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Treprostinil Diolamine

Phase 3

Pulmonary Arterial Hypertension | Small molecule | Cardiovascular |United Therapeutics Corporation|Last Updated: Feb 13, 2020

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment690
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01560624Phase III Clinical Worsening Study of UT-15C in Subjects With PAH Receiving Background Oral MonotherapyPHASE3 COMPLETED 690Jun 26, 2012Jun 24, 2018Feb 13, 2020155 United States, Argentina +21
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Study Endpoints
Primary Endpoints
Time to First Clinical Worsening Event
From randomization to approximately 4 years

Clinical worsening was assessed continuously from randomization until the subject's last study visit. Clinical worsening events were defined as death (all causes), hospitalizations due to worsening pulmonary arterial hypertension (PAH), initiation of an inhaled or infused prostacyclin (PGI2) for the treatment of worsening PAH, disease progression, or unsatisfactory long-term clinical response. All clinical worsening events reported by the study sites were reviewed by the Sponsor Medical Monitors. Once a clinical worsening event occurred, it was entered in the eCRF and a narrative was submitted for review by the Sponsor's Medical Monitor within 48 hours after the event became known to the Investigator or designee. Subsequently, the narratives for subjects with the reported clinical worsening events were sent to an independent adjudication committee. The independent adjudication committee reviewed and adjudicated all clinical worsening events throughout the study.

Secondary Endpoints
Change in 6-Minute Walk Distance
From Baseline to Week 24
Change in Plasma N-Terminal Pro-brain Natriuretic Peptide (NT-proBNP) From Baseline to Week 24
From Baseline to Week 24
Change in World Health Organization Functional Class (WHO FC) From Baseline to Week 48
Baseline to Week 48
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
UT-15CEXPERIMENTALTreprostinil diolamine extended-release tablets (oral) 0.125 to 12 mg TID
PlaceboPLACEBO_COMPARATORMatching placebo tablets (oral)
Interventions
NameTypeDescription
Treprostinil DiolamineDRUGActive
PlaceboDRUGPlacebo
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Eligibility Criteria
Age Range18 Years — 75 Years
SexALL
Healthy VolunteersNo
Study Sites155

Subject Inclusion Criteria: 1. Voluntarily gave informed consent to participate in the study. 2. Are 18 to 75 years of age (inclusive) at Screening. 3. Women of childbearing potential must practice abstinence from intercourse when in line with their preferred and usual lifestyle, or use 2 different...

Countries:United StatesArgentinaAustraliaAustriaBrazilCanadaChileChinaDenmarkFranceGermanyGreeceIndiaIsraelItalyMexicoNetherlandsPolandSingaporeSouth KoreaSwedenTaiwanUnited Kingdom
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