Actively Recruiting
An Adaptive, Randomized, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of IKT-001 in Adults with Pulmonary Arterial Hypertension
Led by Inhibikase Therapeutics · Updated on 2026-05-12
486
Participants Needed
2
Research Sites
26 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the efficacy and safety of a drug called IKT-001 in adults with Pulmonary Arterial Hypertension (PAH), specifically those classified as WHO Group 1. This adaptive, randomized, double-blind, placebo-controlled study aims to compare IKT-001 with placebo in a controlled setting to better understand its effects on this serious lung condition. Participants will be randomly assigned to receive either IKT-001 tablets or matching placebo tablets taken by mouth. The study is divided into two parts: Part A focuses on measuring changes in Pulmonary Vascular Resistance over 24 weeks, and Part B looks at the effects of IKT-001 on symptoms and characteristics of PAH over the same period. There is also monitoring of WHO Functional Class and time to clinical worsening up to 48 weeks. During the study, participants will undergo various assessments including hemodynamic measurements through right heart catheterization at screening, physical tests like the 6-minute walk test, and monitoring of clinical symptoms and safety parameters. The study involves regular visits over at least 24 to 48 weeks to collect data on lung and heart function, symptoms, and overall health. The total participation may last up to several months, allowing researchers to thoroughly evaluate the impact of IKT-001 compared to placebo.
CONDITIONS
Brief Title
An Adaptive Program of IKT-001 in Pulmonary Arterial Hypertension (PAH)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Documented diagnosis of WHO Group 1 Pulmonary Arterial Hypertension, including idiopathic, heritable, drug/toxin-induced, connective tissue disease-associated, or congenital shunt-associated PAH at least 1 year post-repair
- Men and women aged 18 to 75 years inclusive
- Body mass index (BMI) between 18.5 and 35.0 kg/m² at screening
- Baseline right heart catheterization showing pulmonary vascular resistance ≥400 dyn/sec/cm⁵, pulmonary capillary wedge pressure ≤15 mmHg, and mean pulmonary artery pressure >20 mmHg, with enrichment criteria for PVR >700 dyn/sec/cm⁵
- Stable background PAH therapy for at least 90 days prior to screening, excluding current use of sotatercept
- Six-minute walk distance between 100 and 475 meters
You will not qualify if you...
- Diagnosis of PAH WHO Groups 2, 3, 4, or 5
- PAH Group 1 subtypes including HIV-associated PAH, portal hypertension-associated PAH, schistosomiasis-associated PAH, and pulmonary veno-occlusive disease
- Uncontrolled hypertension (systolic BP >160 mmHg or diastolic BP >100 mmHg), systolic BP <90 mmHg at screening, or syncope within 3 months prior to screening
- History of restrictive, constrictive, or congestive cardiomyopathy
- ECG with prolonged QT interval (QTcF ≥450 ms in males, ≥470 ms in females, or ≥500 ms with right bundle branch block)
- Personal or family history of long QT syndrome or sudden cardiac death
- Presence of CardioMEMS or other implanted hemodynamic monitoring devices
- Forced vital capacity <70% on pulmonary function test, or 60-69% with more than mild interstitial lung disease on chest CT
- History of atrial fibrillation or flutter
- History of cerebrovascular accident, intracranial hemorrhage, subdural hematoma, or recent head trauma within 3 months
- Acutely decompensated right heart failure within 30 days prior to screening
- Significant ischemic, valvular, constrictive heart disease or heart failure with preserved ejection fraction
- History of pneumonectomy
- Untreated or inadequately treated obstructive sleep apnea
- Acute or chronic hepatitis B or C infection
- History or current diagnosis of bleeding disorders
- Current use of strong CYP3A inducers or inhibitors (except topical), anticoagulants, or sotatercept within 6 months
- Recent initiation or planned start of cardiopulmonary rehabilitation exercise program
- History of atrial septostomy within 180 days
- Current participation in another investigational trial or prior IKT-001 study participation
- Social, behavioral, or medical reasons preventing study completion
- Currently pregnant, lactating, or planning pregnancy during the study
- Prior solid organ or stem cell transplant
- Planned surgery interfering with study drug or assessments
- Malignancy within 5 years except certain treated non-metastatic cancers
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 24 weeks
Participants receive either IKT-001 tablets or matching placebo to evaluate their effects on Pulmonary Arterial Hypertension.
Regular visits during treatment period
Duration - Up to 24 weeks
Participants are monitored for up to 24 additional weeks after treatment to assess long-term effects and safety.
Scheduled follow-up visits
Trial Site Locations
Total: 2 locations
1
Norton Healthcare
Louisville, Kentucky, United States, 40202
Actively Recruiting
2
Tufts Medical Center
Boston, Massachusetts, United States, 02111
Actively Recruiting
Research Team
M
Medical Director, Inhibikase Therapeutics
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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