Recruiting

Phase 3

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

Sponsor:

Inhibikase Therapeutics

Code:

NCT07365332

Conditions

Pulmonary Arterial Hypertension

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

Interventions

IKT-001

Placebo

Study Details

Brief summary:

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.

Conditions

Pulmonary Arterial Hypertension

Study ID

NCT07365332

Start date

Apr 23, 2026

Status verified date

May, 2026

Completion date

Dec, 2029

Anticipated

Primary completion date

Jun, 2029

Anticipated

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

486 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: IKT-001

IKT-001 tablets for PO administration

placebo comparator: Placebo

Matching placebo to IKT-001 tablets for PO administration

Interventions

IKT-001

IKT-001 tablets for PO administration

Placebo

Placebo to IKT-001 tablets for PO administration

Primary outcome measure

[Part A] To evaluate the effect on Pulmonary Vascular Resistance (PVR) in participants with WHO Group 1 PAH treated with IKT-001 compared to placebo [ Time Frame: Baseline to Week 24 ]
[Part B] To characterize the effects of IKT-001 on symptoms and characteristics of Pulmonary Arterial Hypertension compared to placebo [ Time Frame: Baseline to Week 24 ]

Central Contacts and Locations

Central contacts

Medical Director, Inhibikase Therapeutics

1-302-295-3800 info@inhibikase.com

Locations

Arizona Pulmonary Specialists, LTD

Recruiting

Scottsdale, Arizona, United States, 85258

Cedars Sinai

Recruiting

Beverly Hills, California, United States, 90211

Jeffrey S. Sager, MD Medical Corporation

Recruiting

Santa Barbara, California, United States, 93105

Lundquist at Harbor UCLA

Recruiting

Torrance, California, United States, 90502

University of Colorado Anschutz Medical Campus

Recruiting

Aurora, Colorado, United States, 80045

University of Kansas Medical Center

Recruiting

Kansas City, Kansas, United States, 66103

Norton Healthcare

Recruiting

Louisville, Kentucky, United States, 40202

Tufts Medical Center

Recruiting

Boston, Massachusetts, United States, 02111

Washington University School of Medicine

Recruiting

St Louis, Missouri, United States, 63110

NYU Langone Health

Recruiting

New York, New York, United States, 10016

Westchester Medical Center

Recruiting

Valhalla, New York, United States, 10595

Duke University

Recruiting

Durham, North Carolina, United States, 27710

University of Cincinnati

Recruiting

Cincinnati, Ohio, United States, 45267

Vanderbilt University Medical Center

Recruiting

Nashville, Tennessee, United States, 37232-7610

UT Southwestern Medical Center

Recruiting

Dallas, Texas, United States, 75390

University of Alberta Hospital

Recruiting

Edmonton, Alberta, Canada, T6G 2B7

More Information

Sponsor

Inhibikase Therapeutics

Last update posted

Sep 2, 2026

Last verified

May, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Inhibikase Therapeutics on 2026-09-02.