ACT-293987 in Pulmonary Arterial Hypertension

Study Overview
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Brief Summary
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Long-term, single-arm, multicenter, open-label extension, Phase 3 study, to evaluate the safety and tolerability of ACT-293987 in patients with PAH who participated in the double-blind study AC-065A302 (GRIPHON)

Official Title
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Long-term Single-arm Open-label Study, to Assess the Safety and Tolerability of ACT-293987 in Patients With Pulmonary Arterial Hypertension

Conditions
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Pulmonary Arterial Hypertension

Intervention / Treatment
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* Drug: ACT-293987


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients who participated to the double-blind study AC-065A302 and either had a morbidity event or had completed the study as scheduled per protocol. Signed informed consent. Exclusion Criteria: Patients who have started receiving prostacyclin (epoprostenol) or prostacyclin analogs (i.e., treprostinil, iloprost, beraprost) since the last study drug intake in AC-065A302/GRIPHON. Severe hepatic impairment (Child-Pugh C). Females who are pregnant or who plan to become pregnant during the study, or are breastfeeding. Any known factor or disease that might interfere with treatment compliance, study conduct or interpretation of the results, such as drug or alcohol dependence, or psychiatric disease.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 1<br>ACT-293987, twice daily | Drug: ACT-293987<br>* Tablets, twice daily<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) up to 3 Days After Study Intervention Discontinuation | An adverse event (AE) is any untoward medical event that occurs in a participant administered an investigational product, and it does not necessarily indicate only events with clear causal relationship with the relevant investigational product. A TEAE is any AE temporally associated with the use of study drug (from study drug initiation until 3 days after study drug discontinuation), whether or not considered related to the study drug. | Up to 3 days after study drug discontinuation (Up to 10.5 years) | 
| Number of Participants With Treatment-emergent Serious Adverse Events (SAEs) up to 3 Days After Study Intervention Discontinuation | An adverse event is any untoward medical event that occurs in a participant administered an investigational product, and it does not necessarily indicate only events with clear causal relationship with the relevant investigational product. SAE is any AE that results in: death, persistent or significant disability/incapacity, requires inpatient hospitalization or prolongation of existing hospitalization, is life-threatening experience, is a congenital anomaly/birth defect and may jeopardize participant and/or may require medical or surgical intervention to prevent one of the outcomes listed above. Those SAEs occurring during study drug administration, that is, between study drug initiation and three days after study drug discontinuation, are defined as treatment-emergent SAEs. | Up to 3 days after study drug discontinuation (Up to 10.5 years) | 
| Number of Participants With TEAEs Leading to Permanent Discontinuation of Study Intervention | An adverse event (AE) is any untoward medical event that occurs in a participant administered an investigational product, and it does not necessarily indicate only events with clear causal relationship with the relevant investigational product. A TEAE is any AE temporally associated with the use of study drug (from study drug initiation until 3 days after study drug discontinuation), whether or not considered related to the study drug. | Up to 10.5 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Alive Participants | Percentage of alive participants were analyzed using Kaplan-Meier (KM) estimates. | Baseline (Day 1), Months 3, 6, 9, 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Open-label, PAH, Pulmonary Arterial Hypertension