A Study of AV-101 (Dry Powder Inhaled Imatinib) in Patients With Pulmonary Arterial Hypertension (PAH)
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.
Timeline
- Start
- 2021-12-02
- Primary completion
- 2024-08-09
- Completion
- 2024-08-09
Outcome
Missed primary endpoint
Stopped: “The IMPAHCT study demonstrated that while inhaled imatinib was well tolerated, it did not prove to be efficacious at any of the doses of AV-101 evaluated in the study.”
paper Add-on dry powder inhaled imatinib (AV-101) was not effective in lowering PVR at any of the studied doses in patients with PAH. PMID 41738079 ↗
Publications
- Hill NS, Gillies H, Chakinala MM, Feldman JP, Hoeper MM, Jing ZC, Mclaughlin VV, Rosenkranz S, Escribano-Subias P, Ghio S, Gomberg-Maitland M, Habib N, Kopec G, Langleben D, Lescano A, Ramirez A, Rischard F, Sitbon O, Skride A, Dake BT, Khindri S, Langley J, Niven R, Zhang X, Humbert M. Phase 2b trial of inhaled imatinib for treatment of pulmonary arterial hypertension. Am J Respir Crit Care Med. 2026 Apr 1;212(4):802-812. doi: 10.1093/ajrccm/aamaf128.
- Gillies H, Chakinala MM, Dake BT, Feldman JP, Hoeper MM, Humbert M, Jing ZC, Langley J, McLaughlin VV, Niven RW, Rosenkranz S, Zhang X, Hill NS. IMPAHCT: A randomized phase 2b/3 study of inhaled imatinib for pulmonary arterial hypertension. Pulm Circ. 2024 Mar 25;14(1):e12352. doi: 10.1002/pul2.12352. eCollection 2024 Jan.
- Gillies H, Niven R, Dake BT, Chakinala MM, Feldman JP, Hill NS, Hoeper MM, Humbert M, McLaughlin VV, Kankam M. AV-101, a novel inhaled dry-powder formulation of imatinib, in healthy adult participants: a phase 1 single and multiple ascending dose study. ERJ Open Res. 2023 Mar 13;9(2):00433-2022. doi: 10.1183/23120541.00433-2022. eCollection 2023 Mar.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Imatinib | Small molecule | 10 mg | Inhaled |
| Subject | Imatinib | Small molecule | 35 mg | Inhaled |
| Subject | Imatinib | Small molecule | 70 mg | Inhaled |