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Study on the Safety and Effectiveness of Inhaled Imatinib (AV-101) for Patients with Pulmonary Arterial Hypertension (PAH)

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition known as Pulmonary Arterial Hypertension (PAH). PAH is a type of high blood pressure that affects the arteries in the lungs and the right side of the heart. The study is testing a treatment called AV-101 (imatinib), which is a medication that comes in capsule form and is inhaled using a special device. Imatinib is a type of medicine known as a tyrosine kinase inhibitor, which works by blocking certain proteins that can contribute to the disease.

The purpose of this study is to find the best dose of AV-101 for treating PAH and to evaluate its safety and effectiveness. Participants in the study will be randomly assigned to receive either AV-101 or a placebo. The study will last for 24 weeks, during which time participants will take the medication and have regular check-ups to monitor their health and the effects of the treatment. The study is divided into two parts: Phase 2b, which focuses on finding the optimal dose, and Phase 3, which confirms the effectiveness of the treatment.

Throughout the study, participants will undergo various assessments, including a test called right heart catheterization, which helps measure changes in the pressure in the lungs. Another important measure is the 6-minute walk distance (6MWD), which evaluates how far a person can walk in six minutes. These assessments will help determine how well the treatment is working and ensure it is safe for patients with PAH. The study aims to improve the quality of life for those affected by this condition by finding a more effective treatment option.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, eligibility is confirmed based on specific criteria such as age, diagnosis of pulmonary arterial hypertension (PAH), and ability to walk a certain distance during a test.

  2. Step 2

    Randomization and initial assessment

    Participants are randomly assigned to receive either the study medication, AV-101, or a placebo. Initial assessments are conducted to establish baseline measurements, including a right heart catheterization to assess pulmonary vascular resistance (PVR) and a 6-minute walk distance (6MWD) test.

  3. Step 3

    Medication administration

    Participants receive the study medication, AV-101, or a placebo in the form of inhalation capsules. The dosage and frequency are determined by the study protocol, and the treatment continues for a duration of 24 weeks.

  4. Step 4

    Ongoing assessments

    Throughout the 24-week period, regular assessments are conducted to monitor safety and efficacy. These include measuring changes in PVR, 6MWD, and other health indicators such as NT-proBNP levels and WHO Functional Class.

  5. Step 5

    Final evaluation

    At the end of the 24-week treatment period, a final evaluation is conducted. This includes a comparison of baseline and post-treatment measurements to assess the primary and secondary endpoints of the study.

Who can join the trial?

7 criteria

  • Must be a male or female between 18 and 75 years old at the time of the Screening Visit.
  • Must have a diagnosis of Pulmonary Arterial Hypertension (PAH) that fits into one of the specific subgroups:
    • I/HPAH, PAH-CTD: These are specific types of PAH.
    • PAH caused by drugs, toxins, or chemicals, and must have been under the care of the study doctor for at least one year without any relapses of drug or toxin/chemical abuse.
    • PAH associated with HIV.
    • PAH due to repaired congenital heart disease, with the repair done at least one year ago.
    • Must have symptoms that are classified as World Health Organization (WHO) Functional Class II, III, or IV. This is a way to describe how severe the symptoms are.
    • Must be able to walk at least 100 meters but no more than 475 meters during a test called the 6-minute walk test, which measures how far you can walk in six minutes.

Who cannot join the trial?

15 criteria

  • Patients with other serious health conditions that could interfere with the study.
  • Patients who are pregnant or breastfeeding.
  • Patients who have participated in another clinical trial recently.
  • Patients who have a history of drug or alcohol abuse.
  • Patients who are unable to follow the study procedures or take the study medication as required.
  • Patients with allergies to the study medication or its ingredients.
  • Patients with certain heart conditions that could make participation unsafe.
  • Patients who have had a recent heart attack or stroke.
  • Patients with severe liver or kidney disease.
  • Patients who are taking medications that could interact with the study drug.
  • Patients with uncontrolled high blood pressure.
  • Patients with certain lung diseases other than **Pulmonary Arterial Hypertension (PAH)**.
  • Patients who have had a lung transplant.
  • Patients with a history of cancer within the last five years, except for some skin cancers.
  • Patients with certain infections that could affect the study results.
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Investigated drugs

AV-101 is a medication being studied for its potential to treat pulmonary arterial hypertension (PAH). The trial aims to find the best dose of AV-101 by looking at how it affects the blood vessels in the lungs and its safety for patients. The study measures changes in how far patients can walk in six minutes after 24 weeks of treatment to see how well the medication works.

What is already known about the treatment

AV-101 – AV-101 is an investigational medication currently being studied for the treatment of Pulmonary Arterial Hypertension (PAH). It is administered orally in the form of tablets. The medication is in the clinical trial phase, specifically a Phase 2b/3 study, to determine its safety and effectiveness. AV-101 is designed to help improve blood flow in the lungs by targeting specific pathways that affect blood vessel constriction and relaxation. It belongs to a class of drugs known as vasodilators, which work by widening blood vessels to reduce blood pressure in the lungs.

Investigated diseases

Pulmonary Arterial Hypertension (PAH) – Pulmonary Arterial Hypertension is a condition characterized by high blood pressure in the arteries that supply the lungs. This increased pressure makes it difficult for the heart to pump blood through the lungs, leading to symptoms such as shortness of breath, fatigue, and chest pain. Over time, the heart may become enlarged and weakened due to the increased workload. The disease progresses as the blood vessels in the lungs become more narrowed or blocked, further increasing the pressure. This can lead to a decrease in physical activity tolerance and may cause swelling in the legs and abdomen. The progression of PAH can vary, with some individuals experiencing a gradual worsening of symptoms.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IVTrial ID2023-508734-34-00Protocol codeAV-101-002Estimated enrolment462 patientsSponsorAerovate Therapeutics Inc.

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