ACT-293987 in Pulmonary Arterial Hypertension

Phase 3

Completed

• Conditions: Pulmonary Arterial Hypertension
• Interventions: Drug: ACT-293987

• Registration Number: NCT01112306
• Lead Sponsor: Actelion

Brief Summary

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)

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2010-04-08
• Study First Submitted QC: 2010-04-27
• Study First Posted: 2010-04-28
• Study Start: 2010-07-07
• Primary Completion: 2021-08-26
• Study Completion: 2021-08-26
• Results First Submitted: 2022-08-24
• Results First Submitted QC: 2022-08-24
• Results First Posted: 2022-09-19
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-28
• Last Update Posted: 2025-03-30

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 709

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
1	ACT-293987	ACT-293987, twice daily

Primary Outcome Measures

Name	Time	Method
Number of Participants With Treatment-emergent Serious Adverse Events (SAEs) up to 3 Days After Study Intervention Discontinuation	Up to 3 days after study drug discontinuation (Up to 10.5 years)	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.
Number of Participants With Treatment-emergent Adverse Events (TEAEs) up to 3 Days After Study Intervention Discontinuation	Up to 3 days after study drug discontinuation (Up to 10.5 years)	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.
Number of Participants With TEAEs Leading to Permanent Discontinuation of Study Intervention	Up to 10.5 years	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.

Secondary Outcome Measures

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