Pulmonary Arterial Hypertension (PAH)Phase 2NCT06643143
A Phase 2 Study of IkT-001Pro in Pulmonary Arterial Hypertension (PAH)
A Phase 2 study testing IkT-001Pro in people with Pulmonary Arterial Hypertension (PAH) and Pulmonary Arterial Hypertension (PAH) (WHO Group 1 PH), run by Inhibikase Therapeutics.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Pulmonary Arterial Hypertension (PAH), Pulmonary Arterial Hypertension (PAH) (WHO Group 1 PH)
- What is being tested?
- IkT-001Pro
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 30 Jun 2025to 30 Jun 2027
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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.
- 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.
- For those taking sotatercept their dosing regimen should be stable for at least 6 months prior to screening
- 6MWD ≥ 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).
Who cannot join
- 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
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- 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)
- 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)
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled Trial of IkT-001Pro in Pulmonary Arterial Hypertension
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).
Read the 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)
- Being tested
- IkT-001Pro, Placebo
- Keywords
- Pulmonary Arterial Hypertension, PAH
- Medical subject headings
- Pulmonary Arterial Hypertension
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IkT-001ProDrug
IkT-001Pro
PlaceboDrug
Placebo
| Study group | Role | What they receive |
|---|---|---|
| Placebo Control | Experimental | Placebo Receives: Drug: Placebo |
| 300mg | Experimental | 300mg Receives: Drug: IkT-001Pro |
| 500mg | Experimental | 500mg Receives: Drug: IkT-001Pro |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
Measured: 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
Change from Baseline to Week 26 in 6 minute walk test.
Measured: 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
Time to death or the first occurrence of any of the following clinical worsening events; Lung or heart-lung transplantation, Atrial septostomy, Hospitalization for worsening of PAH (≥ 24 hours) or Deterioration of PAH defined by both of the following events occurring at any time, even if they began at different times, as compared to their baseline values.
Measured: 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
Number of participants who improve in WHO FC or who maintain or achieve a low risk score at Week 26 versus baseline using the ESC/ERS 4-strata model.
Measured: Through study completion, an average of 26 weeks with 36 months of extension
To assess the PK of IkT-001Pro in participants with PAH
PK parameters of IkT-001Pro
Measured: 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
Incidence and temporal profile of treatment-emergent adverse events (TEAEs) evaluated by type/nature, severity/intensity, seriousness, and relationship to study intervention
Measured: 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
Change from Baseline to Week 26 in the Physical Impacts domain score of Pulmonary Arterial Hypertension-Symptoms and Impact (PAH-SYMPACT®)
Measured: 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
Change from Baseline to Week 26 in the Cardiopulmonary Symptoms domain score of Pulmonary Arterial Hypertension-Symptoms and Impact (PAH-SYMPACT®)
Measured: 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
Change from Baseline to Week 26 in the Cognitive/Emotional Impacts domain score of Pulmonary Arterial Hypertension-Symptoms and Impact (PAH-SYMPACT®)
Measured: Through study completion, an average of 26 weeks with 36 months of extension
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Oct 2024
- Study start30 Jun 2025 (estimated)
- Primary completion30 Jun 2027 (estimated)
- Study completion30 Jun 2027 (estimated)
Why it stopped: Study has undergone sponsorship and design changes.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pulmonary Arterial Hypertension (PAH)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Inhibikase Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06643143
- Sponsor's protocol ID
- IkT-001Pro-702
- Status last verified
- Jun 2025
- First submitted
- 9 Oct 2024
- Last updated
- 13 Jun 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled Trial of IkT-001Pro in Pulmonary Arterial Hypertension
- ClinicalTrials.gov ID
- NCT06643143
- Acronym / sponsor ID
- IkT-001Pro-702
- Conditions
- Pulmonary Arterial Hypertension (PAH), Pulmonary Arterial Hypertension (PAH) (WHO Group 1 PH)
- Keywords
- Pulmonary Arterial Hypertension, PAH
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Study has undergone sponsorship and design changes.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Inhibikase TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 30 Jun 2025 (estimated)
- Primary completion
- 30 Jun 2027 (estimated)
- Study completion
- 30 Jun 2027 (estimated)
- First posted
- 16 Oct 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Placebo Control | Experimental | Drug: PlaceboPlacebo |
| 300mg | Experimental | Drug: IkT-001Pro300mg |
| 500mg | Experimental | Drug: IkT-001Pro500mg |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- 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:
- Symptomatic PH classified as WHO FC II or III
- Baseline RHC performed during the Screening Period documenting a minimum PVR of
Key exclusion criteria (13 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| IkT-001ProIkT-001Pro | Drug | 300mg, 500mg |
| PlaceboPlacebo | Drug | Placebo Control |
Primary outcome
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
8 secondary outcomes
To characterize the effects of two IkT-001Pro doses on symptoms and characterics of Pulmonary Arterial Hypertension
Time frame: 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
Time frame: 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
Time frame: Through study completion, an average of 26 weeks with 36 months of extension
To assess the PK of IkT-001Pro in participants with PAH
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: Through study completion, an average of 26 weeks with 36 months of extension
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Jun 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing IkT-001Pro in people with Pulmonary Arterial Hypertension (PAH) and Pulmonary Arterial Hypertension (PAH) (WHO Group 1 PH), run by Inhibikase Therapeutics.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 15 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 13 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Jun 2025.