A Phase 2 Study of IkT-001Pro in Pulmonary Arterial Hypertension (PAH)

NCT06643143 | Withdrawn Phase 2 DMC FDA Drug

• 1 other identifier: IkT-001Pro-702
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

This is a randomized, double-blind, multi-center, placebo-controlled dose-ranging clinical trial of two IkT-001Pro doses in patients with PAH designed to assess safety, tolerability and efficacy. It will enroll approximately 150 participants at up to 50 sites globally. The study consists of two parts, a 26 week placebo controlled treatment period (Part A) followed by a 36 month extension period (Part B).

Conditions

Pulmonary Arterial Hypertension (PAH); Pulmonary Arterial Hypertension (PAH) (WHO Group 1 PH)

Keywords

Pulmonary Arterial Hypertension; PAH

Outcome Measures

Primary Outcomes (2)

• To evaluate the effect on PVR in participants with WHO functional class II-III PAH treated with IkT-001Pro compare to placebo

  Change in PVR at 26 weeks compared to baseline

  Through study completion, an average of 26 weeks with 36 months of extension

• To assess the safety and tolerability of two IkT-001Pro doses in PAH

  Incidence and temporal profile of treatment-emergent adverse events (TEAEs) evaluated by type/nature, severity/intensity, seriousness, and relationship to study intervention

  Through study completion, an average of 26 weeks with 36 months of extension

Secondary Outcomes (8)

• To characterize the effects of two IkT-001Pro doses on symptoms and characterics of Pulmonary Arterial Hypertension

  Through study completion, an average of 26 weeks with 36 months of extension

• To characterize the effects of two IkT-001Pro doses on symptoms and characterics of Pulmonary Arterial Hypertension

  Through study completion, an average of 26 weeks with 36 months of extension

• To characterize the effects of two IkT-001Pro doses on symptoms and characterics of Pulmonary Arterial Hypertension

  Through study completion, an average of 26 weeks with 36 months of extension

• To assess the PK of IkT-001Pro in participants with PAH

  Through study completion, an average of 26 weeks with 36 months of extension

• Part B : To assess the long-term safety and tolerability of two IkT-001Pro doses in PAH

  Through study completion, an average of 26 weeks with 36 months of extension

Study Arms (3)

Placebo Control

EXPERIMENTAL

Placebo

Drug: Placebo

300mg

EXPERIMENTAL

300mg

Drug: IkT-001Pro

500mg

EXPERIMENTAL

500mg

Drug: IkT-001Pro

Interventions

IkT-001Pro DRUG

IkT-001Pro

300mg; 500mg

Placebo DRUG

Placebo

Placebo Control

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Men and women between the ages of 18 and 70 years of age (inclusive) at the time of signing the informed consent.
• Capable of giving signed ICF.
• Documented diagnostic right heart catheterization (RHC) at any time prior to screening confirming the diagnosis of WHO PAH Group 1 in any of the following subtypes:
• Idiopathic PAH
• Heritable PAH
• Drug/toxin-induced PAH
• PAH associated with CTD
• PAH associated with simple, congenital systemic-to-pulmonary shunts at least 1 year following repair
• Symptomatic PH classified as WHO FC II or III
• Baseline RHC performed during the Screening Period documenting a minimum PVR of
• ≥ 400 dyn.sec.cm-5 (≥5 WU) and a pulmonary capillary wedge pressure (PCWP) or left ventricular end-diastolic pressure of ≤ 15 mmHg.
• a. NOTE: At least 50% of study population needs to have PVR ≥ 800 dyn.sec.cm-5.
• On stable doses of background PAH therapy (i.e., patient-specific dose goal for each therapy already achieved) for at least 90 days prior to screening; for infusion prostacyclins, dose adjustment within 10% of optimal dose is allowed per medical practice.
• a. For those taking sotatercept their dosing regimen should be stable for at least 6 months prior to screening
• MWD ≥ 150 and ≤ 500 m repeated twice at screening (measured at least 4 hours apart, but no longer than 1 week), and both values are within 15% of each other (calculated from the highest value).

You may not qualify if:

• Diagnosis of PAH WHO Groups 2, 3, 4, or 5
• Uncontrolled systemic hypertension as evidenced by sitting systolic BP \> 160 mmHg or sitting diastolic BP \> 100 mmHg during screening visit after a period of rest
• Personal or family history of long QT syndrome (LQTS) or sudden cardiac death
• Cerebrovascular accident within 3 months prior to the screening visit
• Currently receiving moderate or strong Cytochrome P450 (CYP) 3A4/5 inducers or CYP3A4/5 inhibitors (except for topical administration)
• Currently receiving or anticipated need to receive anticoagulants
• Initiation of an exercise program for cardiopulmonary rehabilitation within 90 days prior to the screening visit 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 the screening visit
• Current participation in another investigational clinical trial and/or receipt of any investigational medication within 90 days prior to screening
• Estimated glomerular filtration rate (eGFR) \< 30 mL/min/m2 (as defined by the Modification of Diet in Renal Disease \[MDRD\] equation)

Sponsors & Collaborators

• Inhibikase Therapeutics: lead

MeSH Terms

Conditions

Pulmonary Arterial Hypertension

Condition Hierarchy (Ancestors)

Hypertension, Pulmonary; Lung Diseases; Respiratory Tract Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 9, 2024
• First Posted: October 16, 2024
• Study Start: June 30, 2025
• Primary Completion (Estimated): June 30, 2027
• Study Completion (Estimated): June 30, 2027
• Last Updated: June 13, 2025

Record last verified: 2025-06