In short
IKT-001 is an investigational medicine being studied in adults with pulmonary arterial hypertension (PAH). A Phase 4 adaptive trial (NCT07365332) is currently authorised and plans to enroll 508 participants to evaluate the medicine against placebo when added to existing PAH therapy. The trial measures changes in pulmonary vascular resistance (PVR) and six-minute walk distance (6MWD) over 24 weeks.
Key points
- IKT-001 is an investigational medicine studied in pulmonary arterial hypertension (PAH).
- The trial is a Phase 4 adaptive study with a planned enrollment of 508 participants.
- Participants receive IKT-001 or placebo in addition to their background PAH therapy.
- The trial measures pulmonary vascular resistance (PVR) and six-minute walk distance (6MWD) at Week 24.
- The study is currently authorised but not yet recruiting.
- The trial record is available on ClinicalTrials.gov under NCT07365332.
What is IKT-001?
IKT-001 is an investigational medicine being studied in adults with pulmonary arterial hypertension (PAH). The full chemical name of the substance is BUTAN-2-YL [1-METHYL-4-[[4-[[4-METHYL-3-[(4-PYRIDIN-3-YLPYRIMIDIN-2-YL)AMINO]PHENYL]CARBAMOYL]PHENYL]METHYL]PIPERAZIN-1-IUM-1-YL]METHYL CARBONATE METHANESULFONATE. In the trial record, the medicine is referred to as IkT-001Pro, which is the form given to participants. No marketing authorisation is mentioned in the available data, which means IKT-001 is still under investigation rather than an approved medicine.
How does IKT-001 work?
The available trial record does not describe the mechanism of action of IKT-001 in detail. The study is designed to evaluate the efficacy and safety of IKT-001 compared with placebo when added to background PAH therapy in adults with WHO Group 1 PAH. Because the supplementary sources do not provide information on the molecular target or mechanism, the exact way IKT-001 acts in the body is not described here.
Which conditions is it studied for?
- Pulmonary arterial hypertension (PAH) — a condition in which the blood vessels in the lungs become narrowed, increasing the pressure in the pulmonary arteries and making the heart work harder to pump blood through the lungs. The trial studies IKT-001 in adults with WHO Group 1 PAH.
How is IKT-001 given?
IKT-001 is given by mouth (orally) in the form of tablets. The trial record lists an oral form of IkT-001Pro and a placebo designed to match 100 mg IKT-001 tablets. The dose of IKT-001 in the trial is not reported in the register, so the exact amount given to participants is not stated in the available data.
What do the clinical trials show?
One clinical trial of IKT-001 is recorded. It is a Phase 4 adaptive study in pulmonary arterial hypertension with a planned enrollment of 508 participants. The trial is currently authorised, meaning it has received the necessary approvals but has not yet started recruiting participants. The study compares IKT-001 with placebo when both are added to the participants' existing background PAH therapy.
The trial measures two primary outcomes. In Part A, the main measure is the change from baseline in pulmonary vascular resistance (PVR) — a measure of how much resistance the blood vessels in the lungs put up against blood flow — at Week 24. In Part B, the main measure is the change from baseline in six-minute walk distance (6MWD) — how far a person can walk in six minutes — at Week 24. The study is open to adults with WHO Group 1 PAH who are already receiving background PAH therapy.
What side effects are known?
The available sources do not provide information on side effects of IKT-001. The trial is designed to evaluate safety, but no safety results are reported in the registry record.