A Study of AV-101 (Dry Powder Inhaled Imatinib) in Patients With Pulmonary Arterial Hypertension (PAH) (IMPAHCT)

Aerovate Therapeutics

Status and phase: Terminated; Phase 3; Phase 2

Conditions: Pulmonary Arterial Hypertension

Treatments: Drug: AV-101; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT05036135

AV-101-002

Details and patient eligibility

About

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.

Enrollment: 202 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

---

202 participants in 4 patient groups, including a placebo group

AV-101 10 mg

Experimental group

Treatment:

Drug: AV-101

AV-101 35 mg

Experimental group

Treatment:

Drug: AV-101

AV-101 70 mg

Experimental group

Treatment:

Drug: AV-101

Placebo

Placebo Comparator group

Treatment:

Drug: Placebo