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A study to evaluate the safety and effectiveness of IKT-001 compared to a placebo in adults with pulmonary arterial hypertension.

Fast replyInvestigationalNo placebo
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What is this trial about?

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

This study focuses on individuals living with Pulmonary Arterial Hypertension, a type of high blood pressure that affects the arteries in the lungs and the right side of the heart. The purpose of the study is to evaluate the efficacy and safety of a new medication called IKT-001 compared to a placebo when used alongside standard existing treatments.

Participants in the study will receive either the IKT-001 film-coated tablet or a placebo. The study is designed in two parts to observe how the medication affects different aspects of the condition over a period of time. During the study, changes in pulmonary vascular resistance, which is the resistance to blood flow through the lung vessels, and the six-minute walk distance, which is a test measuring how far a person can walk in six minutes to assess physical capacity, will be monitored.

The research process

The trial runs in 2 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Participation in part a

    During the first part of the study, ikt-001 or a placebo (a pill with no active medicine) is taken in addition to the current treatment for pulmonary arterial hypertension, which is a type of high blood pressure in the lungs.

    The treatment lasts for 24 weeks.

    The pulmonary vascular resistance, which is a measurement of how much pressure is in the lung arteries, is monitored to see if there is a change from the start of the study.

  2. Step 2

    Participation in part b

    In the second part of the study, ikt-001 or a placebo is administered alongside existing pulmonary arterial hypertension treatments.

    This stage also lasts for 24 weeks.

    The 6-minute walk distance, which is a test of how far a person can walk in six minutes to measure physical ability, is used to evaluate the effect of the medication.

Who can join the trial?

16 criteria

  • You must be able to provide signed informed consent, which means you agree to participate in the study after understanding all the details and following the rules provided.
  • You must have a blood level of NT-proBNP (a protein used to check how well the heart is working) that is higher than 300 ng/L.
  • Men with a partner who could become pregnant must agree to use a condom from the first dose until 7 days after the last dose.
  • Women of childbearing potential must use a highly effective contraceptive method (birth control with a failure rate of less than 1% per year) from the first dose until 7 days after the last dose.
  • Women of childbearing potential must agree not to donate eggs for reproduction from 14 days before the first dose until the end of the study.
  • Women of childbearing potential must have a negative pregnancy test at the start and before receiving the first dose.
  • You must have a confirmed diagnosis of WHO Group 1 Pulmonary Arterial Hypertension (PAH), which is a specific type of high blood pressure in the lungs.
  • You must be between 18 and 75 years old at the time of signing the consent form.
  • Your BMI (Body Mass Index, a measure of body fat based on height and weight) must be between 18.5 and 35.0.
  • A Right Heart Catheterization (RHC), which is a procedure to measure pressures in the heart and lungs, must show a PVR (pulmonary vascular resistance, or how hard it is for blood to flow through the lungs) of 400 or more.
  • Your PCWP (pulmonary capillary wedge pressure, a measure of pressure in the left side of the heart) or left ventricular end-diastolic pressure must be 15 mmHg or less.
  • Your mPAP (mean pulmonary artery pressure, the average blood pressure in the lung arteries) must be higher than 20 mmHg.
  • You must be taking stable doses of up to three existing medications for your lung condition for at least 90 days before the study begins.
  • You must not be currently using a medication called sotatercept.
  • You must be able to complete a 6-minute walk test (6MWT), which measures how far you can walk in 6 minutes, throughout the study.
  • Your 6-minute walk distance (6MWD) must be between 100 and 475 meters, and two separate tests must be within 15% of each other.

Who cannot join the trial?

34 criteria

  • Having pulmonary arterial hypertension (PAH) caused by groups 2, 3, 4, or 5 rather than group 1.
  • Having specific types of group 1 PAH, such as those related to portal hypertension (high blood pressure in the veins connected to the liver), schistosomiasis (a disease caused by parasitic worms), or pulmonary veno-occlusive disease (a condition where small veins in the lungs are blocked).
  • Having obstructive sleep apnea (a condition where breathing repeatedly stops and starts during sleep) that is not being properly treated.
  • Having an active or past infection of hepatitis B or hepatitis C (infections that cause liver inflammation).
  • Having a bleeding disorder, such as hemophilia (a condition where blood does not clot normally), von Willebrand disease (a disorder that affects blood clotting), or thrombocytopenia (a low number of blood platelets, which help the blood clot).
  • Having a history of any bleeding event that required medical help, such as a transfusion (transferring blood or blood products into the body).
  • Taking certain medications, including strong CYP3A inducers or inhibitors (medicines that change how the body processes other drugs), anticoagulants (blood thinners like heparin), or the drug sotatercept.
  • Starting a new cardiopulmonary rehabilitation (a program to improve heart and lung health) exercise program within 90 days of joining the study or planning to start one during the study.
  • Having had an atrial septostomy (a procedure to make a hole in the wall between the heart's upper chambers) within the last 180 days.
  • Being part of another medical study or taking an experimental medicine within the last 90 days.
  • Having previously participated in this or another study involving IKT-001.
  • Having any medical or social reason that would prevent you from finishing the study.
  • Having low FVC (forced vital capacity, which is the amount of air you can exhale forcefully) on a breathing test, or having interstitial lung disease (scarring or inflammation in the lung tissue) if your breathing test results are in a specific mid-range.
  • Having abnormal blood test results, such as high levels of liver enzymes (ALT or AST) or bilirubin, low white blood cell counts (ANC), low hemoglobin or hematocrit (levels related to red blood cells), low platelets, or low eGFR (a measure of how well the kidneys filter blood).
  • Being pregnant, breastfeeding, or planning to become pregnant during the study.
  • Having previously received a solid organ transplant or a stem cell transplant.
  • Having a history of using methamphetamine or cocaine, or having a diagnosis related to these substances within the last 24 months.
  • Planning a surgery that would require stopping the study medicine or interfere with the study checks.
  • Having had malignancy (cancer) within the last 5 years, with some very specific low-risk exceptions.
  • Having uncontrolled diabetes mellitus (a condition affecting blood sugar), defined by a high HbA1c level.
  • Having certain blood pressure issues, such as uncontrolled systemic hypertension (very high blood pressure), very low blood pressure, or syncope (fainting) within the last 3 months.
  • Having a history of heart muscle problems like cardiomyopathy (diseases of the heart muscle).
  • Having an abnormal ECG (an electrical recording of the heart's activity), specifically regarding the QTcF interval (a measure of the heart's electrical timing).
  • Having a low LVEF (left ventricular ejection fraction, a measure of how much blood the heart pumps out with each beat) or signs of impaired heart relaxation.
  • Having a personal or family history of long QT syndrome (a heart rhythm disorder) or sudden cardiac death.
  • Having an implanted device for monitoring heart pressure, such as a CardioMEMS device.
  • Having significant eye diseases, such as macular edema (swelling in the part of the eye responsible for sharp vision), glaucoma (high pressure in the eye), or optic neuropathy (damage to the optic nerve).
  • Having an allergy or hypersensitivity to the study medicine (IKT-001) or its ingredients.
  • Having a history of irregular heart rhythms like atrial fibrillation or atrial flutter.
  • Having a history of a cerebrovascular accident (stroke), brain bleeding, or a head injury from a fall within the last 3 months.
  • Having experienced a heart event like a myocardial infarction (heart attack) or heart surgery within the last 6 months.
  • Having sudden right heart failure within the last 30 days.
  • Having significant heart disease, such as problems with heart valves or ischemic heart disease (reduced blood flow to the heart).
  • Having a history of a pneumonectomy (surgical removal of a lung).
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Investigated drugs

IkT-001Pro is an experimental medicine being tested to see if it can help treat pulmonary arterial hypertension, a type of high blood pressure in the lungs. It is taken as a tablet that you swallow.

What is already known about the treatment

  • IkT-001Pro

    This medication is an experimental oral film-coated tablet currently being studied in clinical trials for its potential to treat pulmonary arterial hypertension. It belongs to a class of drugs designed to target specific pathways in the body to help improve blood flow in the lungs. On a molecular level, it works by blocking certain proteins that cause blood vessels to narrow and tighten. Because it is still in the testing phase, its full safety and effectiveness are currently being evaluated by researchers.

  • Placebo to match 100 mg IKT-001 tablets

    This is an inactive substance used in clinical trials that looks, tastes, and is taken exactly like the experimental medication. It contains no active medicinal ingredients and is administered orally in the same film-coated tablet form. In medical research, the placebo serves as a control to help scientists determine if the effects seen in patients are actually caused by the new drug. It is a standard tool used to ensure the accuracy and reliability of the study results.

Investigated diseases

Pulmonary Arterial Hypertension - This condition involves high blood pressure within the arteries that carry blood from the heart to the lungs. Over time, the walls of these arteries become thick, stiff, and narrow. This narrowing makes it harder for blood to flow through the lung vessels. As a result, the heart must work much harder to pump blood through the lungs. This increased workload can lead to the heart muscle becoming enlarged or weakened.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IVTrial ID2024-520218-23-00Protocol codeIKT-001-201Estimated enrolment508 patients

sourced from the EU Clinical Trials Register and site verification

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