Placebo: Placebo to IKT-001 tablets for PO administration
Study 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
Sex
ALL
Min age
18 Years
Max age
75 Years
Healthy volunteers
No
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.
Primary outcome measure(s)
[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 Change in Pulmonary Vascular Resistance (PVR)
[Part B] To characterize the effects of IKT-001 on symptoms and characteristics of Pulmonary Arterial Hypertension compared to placebo — Baseline to Week 24 Change in 6-minute walk distance (6MWD)
Trial sites (43)
Facility
City
Region
Status
Arizona Pulmonary Specialists, LTD
Scottsdale
Arizona
Recruiting
Cedars Sinai
Beverly Hills
California
Recruiting
Jeffrey S. Sager, MD Medical Corporation
Santa Barbara
California
Recruiting
Lundquist at Harbor UCLA
Torrance
California
Recruiting
University of Colorado Anschutz Medical Campus
Aurora
Colorado
Recruiting
University of Kansas Medical Center
Kansas City
Kansas
Recruiting
Norton Healthcare
Louisville
Kentucky
Recruiting
Tufts Medical Center
Boston
Massachusetts
Recruiting
Washington University School of Medicine
St Louis
Missouri
Recruiting
NYU Langone Health
New York
New York
Recruiting
Westchester Medical Center
Valhalla
New York
Recruiting
Duke University
Durham
North Carolina
Recruiting
University of Cincinnati
Cincinnati
Ohio
Recruiting
Vanderbilt University Medical Center
Nashville
Tennessee
Recruiting
UT Southwestern Medical Center
Dallas
Texas
Recruiting
Sanatorio de la Trinidad Palermo
Buenos Aires
Buenos Aires
Recruiting
Pulmonary Hypertension Research Queensland PHRQ
Auchenflower
Queensland
Recruiting
Hôpital Universitaire de Bruxelles - Erasme
Anderlecht
Brussels Capital
Recruiting
University of Alberta Hospital
Edmonton
Alberta
Recruiting
Hôpital Bicêtre
Le Kremlin-Bicêtre
France
Recruiting
CHRU Strasbourg
Strasbourg
Grand Est
Recruiting
Medizinische Hochschule Hannover, Abteilung Pneumologie und Infektiologie
Hanover
Lower Saxony
Recruiting
Fondazione IRCCS San Gerardo dei Tintori
Monza
Monza and Brianza
Recruiting
Amsterdam UMC site VUMC
Amsterdam
North Holland
Recruiting
Auckland City Hospital
Auckland
Auckland
Recruiting
University of Otago Christchurch
Christchurch
Canterbury
Recruiting
Te Whato Ora, Health New Zealand, Waikato Hospital
Hamilton
Waikato Region
Recruiting
Unidade Local de Saúde de Santo António - Hospital de Santo António
Porto
Porto District
Recruiting
Chonnam National University Hospital
Gwangju
South Korea
Recruiting
Samsung Medical Center
Seoul
South Korea
Recruiting
Hospital Universitario Virgen de la Victoria
Málaga
Andalusia
Recruiting
Hospital Clínic de Barcelona
Barcelona
Barcelona
Recruiting
Hospital Universitario Marqués De Valdecilla
Santander
Cantabria
Recruiting
Hospital Universitari Vall d'Hebron
Barcelona
Catalonia
Recruiting
Hospital Universitario Doce de Octubre
Madrid
Madrid
Recruiting
Hospital Universitario la Paz
Madrid
Madrid
Recruiting
Hospital Costa del Sol
Marbella
Malaga
Recruiting
Hospital Universitario Virgen Macarena
Seville
Sevilla
Recruiting
Hospital General Universitario de Toledo
Toledo
Toledo
Recruiting
Hospital Universitari i Politecnic La Fe de Valencia
Valencia
Valencia
Recruiting
+ 3 more sites — see the full list on the official registry below.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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