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

NCT07365332 | Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: IKT-001-201
• Study Type: interventional
• Target: 486
• Locations: 14 countries
• Sites: 43

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

Outcome Measures

Primary Outcomes (2)

• [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

  Change in Pulmonary Vascular Resistance (PVR)

  Baseline to Week 24

• [Part B] To characterize the effects of IKT-001 on symptoms and characteristics of Pulmonary Arterial Hypertension compared to placebo

  Change in 6-minute walk distance (6MWD)

  Baseline to Week 24

Secondary Outcomes (2)

• To characterize the effects of IKT-001 on WHO Functional Class

  Baseline up to Week 48

• To characterize the effects of IKT-001 on time to clinical worsening

  Baseline up to Week 48

Study Arms (2)

IKT-001

EXPERIMENTAL

IKT-001 tablets for PO administration

Drug: IKT-001

Placebo

PLACEBO COMPARATOR

Matching placebo to IKT-001 tablets for PO administration

Drug: Placebo

Interventions

Placebo DRUG

Placebo to IKT-001 tablets for PO administration

Placebo

IKT-001 DRUG

IKT-001 tablets for PO administration

IKT-001

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• MWD ≥ 100 and ≤ 475 m

You may not qualify if:

• 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:

Sponsors & Collaborators

• Inhibikase Therapeutics: lead

Study Sites (43)

Arizona Pulmonary Specialists, LTD

Scottsdale, Arizona, 85258, United States

RECRUITING

Cedars Sinai

Beverly Hills, California, 90211, United States

RECRUITING

Jeffrey S. Sager, MD Medical Corporation

Santa Barbara, California, 93105, United States

RECRUITING

Lundquist at Harbor UCLA

Torrance, California, 90502, United States

RECRUITING

University of Colorado Anschutz Medical Campus

Aurora, Colorado, 80045, United States

RECRUITING

University of Kansas Medical Center

Kansas City, Kansas, 66103, United States

RECRUITING

Norton Healthcare

Louisville, Kentucky, 40202, United States

RECRUITING

Tufts Medical Center

Boston, Massachusetts, 02111, United States

RECRUITING

Washington University School of Medicine

St Louis, Missouri, 63110, United States

RECRUITING

NYU Langone Health

New York, New York, 10016, United States

RECRUITING

Westchester Medical Center

Valhalla, New York, 10595, United States

RECRUITING

Duke University

Durham, North Carolina, 27710, United States

RECRUITING

University of Cincinnati

Cincinnati, Ohio, 45267, United States

RECRUITING

Vanderbilt University Medical Center

Nashville, Tennessee, 37232-7610, United States

RECRUITING

UT Southwestern Medical Center

Dallas, Texas, 75390, United States

RECRUITING

Sanatorio de la Trinidad Palermo

Buenos Aires, Buenos Aires, 1425, Argentina

RECRUITING

Pulmonary Hypertension Research Queensland PHRQ

Auchenflower, Queensland, 4066, Australia

RECRUITING

Hôpital Universitaire de Bruxelles - Erasme

Anderlecht, Brussels Capital, 1070, Belgium

RECRUITING

University of Alberta Hospital

Edmonton, Alberta, T6G 2B7, Canada

RECRUITING

Hôpital Bicêtre

Le Kremlin-Bicêtre, France, 94270, France

RECRUITING

CHRU Strasbourg

Strasbourg, Grand Est, 67091, France

RECRUITING

Medizinische Hochschule Hannover, Abteilung Pneumologie und Infektiologie

Hanover, Lower Saxony, 30625, Germany

RECRUITING

Fondazione IRCCS San Gerardo dei Tintori

Monza, Monza and Brianza, 20900, Italy

RECRUITING

Amsterdam UMC site VUMC

Amsterdam, North Holland, 1081 BT, Netherlands

RECRUITING

Auckland City Hospital

Auckland, Auckland, 1051, New Zealand

RECRUITING

University of Otago Christchurch

Christchurch, Canterbury, 8011, New Zealand

RECRUITING

Te Whato Ora, Health New Zealand, Waikato Hospital

Hamilton, Waikato Region, 3204, New Zealand

RECRUITING

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

Porto, Porto District, 4099-001, Portugal

RECRUITING

Chonnam National University Hospital

Gwangju, 61469, South Korea

RECRUITING

Samsung Medical Center

Seoul, 06351, South Korea

RECRUITING

Hospital Universitario Virgen de la Victoria

Málaga, Andalusia, 29010, Spain

RECRUITING

Hospital Clínic de Barcelona

Barcelona, Barcelona, 08036, Spain

RECRUITING

Hospital Universitario Marqués De Valdecilla

Santander, Cantabria, 39008, Spain

RECRUITING

Hospital Universitari Vall d'Hebron

Barcelona, Catalonia, 08035, Spain

RECRUITING

Hospital Universitario Doce de Octubre

Madrid, Madrid, 28041, Spain

RECRUITING

Hospital Universitario la Paz

Madrid, Madrid, 28046, Spain

RECRUITING

Hospital Costa del Sol

Marbella, Malaga, 29603, Spain

RECRUITING

Hospital Universitario Virgen Macarena

Seville, Sevilla, 41009, Spain

RECRUITING

Hospital General Universitario de Toledo

Toledo, Toledo, 45007, Spain

RECRUITING

Hospital Universitari i Politecnic La Fe de Valencia

Valencia, Valencia, 46026, Spain

RECRUITING

Royal Papworth Hospital

Cambridge, Cambridgeshire, CB2 0AY, United Kingdom

RECRUITING

Golden Jubilee National Hospital

Glasgow, West Dunbartonshire, G81 4DY, United Kingdom

RECRUITING

Imperial College Healthcare NHS Trust

London, W12 0HS, United Kingdom

RECRUITING

MeSH Terms

Conditions

Pulmonary Arterial Hypertension

Condition Hierarchy (Ancestors)

Hypertension, Pulmonary; Lung Diseases; Respiratory Tract Diseases

Central Study Contacts

Medical Director, Inhibikase Therapeutics

CONTACT

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

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Adaptive, 2-part, randomized, multicenter, double-blind, placebo-controlled, parallel-group study

Study Record Dates

• First Submitted: January 22, 2026
• First Posted: January 26, 2026
• Study Start: April 23, 2026
• Primary Completion (Estimated): June 1, 2029
• Study Completion (Estimated): December 1, 2029
• Last Updated: September 2, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share

Locations

NZ (3); GB (3); AU (1); FR (2); CA (1); AR (1); NL (1); DE (1); IT (1); PT (1); KR (2); BE (1); ES (10); US (15)