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treprostinil diethanolamine

Phase 2

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

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials2
Total Enrollment176

FDA Designations

No designations recorded

Clinical trial landscape

treprostinil diethanolamine · 7 trials · 4 indications

Phase 2 1Phase 1 6
NCT00775463Digital Ischemic Lesions in Scleroderma Treated With Oral Treprostinil DiethanolamineSystemic Sclerosis
COMPLETED148 Analytics
PHASE2COMPLETED
Digital Ischemic Lesions in Scleroderma Treated With Oral Treprostinil Diethanolamine
Systemic SclerosisUnlock trial analytics

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.

Plasma treprostinil concentrations
1 month

Pharmacokinetic blood sampling will occur twice for each subject. Pharmacokinetic parameters will be derived from treprostinil concentration-time data

Treprostinil pharmacokinetics
36 hours

Treprostinil pharmacokinetics in healthy volunteers following a single oral dose of 0.5 mg, 1 mg and 2.5 mg treprostinil diethanolamine sustained release tablets immediately prior to through 36 hours post treprostinil diethanolamine dosing.

Treprostinil pharmacokinetics in healthy volunteers following a single oral dose of a 1 mg treprostinil diethanolamine sustained release tablet after four different meal types.
Immediately prior to through 36 hours post treprostinil diethanolamine dosing for each treatment period (4 treatment periods each separated by a 7-day washout period)
Adverse event monitoring
From the first dose of treprostinil diethanolamine through study end (Study Day 23/24)
Treprostinil pharmacokinetics in healthy volunteers following a single oral dose of a 1 mg treprostinil diethanolamine sustained release tablet before and after repeated dosing with esomeprazole.
Within 10 minutes prior to through 36 hours post treprostinil diethanolamine dosing
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

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.
1 mg tablet; 1mg solutionEXPERIMENTAL -
1mg solution; 1mg tabletEXPERIMENTAL -
0.5 mg treprostinil diethanolamineEXPERIMENTAL0.5 mg treprostinil diethanolamine
2.5 mg treprostinil diethanolamineEXPERIMENTAL2.5 mg treprostinil diethanolamine
1 mg treprostinil diethanolamineEXPERIMENTAL1 mg treprostinil diethanolamine
manufacturer #1ACTIVE_COMPARATORtreprostinil diethanolamine from manufacturer #1
manufacturer #2EXPERIMENTALtreprostinil diethanolamine from manufacturer #2

Interventions

NameTypeDescription
treprostinil diethanolamineDRUGoral sustained release tablet. Maximum tolerable dose not exceeding 16 mg twice daily (BID)
placeboDRUG -
Standardized mealsOTHEREach subject will receive one of four different standardized meals of varying caloric and fat content in a randomized sequence such that all subjects will receive all four meals over the course of the study. Each subject will receive one standardized meal for breakfast on Study Days 1, 8, 15, and 22.
EsomeprazoleDRUGEach subject will receive a single 40 mg capsule of esomeprazole by mouth in the morning for seven days.
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Eligibility Criteria

Age Range18 Years to 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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Frequently asked questions about treprostinil diethanolamine

What is Treprostinil diethanolamine used for?

Treprostinil diethanolamine is an investigational small molecule being developed for pulmonary arterial hypertension, a form of pulmonary hypertension. It is also studied in the context of systemic sclerosis. The drug is in Phase 1 clinical development and is not yet approved by the FDA.

Who makes Treprostinil diethanolamine?

Treprostinil diethanolamine is being developed by United Therapeutics Corporation, a biopharmaceutical company traded on the NASDAQ under the ticker UTHR. The company is conducting Phase 1 clinical trials to evaluate the drug's pharmacokinetics and bioavailability in healthy volunteers and patients with pulmonary arterial hypertension.

What phase is Treprostinil diethanolamine in?

Treprostinil diethanolamine is in Phase 1 clinical development. All three completed trials are Phase 1 studies focused on pharmacokinetics, food effects, drug interactions, and bioavailability. The drug is investigational and has not received FDA approval.

What clinical trials is Treprostinil diethanolamine in?

Treprostinil diethanolamine has three completed Phase 1 trials: NCT00963001 (food effect on pharmacokinetics), NCT00963027 (effect of esomeprazole on pharmacokinetics), and NCT01153386 (pharmacokinetic linearity and comparative bioavailability). A fourth trial, NCT01165476, also completed, compared bioavailability from two manufacturing facilities.

Is Treprostinil diethanolamine the same as oral treprostinil?

Treprostinil diethanolamine is an oral formulation of treprostinil, a prostacyclin analog. The clinical trials listed for this drug specifically study the diethanolamine salt form, which is designed for oral administration. The drug is being developed by United Therapeutics for pulmonary arterial hypertension.