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ACT-293987

Phase 3

Pulmonary Arterial Hypertension | Unknown | Cardiovascular |Johnson & Johnson|Last Updated: Sep 12, 2025

Success Probability

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Market & Valuation

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Trial Design

RandomizedUNCONTROLLEDDMC
Total Trials2
Total Enrollment752

FDA Designations

No designations recorded

Clinical trial landscape

ACT-293987 · 2 trials · 1 indication

Phase 3 1Phase 2 1
NCT01112306ACT-293987 in Pulmonary Arterial HypertensionPulmonary Arterial Hypertension
COMPLETED709 Analytics
PHASE3COMPLETED
ACT-293987 in Pulmonary Arterial Hypertension
Pulmonary Arterial HypertensionUnlock trial analytics

Study Endpoints

Primary Endpoints

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 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 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.

Assessment of hemodynamic parameters after single oral dose of ACT-293987.
17 weeks
Proof-of-concept assessment of the efficacy of ACT-293987 in subjects with PAH by measuring the change from baseline in the PVR at Week 17 compared to placebo.
17 weeks

Secondary Endpoints

Percentage of Alive Participants
Baseline (Day 1), Months 3, 6, 9, 12, 24, 36, 48, 60, 72, 84, 96, 108, and 120
Safety and tolerability of a single oral dose of ACT-293987.
17 weeks
Assessments of preliminary efficacy of ACT-293987 regarding 6 minute walk test (6MWT), proportion of subjects with aggravation of PAH, hemodynamic parameters other than PVR
17 weeks
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
1EXPERIMENTALACT-293987, twice daily
ACT-293987 (NS-304) and matching placeboEXPERIMENTALSubjects will be randomized to the study following screening. Each subject will then undergo an acute hemodynamic study with right heart catheterization after a single oral administration of ACT-293987 (NS-304)on Day 0. The objectives are to collect data about the drug effect on the right heart hemodynamic parameters (PVR, calculated SVR and PVR/SVR) measured by right heart catheterization after single oral dose administration of NS-304 and to assess the safety and tolerability of a single oral dose of NS-304.

Interventions

NameTypeDescription
ACT-293987DRUGTablets, twice daily
ACT-293987 (NS-304)DEVICE -
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites157

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 prostacyc...

Countries:United StatesArgentinaAustraliaAustriaBelarusBelgiumCanadaChileChinaColombiaCzechiaDenmarkFranceGermanyGreeceHungaryIndiaIrelandIsraelMalaysiaMexicoNetherlandsPeruPolandRomaniaRussiaSerbiaSingaporeSlovakiaSouth KoreaSpainSwedenSwitzerlandTaiwanThailandTurkey (Türkiye)UkraineUnited KingdomItaly
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Frequently asked questions about ACT-293987

What is ACT-293987 used for?

ACT-293987 is an investigational drug being studied for the treatment of Pulmonary Arterial Hypertension (PAH), a type of high blood pressure affecting the arteries in the lungs and the right side of the heart. It is being developed by Johnson & Johnson (JNJ) and is currently in Phase 3 clinical development.

Who makes ACT-293987?

ACT-293987 is being developed by Johnson & Johnson, a multinational pharmaceutical company traded on the New York Stock Exchange under the ticker JNJ. The drug is currently in Phase 3 clinical trials for the treatment of Pulmonary Arterial Hypertension.

What phase is ACT-293987 in?

ACT-293987 is in Phase 3 clinical development for Pulmonary Arterial Hypertension. It is an investigational drug, meaning it has not been approved by regulatory authorities and is still undergoing clinical trials to evaluate its safety and efficacy. Two clinical trials have been completed, including a Phase 3 study.

What clinical trials is ACT-293987 in?

ACT-293987 has been studied in two completed clinical trials. NCT00993408 was a Phase 2 study with 43 participants, and NCT01112306 was a Phase 3 study with 709 participants. Both trials focused on Pulmonary Arterial Hypertension and enrolled adults aged 18 years and older.

Is ACT-293987 the same as NS-304?

ACT-293987 is also known as NS-304, as indicated in the title of the Phase 2 clinical trial NCT00993408, which refers to the drug as ACT-293987 (NS-304). Both names refer to the same investigational compound being developed for Pulmonary Arterial Hypertension.