Open-Label Study of Oral Treprostinil in Digital Ulcers

Phase 2TerminatedNCT00848107

United Therapeutics115 enrolled

Overview

This open label extension trial will allow ongoing treatment of subjects who participated in the randomized controlled trials, and will provide long term information about the safety of treprostinil diethanolamine SR in subjects with SSc and digital ulcers.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

115

Conditions

Systemic Sclerosis

Interventions

treprostinil diethanolamineDRUG

sustained release tablet; BID dosing; up to 16 mg BID

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Eligible subjects who completed assessments for the final visit of the previous TDE-DU-201 controlled trial and who signed appropriate informed consent were eligible to enroll in this extension study. * If a female of childbearing potential, the subject must have agreed to continue practicing an acceptable method of birth control (i.e., surgical sterilization, approved hormonal contraceptives, barrier methods \[such as a condom or diaphragm\] used with a spermicide, or an intrauterine device). Exclusion Criteria: * Have had any change in clinical status that, in the opinion of the Investigator, would pose a safety risk for participation in this extension study; * Have been found to be unable to complete study assessments in the previous controlled trial; * Have prematurely discontinued study drug during the previous study due to clinical worsening or treatment related adverse events.

Locations (26)

Outcomes

Primary Outcomes

Net Ulcer Burden- Mean Change From Time of Study Entry for Each Scheduled Visit Assessment

Net ulcer burden at any given assessment was defined as the number of "new" or "active" ulcers at that assessment, plus the number of "indeterminate" ulcers at that assessment that had previously been classified as either "active" or "new" at any earlier assessment during the study. The mean change in net ulcer burden from time of study entry was summarized for each scheduled visit assessment.

Time frame: Baseline and Months 1, 3, 6, 9, 12, and 18

Total Ulcer Number- Mean Change From Time of Study Entry for Each Scheduled Visit Assessment

The total ulcer number includes all ulcers designated as "active", "indeterminate", or "new" for a given visit. The mean change in the total number of ulcers present from time of study entry was summarized for each scheduled visit assessment.

Time frame: Baseline and Months 1, 3, 6, 9, 12, and 18

Formation of New Ulcers

The number and percentage of subjects who developed new ulcers during the study were summarized.

Time frame: 18 months (or last study visit)

Secondary Outcomes

Patient Function and QOL Measure: Scleroderma Health Assessments Questionnaire (SHAQ)- Mean Change From Study Entry in SHAQ Component Scores at Each Scheduled Assessment

The SHAQ consists of 20 health assessment questionnaire questions with integer responses of 0 (without any difficulty) to 3 (unable to do), and five scleroderma-specific visual analog scale (VAS) domains (Overall Disease Activity, Raynaud's Phenomenon, Finger Ulcers, Breathing, and Intestinal Problems) with values ranging from 0.0 to 15.0 centimeters. The questions are divided into eight component domains: Dressing \& Grooming, Arising, Eating, Walking, Hygiene, Reach, Grip, and Activities. Each domain score is calculated by summing the domain responses and dividing by the number of questions in that domain. Each VAS domain score is calculated by dividing the value in centimeters by 5. SHAQ component and VAS domain score ranges from 0 (least limitation) to 3 (most limitation). The aggregate SHAQ score is calculated by dividing the sum of all domain scores by 13, with a score ranging from 0 (least limitation) to 3 (most limitation).

Data from ClinicalTrials.gov

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University of Alabama - Birmingham - Arthritis Clinical Intervention Program

Birmingham, Alabama, United States

Mayo Clinic Scottsdale

Scottsdale, Arizona, United States

UCLA

Los Angeles, California, United States

Barbara Davis Centre

Aurora, Colorado, United States

University of Connecticut Health Center

Farmington, Connecticut, United States

Georgetown University - Dept. of Medicine/Rheumatology

Washington D.C., District of Columbia, United States

Northwestern University - Feinberg School of Medicine

Chicago, Illinois, United States

Johns Hopkins University - Division of Rheumatology

Baltimore, Maryland, United States

Boston University School of Medicine

Boston, Massachusetts, United States

University of Michigan - Scleroderma Program

Ann Arbor, Michigan, United States

...and 16 more locations

Patient Function and Quality of Life Measure: Cochin Hand Function Scale (CHFS)-Mean Change From Study Entry in CHFS Score at Each Scheduled Assessment

The CHFS score is derived from 18 validated questions that assess functional disability and handicap due to hand involvement in rheumatoid arthritis. Each answer is scored on a scale with possible integer responses of 0(without difficulty) to 5 (impossible). The CHFS score is simply the sum of all 18 questions, divided by the number of questions actually answered, multiplied by 18. At least 10 of the 18 questions must have been answered in order for CHFS to be calculated. Therefore, CHFS score values can range from 0 (least limitation) to 90 (most limitation), with improvements in function or reduction of limitation indicated a decreased score value. The mean change from study entry in CHFS scores at each scheduled assessment are summarized.

Time frame: Baseline and Months 1, 3, 6, and 12