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

NCT07365332 | Not Yet Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: IKT-001-201
• Study Type: interventional
• Target: 486
• Locations: 0 countries
• Sites: N/A

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

IKT-001 DRUG

IKT-001 tablets for PO administration

IKT-001

Placebo DRUG

Placebo to IKT-001 tablets for PO administration

Placebo

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

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: March 1, 2026
• Primary Completion (Estimated): June 1, 2029
• Study Completion (Estimated): December 1, 2029
• Last Updated: January 27, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share