP ≥ 20 mmHg), a pulmonary artery wedge pressure ≤ 15 mmHg, and PVR > 3 Wood units. There is general consensus in the literature that PAH is a rare disease (Awdish et al., 2016). Typically prevalence rates in the range 0.15 – 0.25 per 10,000 are reported (McGoon et al., 2013). Similarly, Orphanet reports a prevalence of 0.2 per 10,000 (Orphanet, 2020). AV-101 received orphan drug designation from the CHMP on June 21, 2021.
Aerovate Therapeutics is developing AV-101 (imatinib) dry powder for oral inhalation for the treatment of pulmonary arterial hypertension (PAH; WHO Group 1) in adults with WHO Functional Class II–IV symptoms as a disease modifying drug to improve exercise capacity and delay disease progression. AV-101 is a drug-device combination product designed to deliver imatinib directly to respiratory tissues. Participants will be followed by the Investigator, with assessments conducted at the Screening/Enrollment Visit and at Clinic Visits. Study visits will occur at Screening, Day 1, Weeks 1, 4, 8, 12, 16, and 24. Visits on Weeks 1, 8, and 16 will be conducted by phone. There will be a 4-week follow-up visit by telephone after the participant’s end of treatment (EOT) Week 24 Visit, if they do not enter the Follow up Long-Term extension (LTE).
REC name
West Midlands - Coventry & Warwickshire Research Ethics Committee
REC reference
22/WM/0020
Date of REC Opinion
23 Mar 2022
REC opinion
Further Information Favourable Opinion