A Randomized, Double-Blind, Placebo-Controlled Trial of IkT-001Pro in Pulmonary Arterial Hypertension
Overview
- Phase
- Phase 2
- Intervention
- Placebo
- Conditions
- Pulmonary Arterial Hypertension (PAH)
- Sponsor
- Inhibikase Therapeutics
- Enrollment
- 150
- Primary Endpoint
- To evaluate the effect on PVR in participants with WHO functional class II-III PAH treated with IkT-001Pro compare to placebo
- Status
- Withdrawn
- Last Updated
- 10 months ago
Overview
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).
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.
Investigators
Eligibility Criteria
Inclusion Criteria
- •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
Exclusion Criteria
- •Diagnosis of PAH WHO Groups 2, 3, 4, or 5
- •Diagnosis of the following PAH Group 1 subtypes: human immunodeficiency virus (HIV)-associated PAH and PAH associated with portal hypertension. Exclusions in PAH Group 1 should also include schistosomiasis-associated PAH and pulmonary veno-occlusive disease
- •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
- •Pulmonary function tests (PFT) completed no more than 24 weeks before the screening period (or completed at the Screening Visit). Following criteria must are considered exclusionary
- •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
- •Currently receiving or anticipated need to receive more than one anti-platelet medication. The use of prostacyclins is not considered exclusionary. Note that sotatercept is not considered anti-platelet therapy
- •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)
Arms & Interventions
Placebo Control
Placebo
Intervention: Placebo
300mg
300mg
Intervention: IkT-001Pro
500mg
500mg
Intervention: IkT-001Pro
Outcomes
Primary Outcomes
To evaluate the effect on PVR in participants with WHO functional class II-III PAH treated with IkT-001Pro compare to placebo
Time Frame: Through study completion, an average of 26 weeks with 36 months of extension
Change in PVR at 26 weeks compared to baseline
To assess the safety and tolerability of two IkT-001Pro doses in PAH
Time Frame: Through study completion, an average of 26 weeks with 36 months of extension
Incidence and temporal profile of treatment-emergent adverse events (TEAEs) evaluated by type/nature, severity/intensity, seriousness, and relationship to study intervention
Secondary Outcomes
- 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)
- 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)