NCT06643143

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

A Randomized, Double-Blind, Placebo-Controlled Trial of IkT-001Pro in Pulmonary Arterial Hypertension

• Status: Withdrawn
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Inhibikase Therapeutics · Industry
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

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).

Detailed description

This is a randomized, double-blind, multi-center, placebo-controlled dose-ranging clinical trial of two IkT-001Pro doses in patients with PAH. This study 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). Those participants that pass the screening process will be randomized during the baseline visit to either the low dose (300mg) or placebo in a 2:1 ratio. After two weeks participants will return to the clinic; upon confirmation that the participants are tolerating their dose, those that are on active treatment will be randomized to either 300mg or 500mg active treatment arms in a 1:1 manner. Those participants on placebo will remain on placebo for the rest of the 26-week placebo controlled treatment period. The final randomization structure for the 26-week placebo controlled treatment period will be such that participants are randomized in a 1:1:1 scheme to the 300 mg, 500 mg or placebo groups. Participants who have not discontinued early will transition to a 36 month extension period. Participants who transition to the extension period will remain on the dose that they were assigned to after the two week acclimation period. Participants who were randomized to the placebo group will be re-randomized 1:1 to either the 300mg or 500mg IkT-001Pro treatment groups. The study will be unblinded and investigators will be given treatment assignments once the primary endpoint analysis is completed.

Conditions

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

Interventions

Type	Name	Description
DRUG	IkT-001Pro	IkT-001Pro
DRUG	Placebo	Placebo

Timeline

• Start date: 2025-06-30
• Primary completion: 2027-06-30
• Completion: 2027-06-30
• First posted: 2024-10-16
• Last updated: 2025-06-13

Regulatory

• FDA-regulated drug study