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

NCT07365332 · Inhibikase Therapeutics
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Official title
An Adaptive, 2-Part, Randomized, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of IKT-001 in Pulmonary Arterial Hypertension (PAH)
About this study
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
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 target: 486 participants
Allocation: randomized
Masking: quadruple
Age groups: adult, older_adult
Timeline
Starts: 2026-04-23
Estimated completion: 2029-12
Last updated: 2026-09-02
Interventions
Drug: IKT-001Drug: Placebo
Primary outcomes
  • [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 (Baseline to Week 24)
  • [Part B] To characterize the effects of IKT-001 on symptoms and characteristics of Pulmonary Arterial Hypertension compared to placebo (Baseline to Week 24)
Sponsor
Inhibikase Therapeutics · industry
Contacts & investigators
ContactMedical Director, Inhibikase Therapeutics · contact · info@inhibikase.com · 1-302-295-3800
All locations (43)
Arizona Pulmonary Specialists, LTDRecruiting
Scottsdale, Arizona, United States
Cedars SinaiRecruiting
Beverly Hills, California, United States
Jeffrey S. Sager, MD Medical CorporationRecruiting
Santa Barbara, California, United States
Lundquist at Harbor UCLARecruiting
Torrance, California, United States
University of Colorado Anschutz Medical CampusRecruiting
Aurora, Colorado, United States
University of Kansas Medical CenterRecruiting
Kansas City, Kansas, United States
Norton HealthcareRecruiting
Louisville, Kentucky, United States
Tufts Medical CenterRecruiting
Boston, Massachusetts, United States
Washington University School of MedicineRecruiting
St Louis, Missouri, United States
NYU Langone HealthRecruiting
New York, New York, United States
Westchester Medical CenterRecruiting
Valhalla, New York, United States
Duke UniversityRecruiting
Durham, North Carolina, United States
University of CincinnatiRecruiting
Cincinnati, Ohio, United States
Vanderbilt University Medical CenterRecruiting
Nashville, Tennessee, United States
UT Southwestern Medical CenterRecruiting
Dallas, Texas, United States
Sanatorio de la Trinidad PalermoRecruiting
Buenos Aires, Buenos Aires, Argentina
Pulmonary Hypertension Research Queensland PHRQRecruiting
Auchenflower, Queensland, Australia
Hôpital Universitaire de Bruxelles - ErasmeRecruiting
Anderlecht, Brussels Capital, Belgium
University of Alberta HospitalRecruiting
Edmonton, Alberta, Canada
Hôpital BicêtreRecruiting
Le Kremlin-Bicêtre, France, France
CHRU StrasbourgRecruiting
Strasbourg, Grand Est, France
Medizinische Hochschule Hannover, Abteilung Pneumologie und InfektiologieRecruiting
Hanover, Lower Saxony, Germany
Fondazione IRCCS San Gerardo dei TintoriRecruiting
Monza, Monza and Brianza, Italy
Amsterdam UMC site VUMCRecruiting
Amsterdam, North Holland, Netherlands
Auckland City HospitalRecruiting
Auckland, Auckland, New Zealand
University of Otago ChristchurchRecruiting
Christchurch, Canterbury, New Zealand
Te Whato Ora, Health New Zealand, Waikato HospitalRecruiting
Hamilton, Waikato Region, New Zealand
Unidade Local de Saúde de Santo António - Hospital de Santo AntónioRecruiting
Porto, Porto District, Portugal
Chonnam National University HospitalRecruiting
Gwangju, South Korea
Samsung Medical CenterRecruiting
Seoul, South Korea
Hospital Universitario Virgen de la VictoriaRecruiting
Málaga, Andalusia, Spain
Hospital Clínic de BarcelonaRecruiting
Barcelona, Barcelona, Spain
Hospital Universitario Marqués De ValdecillaRecruiting
Santander, Cantabria, Spain
Hospital Universitari Vall d'HebronRecruiting
Barcelona, Catalonia, Spain
Hospital Universitario Doce de OctubreRecruiting
Madrid, Madrid, Spain
Hospital Universitario la PazRecruiting
Madrid, Madrid, Spain
Hospital Costa del SolRecruiting
Marbella, Malaga, Spain
Hospital Universitario Virgen MacarenaRecruiting
Seville, Sevilla, Spain
Hospital General Universitario de ToledoRecruiting
Toledo, Toledo, Spain
Hospital Universitari i Politecnic La Fe de ValenciaRecruiting
Valencia, Valencia, Spain
Royal Papworth HospitalRecruiting
Cambridge, Cambridgeshire, United Kingdom
Golden Jubilee National HospitalRecruiting
Glasgow, West Dunbartonshire, United Kingdom
Imperial College Healthcare NHS TrustRecruiting
London, United Kingdom