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

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What is this study about?

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.

Who Can Join the Study?

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 Study?

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).

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Medizinische Hochschule Hannover	Hanover	Germany
Hospital Universitario Y Politecnico La Fe	Valencia	Spain
Hospital Universitario De Salamanca	Salamanca	Spain
Technische Universitaet Dresden	Dresden	Germany
Katholieke Universiteit te Leuven	Leuven	Belgium
Unidade Local De Saúde De Santa Maria, E.P.E.	Lisbon	Portugal
Université Libre de Bruxelles – Hôpital Erasme	Brussels	Belgium

Other Sites

Site Name	City	Country
Pauls Stradins Clinical University Hospital	Riga	Latvia
Unidade Local De Saude De Almada-Seixal E.P.E.	Almada	Portugal
Mater Misericordiae University Hospital	Dublin	Ireland
Hospital Universitario 12 De Octubre	Madrid	Spain
Wojewodzki Specjalistyczny Szpital Im Dr Wl Bieganskiego	Lodz	Poland
Fondazione IRCCS San Gerardo Dei Tintori	Monza	Italy
Multimedica S.p.A.	Milan	Italy
Hospital Universitario De Toledo Ute	Toledo	Spain
Hospital Universitario Virgen Macarena	Sevilla	Spain
Gornoslaskie Centrum Mwdyczne	Katowice	Poland
Fondazione IRCCS Policlinico San Matteo	Pavia	Italy
Hospital Clinic De Barcelona	Barcelona	Spain
Justus-Liebig-Universitaet Giessen	Giessen	Germany
Region Midtjylland	Aarhus	Denmark
Vseobecna Fakultni Nemocnice V Praze	Prague	Czechia
Thoraxklinik Heidelberg gGmbH	Heidelberg	Germany
Rigshospitalet	Copenhagen	Denmark
Centro Hospitalar Universitario De Santo Antonio E.P.E.	Porto	Portugal
Hopital Beaujon	Clichy	France
Hospital Costa del Sol	Marbella	Spain
University General Hospital Of Thessaloniki Ahepa	Thessaloniki	Greece
Hospital Universitario Virgen De La Victoria	Malaga	Spain
University General Hospital Attikon General Hospital Of West Attica H Agia Varvara	Chaidari	Greece
Inwbmhpsu Fmy Chmaecif Aam Emcrolbgylmj Mukjoznw	Prague	Czechia
Hegdqact Uvljkjfqyjmzz Mgbmadh Do Vqwojgdzwv	Santander	Spain
Avccykjxa Upi	Amsterdam	The Netherlands
Ayemjrhcwm Pwhgjzxl Hepmsvyg Dc Mcluodfny	Marseille	France
Uxzpmbldmt Dqwan Sgrqt Dh Rukp La Siszcxly	Rome	Italy
Fdrstbklx Pzdo Lu Itomkgonfvxmu Bqxnongpe Dlq Hixoeynf Uzgkhwdzswugg Li Phs	Madrid	Spain
Imbzsreh dy Cbumosgrzzpf Haoolgoehki Uodpfofahqfhi du Slryx Eqdtnfe (loyroiy	Saint Priest En Jarez	France
Hxgccork Vlma djakkrmb	Barcelona	Spain
Klnvvzrwr Siueffr Sekbawzkmjtjxwv ia Jipv Pjjtb Ia	Cracow	Poland
Hjhqpqwe Ukmymkrdxdqeqy Siicnarusc &fukmlh Hstzcpj du Hunueeeybqy	STRASBOURG, Alsace	France

Trial status

Country	Status	Recruitment Start
Belgium	Not yet recruiting	01.06.2026
Czechia	Not yet recruiting	01.06.2026
Denmark	Not yet recruiting	01.06.2026
France	Not yet recruiting	01.06.2026
Germany	Not yet recruiting	01.06.2026
Greece	Not yet recruiting	01.06.2026
Ireland	Not yet recruiting	01.06.2026
Italy	Not yet recruiting	01.06.2026
Latvia	Not yet recruiting	01.06.2026
Poland	Not yet recruiting	01.06.2026
Portugal	Not yet recruiting	01.06.2026
Spain	Not yet recruiting	01.06.2026
The Netherlands	Not yet recruiting	01.06.2026

Trial locations

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.

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 ID:

2024-520218-23-00

Protocol code:

IKT-001-201

NCT ID:

NCT07365332

Trial Phase:

Therapeutic use (Phase IV)

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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.

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?