Skip to main content
TTrialFinderData
TrialFinderData is for informational purposes only and does not provide medical advice. Always talk to your doctor.

Updated June 2026 · ClinicalTrials.gov

RECRUITINGPhase 3INTERVENTIONAL

An Adaptive Program of IKT-001 in Pulmonary Arterial Hypertension (PAH)

An Adaptive, 2-Part, Randomized, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of IKT-001 in Pulmonary Arterial Hypertension (PAH)

An Adaptive Program of IKT-001 in Pulmonary Arterial Hypertension (PAH) (NCT07365332) is a Phase 3 interventional studying Pulmonary Arterial Hypertension, sponsored by Inhibikase Therapeutics. RECRUITING as of the most recent ClinicalTrials.gov update. Talk to your doctor before contacting the trial site.

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

This is an adaptive, 2-part, randomized, multicenter, double-blind, placebo-controlled, parallel-group study designed to evaluate the efficacy and safety of IKT-001 in adult participants with WHO Group 1 PAH.

What Stage of Research Is This?

Phase 3 trials confirm efficacy and safety in large patient groups (often 300–3,000+) and form the evidence base for an FDA approval submission. For Pulmonary Arterial Hypertension, Phase 3 studies typically randomize participants between the investigational treatment and either a placebo or current standard of care. A successful Phase 3 result is the threshold most treatments need to clear before regulatory approval.

This trial is currently recruiting participants. The sponsor has registered the study with ClinicalTrials.gov as actively enrolling, which means new applicants who meet the eligibility criteria can be considered for screening. Trial status can change between updates — confirm current recruiting status with the study contact before traveling for a screening visit.

A target enrollment of 486 participants makes this a sizable late-stage trial. Studies in this range typically have enough power to detect clinically meaningful differences from a comparator and to characterize less-common side effects.

Who May Be Eligible (Plain English)

Who May Qualify: - Documented diagnosis of WHO PAH Group 1 in any of the following subtypes: - Idiopathic PAH - Heritable PAH - Drug/toxin-induced PAH - PAH associated with connective tissue disease (CTD) - PAH associated with simple, congenital systemic-to-pulmonary shunts at least 1 year following repair - Men and women 18 and 75 years of age (inclusive) - Must have a body mass index (BMI) of ≥18.5 kg/m\^2 and ≤35.0 kg/m\^2 at screening. - Baseline RHC performed during the Screening Period documenting a PVR of ≥ 400 dyn/sec/cm\^5 ; pulmonary capillary wedge pressure (PCWP) ≤15 mmHg and mean pulmonary artery pressure (mPAP) \>20 mmHg. PVR enrichment criteria to ensure population baseline PVR \>700 dynes/sec/cm\^5 - On stable doses of background PAH therapy including endothelin receptor antagonists, phosphodiesterase-5 inhibitors, prostacyclins, and soluble guanylate cyclase stimulators for ≥90 days prior to screening. Current use of sotatercept is not permitted. - 6MWD ≥ 100 and ≤ 475 m Who Should NOT Join This Trial: - Diagnosis of PAH WHO Groups 2, 3, 4, or 5. - Diagnosis of the following PAH Group 1 subtypes: human weakened immune system virus (HIV)-associated PAH, PAH associated with portal hypertension, schistosomiasis-associated PAH, and pulmonary veno-occlusive disease. - Any of the following blood pressure-related values or abnormalities: Uncontrolled systemic hypertension as evidenced by sitting systolic BP \>160 mmHg or sitting diastolic BP \>100 mmHg at screening, Baseline systolic BP \<90 mmHg at screening, Syncope within 3 months prior to screening - History of restrictive, constrictive, or congestive cardiomyopathy. - ECG with Fridericia's corrected QT interval (QTcF) ≥ 450 msec in males or ≥ 470 msec in females at screening or ≥500 msec in the presence of a right bundle branch block. - Personal or family history of long QT syndrome or sudden cardiac death. - Presence of a CardioMEMS device or any other implanted hemodynamic monitoring device. ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

These are translations of the protocol\'s inclusion and exclusion criteria, simplified for patients and caregivers. The original clinical text appears below. Eligibility is ultimately confirmed by the trial site\'s screening process — this summary is a starting point for a conversation with your doctor, not a final determination.

Original Eligibility Criteria

View original clinical language
Inclusion Criteria: * Documented diagnosis of WHO PAH Group 1 in any of the following subtypes: * Idiopathic PAH * Heritable PAH * Drug/toxin-induced PAH * PAH associated with connective tissue disease (CTD) * PAH associated with simple, congenital systemic-to-pulmonary shunts at least 1 year following repair * Men and women 18 and 75 years of age (inclusive) * Must have a body mass index (BMI) of ≥18.5 kg/m\^2 and ≤35.0 kg/m\^2 at screening. * Baseline RHC performed during the Screening Period documenting a PVR of ≥ 400 dyn/sec/cm\^5 ; pulmonary capillary wedge pressure (PCWP) ≤15 mmHg and mean pulmonary artery pressure (mPAP) \>20 mmHg. PVR enrichment criteria to ensure population baseline PVR \>700 dynes/sec/cm\^5 * On stable doses of background PAH therapy including endothelin receptor antagonists, phosphodiesterase-5 inhibitors, prostacyclins, and soluble guanylate cyclase stimulators for ≥90 days prior to screening. Current use of sotatercept is not permitted. * 6MWD ≥ 100 and ≤ 475 m Exclusion Criteria: * Diagnosis of PAH WHO Groups 2, 3, 4, or 5. * Diagnosis of the following PAH Group 1 subtypes: human immunodeficiency virus (HIV)-associated PAH, PAH associated with portal hypertension, schistosomiasis-associated PAH, and pulmonary veno-occlusive disease. * Any of the following blood pressure-related values or abnormalities: Uncontrolled systemic hypertension as evidenced by sitting systolic BP \>160 mmHg or sitting diastolic BP \>100 mmHg at screening, Baseline systolic BP \<90 mmHg at screening, Syncope within 3 months prior to screening * History of restrictive, constrictive, or congestive cardiomyopathy. * ECG with Fridericia's corrected QT interval (QTcF) ≥ 450 msec in males or ≥ 470 msec in females at screening or ≥500 msec in the presence of a right bundle branch block. * Personal or family history of long QT syndrome or sudden cardiac death. * Presence of a CardioMEMS device or any other implanted hemodynamic monitoring device. * Forced vital capacity (FVC) \<70 percent on pulmonary function test (PFT) performed no more than 6 months prior to screening; or if FVC is 60 percent to 69 percent, must have a chest computed tomography scan within 12 months with no more than mild interstitial lung disease. * History of atrial fibrillation or atrial flutter. * History of cerebrovascular accident, intracranial hemorrhage, or subdural hematoma at anytime, or a fall associated with head trauma within 3 months of screening. * Acutely decompensated right heart failure within 30 days prior to screening, as per investigator assessment. * Clinically significant ischemic, valvular, constrictive heart disease, or heart failure with preserved ejection fraction in the opinion of the investigator. * History of pneumonectomy. * Untreated or inadequately treated (in the opinion of the investigator) obstructive sleep apnea. * Acute or chronic hepatitis B or C infection, defined as: * Hepatitis B virus: a positive hepatitis B surface antigen test or a positive hepatitis B core antibody test with detectable DNA * Hepatitis C virus (HCV): a positive hepatitis C antibody test with detectable HCV ribonucleic acid (RNA).Participants with a positive hepatitis C antibody test, but no detectable HCV RNA who completed treatment with direct-acting antivirals may be considered after discussion with the medical monitor. * History of or currently diagnosed with a bleeding disorder, including but not limited to hemophilia, von Willebrand disease, thrombocytopenia, or significant bleeding history defined as any bleeding event requiring medical intervention. * Received treatment with any of the following excluded medications: * Currently receiving strong cytochrome P450 (CYP) 3A inducers or CYP3A inhibitors (except for topical administration) * Currently receiving or anticipated need to receive any anticoagulant (e.g., heparins, vitamin K antagonists, direct oral anticoagulants, or direct thrombin inhibitors). * Current use of sotatercept. Note: participants who previously received sotatercept may be considered if the last dose administered was \>6 months prior to screening, participant had no significant bleeding events while on sotatercept. * Initiation of an exercise program for cardiopulmonary rehabilitation within 90 days prior to screening or planned initiation during the study (participants who are stable in the maintenance phase of a program and who will continue for the duration of the study are eligible). * History of atrial septostomy within 180 days prior to screening. * Current participation in another investigational clinical trial and/or receipt of any investigational medication within 90 days prior to screening. * Previous randomization into this or another IKT-001 study. * Any social, behavioral, or medical reason that would preclude completion of the study, in the judgement of the investigator. * Currently lactating, pregnant or planning on becoming pregnant during the study. * Prior receipt of a solid organ transplant or stem cell transplant. * Planned surgery that would require any study drug interruption or interfere with study assessments during the study (minor procedures may be allowed in consultation with the medical monitor). * Malignancy within the last 5 years prior to consent except completely treated non-metastatic-basal cell, squamous cell, in situ cervical cancer, and clinically localized National Comprehensive Cancer Network very low to low risk prostate cancer under active surveillance.

Treatments Being Tested

DRUG

IKT-001

IKT-001 tablets for PO administration

DRUG

Placebo

Placebo to IKT-001 tablets for PO administration

Locations (2)

Trial sites listed on ClinicalTrials.gov for this study. Site activation status can vary — confirm with the specific site before traveling for a screening visit.

Norton Healthcare
Louisville, Kentucky, United States
Tufts Medical Center
Boston, Massachusetts, United States

How to Talk to Your Doctor About This Trial

Bring the printable summary of this trial — including the NCT ID (NCT07365332), the sponsor (Inhibikase Therapeutics), and the key eligibility criteria — to your next appointment. Your doctor can review the inclusion and exclusion criteria against your medical history, lab values, and current treatments to assess whether you are likely to qualify. They can also help you weigh whether trial participation makes sense alongside your existing care plan.

Useful questions to walk through together: What does the trial protocol require beyond standard care? How long is the active treatment phase, and how long is follow-up? Are there study visits at sites I can reach? Who pays for the trial-specific procedures, and who pays for standard-of-care portions? See our 25 questions to ask about clinical trials guide for a more complete checklist.

Authoritative Sources

The official record for this trial lives on ClinicalTrials.gov — the federal registry maintained by the National Library of Medicine at NIH. For background on how this trial fits into the FDA approval pathway, see the FDA drug approval process. For oncology-specific guidance for patients considering trials, the National Cancer Institute publishes patient-oriented overviews. International trial registries are aggregated by the WHO ICTRP.

Frequently Asked Questions

What is the NCT07365332 clinical trial studying?

This is an adaptive, 2-part, randomized, multicenter, double-blind, placebo-controlled, parallel-group study designed to evaluate the efficacy and safety of IKT-001 in adult participants with WHO Group 1 PAH. The full protocol is registered on ClinicalTrials.gov and includes the primary outcome measures, eligibility criteria, and study endpoints.

Who can participate in NCT07365332?

Eligibility for this trial depends on the specific inclusion and exclusion criteria set by the sponsor. The plain-English summary above translates the most important criteria into accessible language; the official clinical text is preserved in the collapsible section underneath. Whether you fit any specific trial is a medical decision your doctor needs to confirm — bring the trial information to your treating physician for a full review against your medical history.

How do I contact the trial site for NCT07365332?

Contact information registered with ClinicalTrials.gov is shown in the sidebar of this page. Before reaching out, confirm with your treating physician that this trial is appropriate for your situation. The trial site will then walk you through the screening process to determine final eligibility.

Is participating in a clinical trial safe?

Clinical trials in the United States are regulated by the FDA and overseen by Institutional Review Boards (IRBs) that review the protocol for safety. Risk varies by trial — Phase 1 studies test new treatments in humans for the first time, while Phase 3 trials use treatments that have already passed earlier safety screening. The informed consent document for any specific trial details the known risks and what to expect. Discuss those risks with your physician before deciding whether to participate.

Where can I verify the data on this page?

Every detail on this page comes directly from the ClinicalTrials.gov API. Click "View on ClinicalTrials.gov" in the sidebar to see the official, unmodified record. The federal record is always authoritative; this page is a structured presentation with a plain-English eligibility translation. For background on how clinical trials are regulated, see the FDA drug approval process documentation.

How This Page Is Built

Every field on this page is pulled directly from the ClinicalTrials.gov API v2 — no estimates, no proxies. The plain-English eligibility translation is generated from the original protocol text and reviewed for fidelity to the underlying clinical criteria. The original clinical text remains visible in the collapsible section above so users and clinicians can verify the translation. Read the full methodology for the data pipeline and known limitations.

Source: ClinicalTrials.gov API v2 record for NCT07365332. Maintained by the National Library of Medicine at NIH. Public domain. Cite as: "TrialFinderData. NCT07365332. Data: ClinicalTrials.gov."

Medical disclaimer: This page is informational, not medical advice. Talk to your doctor about whether a clinical trial is right for you.

Last updated 2026-06-07 · Data from ClinicalTrials.gov.