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treprostinil diethanolamine

Phase 2

Systemic Sclerosis | Small molecule | Other |United Therapeutics Corporation|Last Updated: Dec 28, 2023

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_CONTROLLEDBiomarker
Total Trials2
Total Enrollment176
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00775463Digital Ischemic Lesions in Scleroderma Treated With Oral Treprostinil DiethanolaminePHASE2 COMPLETED 148May 1, 2009Jul 1, 2011Dec 28, 202330 United States, Canada +1
NCT00848939Pharmacokinetics of Oral Treprostinil in Patients With Systemic SclerosisPHASE1 COMPLETED 28Dec 1, 2008Apr 1, 2010Oct 23, 20123 United States
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Study Endpoints
Primary Endpoints
Net Ulcer Burden
Week 20

Net ulcer burden was defined as the number of "new" or "active" digital ulcers (DU), plus the number of "indeterminate" DUs at that assessment that have previously been classified as either "active" or "new" at any earlier assessment during the study. A DU was defined as an area with visually discernable depth and a loss of continuity of epithelial coverage, which could be denuded or covered by a scab or necrotic tissue. If denuded, the DU was pronounced "active." If denudation could not be judged because of the presence of scab or necrotic tissue, DU presenting with features, including underlying pain, based on Investigator clinical judgment to be consistent with loss of epithelialization, epidermis, or dermis, and requiring treatment were designated as "active." Otherwise, the DU was pronounced "indeterminate." Only DUs distal to the proximal interphalangeal joints, volar to the equator of the finger, not localized in creases and vascular in origin were assessed.

Cohort 1: treprostinil pharmacokinetics in patients with systemic sclerosis following single oral administration of a 1 mg treprostinil diethanolamine SR dose.
pre-24hrs post dose
Cohort 2: treprostinil pharmacokinetics at dose levels of 2 mg BID and 4 mg BID, respectively, in patients with systemic sclerosis following repeated oral administration of treprostinil diethanolamine SR tablets
0-12 hrs post-dose
adverse event monitoring
Cohort 1:Day 0 to Day 2; Cohort 2: Day 0 to Day 47
Secondary Endpoints
Digital Ulcer Pain VAS
Week 20
Patient Global Assessment of Digital Ulcer Severity VAS
Week 20
Physician Global Assessment of Digital Ulcer Severity VAS
Week 20
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
treprostinil diethanolamineEXPERIMENTALTreprostinil diethanolamine sustained release tablet initiated at 0.25 mg BID and titrated up to a maximum dose of 16 mg BID or the individual's maximum tolerated dose.
placebo (sugar pill)PLACEBO_COMPARATORMatching placebo sustained release tablet initiated at 0.25 mg BID and titrated up to a maximum dose of 16 mg BID or the individual's maximum tolerated dose.
Interventions
NameTypeDescription
treprostinil diethanolamineDRUGoral sustained release tablet. Maximum tolerable dose not exceeding 16 mg twice daily (BID)
placeboDRUG -
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites30

Inclusion Criteria: * Subject gave voluntary written informed consent to participate in the study * Diagnosis of systemic sclerosis (SSc) as defined by American College of Rheumatology (ACR) criteria * Males and females age greater than 18 years at Screening * Presence of at least one active digita...

Countries:United StatesCanadaUnited Kingdom
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