Skip to main content
Back to Trials
NCT07365332PHASE3Recruiting

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

Inhibikase Therapeutics

Start Date

4/23/2026

Completion Date

12/1/2029

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.

Eligibility Criteria

Age Range: 18 years to 75 years

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.

Interventions

DRUG

IKT-001

DRUG

Placebo

Interested in This Trial?

Contact the trial locations directly using the information below to learn more about enrollment.

Expressing interest lets you review the study and consent before we connect you with the research site.

Conditions

Pulmonary Arterial Hypertension

Locations

Arizona Pulmonary Specialists, LTD

Scottsdale, Arizona 85258

United States

Cedars Sinai

Beverly Hills, California 90211

United States

Jeffrey S. Sager, MD Medical Corporation

Santa Barbara, California 93105

United States

Lundquist at Harbor UCLA

Torrance, California 90502

United States

University of Colorado Anschutz Medical Campus

Aurora, Colorado 80045

United States

University of Kansas Medical Center

Kansas City, Kansas 66103

United States

Norton Healthcare

Louisville, Kentucky 40202

United States

Tufts Medical Center

Boston, Massachusetts 02111

United States

Washington University School of Medicine

St Louis, Missouri 63110

United States

NYU Langone Health

New York, New York 10016

United States

Westchester Medical Center

Valhalla, New York 10595

United States

Duke University

Durham, North Carolina 27710

United States

University of Cincinnati

Cincinnati, Ohio 45267

United States

Vanderbilt University Medical Center

Nashville, Tennessee 37232-7610

United States

UT Southwestern Medical Center

Dallas, Texas 75390

United States

Sanatorio de la Trinidad Palermo

Buenos Aires, Buenos Aires 1425

Argentina

Pulmonary Hypertension Research Queensland PHRQ

Auchenflower, Queensland 4066

Australia

Hôpital Universitaire de Bruxelles - Erasme

Anderlecht, Brussels Capital 1070

Belgium

University of Alberta Hospital

Edmonton, Alberta T6G 2B7

Canada

Hôpital Bicêtre

Le Kremlin-Bicêtre, France 94270

France

CHRU Strasbourg

Strasbourg, Grand Est 67091

France

Medizinische Hochschule Hannover, Abteilung Pneumologie und Infektiologie

Hanover, Lower Saxony 30625

Germany

Fondazione IRCCS San Gerardo dei Tintori

Monza, Monza and Brianza 20900

Italy

Amsterdam UMC site VUMC

Amsterdam, North Holland 1081 BT

Netherlands

Auckland City Hospital

Auckland, Auckland 1051

New Zealand

University of Otago Christchurch

Christchurch, Canterbury 8011

New Zealand

Te Whato Ora, Health New Zealand, Waikato Hospital

Hamilton, Waikato Region 3204

New Zealand

Unidade Local de Saúde de Santo António - Hospital de Santo António

Porto, Porto District 4099-001

Portugal

Chonnam National University Hospital

Gwangju, 61469

South Korea

Samsung Medical Center

Seoul, 06351

South Korea

Hospital Universitario Virgen de la Victoria

Málaga, Andalusia 29010

Spain

Hospital Clínic de Barcelona

Barcelona, Barcelona 08036

Spain

Hospital Universitario Marqués De Valdecilla

Santander, Cantabria 39008

Spain

Hospital Universitari Vall d'Hebron

Barcelona, Catalonia 08035

Spain

Hospital Universitario Doce de Octubre

Madrid, Madrid 28041

Spain

Hospital Universitario la Paz

Madrid, Madrid 28046

Spain

Hospital Costa del Sol

Marbella, Malaga 29603

Spain

Hospital Universitario Virgen Macarena

Seville, Sevilla 41009

Spain

Hospital General Universitario de Toledo

Toledo, Toledo 45007

Spain

Hospital Universitari i Politecnic La Fe de Valencia

Valencia, Valencia 46026

Spain

Royal Papworth Hospital

Cambridge, Cambridgeshire CB2 0AY

United Kingdom

Golden Jubilee National Hospital

Glasgow, West Dunbartonshire G81 4DY

United Kingdom

Imperial College Healthcare NHS Trust

London, W12 0HS

United Kingdom