Open-Label Study of IKT-001 in Pulmonary Arterial Hypertension (PAH)

Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability of IKT-001 in Pulmonary Arterial Hypertension

This is a multicenter, open-label extension (OLE), single-arm study designed to assess the long-term safety and tolerability of oral IKT-001 administered once daily.

Study Overview

• Status: Not yet recruiting
• Conditions: Pulmonary Arterial Hypertension (PAH)
• Intervention / Treatment: Drug: IKT-001

• Study Type: Interventional
• Enrollment (Estimated): 484
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Medical Director, Inhibikase Therapeutics; Phone Number: 1-302-295-3800; Email: info@inhibikase.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Capable of giving signed informed consent.
• Participants who have completed Study IKT-001-201 or any other Phase 2 or 3 controlled clinical study with IKT-001.

Exclusion Criteria:

• Any of the following clinical laboratory values at screening:

  • ALT or AST levels >3× the ULN
  • Bilirubin levels >2× the ULN
  • ANC <1.2 ×10^9 cells/L, hemoglobin <9 g/dL, hematocrit <30%, or platelets <75 × 10^9 cells/L
  • Absolute eGFR <30 mL/min using creatinine or cystatin C as defined by the CKD-EPI equation (2021)
• Currently lactating, pregnant or planning on becoming pregnant during the study.
• Receiving treatment with sotatercept.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: IKT-001; IKT-001 tablets for PO administration	Drug: IKT-001; IKT-001 tablets for PO administration

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of Treatment-emergent adverse events (TEAEs), serious TEAEs, and TEAEs of special interest	The number of participants who experience a TEAE, serious TEAE or TEAE of special interest will be reported.	From baseline to end of study (up to 2 years or availability of commercial product)

Secondary Outcome Measures

Outcome Measure	Time Frame
Change from baseline in 6-minute walk distance (6MWD)	From baseline to end of study (up to 2 years or availability of commercial product)
Change from baseline in NT-proBNP	From baseline to end of study (up to 2 years or availability of commercial product)

Collaborators and Investigators

• Sponsor: Inhibikase Therapeutics

Study record dates

Study Major Dates

• Study Start (Estimated): October 23, 2026
• Primary Completion (Estimated): June 30, 2030
• Study Completion (Estimated): December 30, 2030

Study Registration Dates

• First Submitted: May 26, 2026
• First Submitted That Met QC Criteria: June 2, 2026
• First Posted (Actual): June 8, 2026

Study Record Updates

• Last Update Posted (Actual): June 8, 2026
• Last Update Submitted That Met QC Criteria: June 2, 2026
• Last Verified: June 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Respiratory Tract Diseases; Lung Diseases; Hypertension, Pulmonary; Pulmonary Arterial Hypertension
• Other Study ID Numbers: IKT-001-202

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No