Open-Label Study of Oral Treprostinil in Digital Ulcers
- Registration Number
- NCT00848107
- Lead Sponsor
- United Therapeutics
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 115
- 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).
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Treprostinil treprostinil diethanolamine Treprostinil diethanolamine sustained release tablet initiated at 0.25 mg and titrated up to a maximum dose of 16 mg BID or the individual's maximum tolerated dose (MTD).
- Primary Outcome Measures
Name Time Method Formation of New Ulcers 18 months (or last study visit) The number and percentage of subjects who developed new ulcers during the study were summarized.
Net Ulcer Burden- Mean Change From Time of Study Entry for Each Scheduled Visit Assessment Baseline and Months 1, 3, 6, 9, 12, and 18 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.
Total Ulcer Number- Mean Change From Time of Study Entry for Each Scheduled Visit Assessment Baseline and Months 1, 3, 6, 9, 12, and 18 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.
- Secondary Outcome Measures
Name Time Method Patient Function and Quality of Life Measure: Cochin Hand Function Scale (CHFS)-Mean Change From Study Entry in CHFS Score at Each Scheduled Assessment Baseline and Months 1, 3, 6, and 12 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.
Patient Function and QOL Measure: Scleroderma Health Assessments Questionnaire (SHAQ)- Mean Change From Study Entry in SHAQ Component Scores at Each Scheduled Assessment Baseline and Months 1, 3, 6, and 12 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).
Trial Locations
- Locations (26)
Northwestern University - Feinberg School of Medicine
🇺🇸Chicago, Illinois, United States
Boston University School of Medicine
🇺🇸Boston, Massachusetts, United States
The Cleveland Clinic Foundation
🇺🇸Cleveland, Ohio, United States
UCLA
🇺🇸Los Angeles, California, United States
UMDNJ Clinical Research Center
🇺🇸New Brunswick, New Jersey, United States
University of Connecticut Health Center
🇺🇸Farmington, Connecticut, United States
Royal Free Hospital - Center for Rheumatology
🇬🇧London, United Kingdom
Johns Hopkins University - Division of Rheumatology
🇺🇸Baltimore, Maryland, United States
Virginia Mason Medical
🇺🇸Seattle, Washington, United States
The Hospital for Special Surgery
🇺🇸New York, New York, United States
Mayo Clinic Scottsdale
🇺🇸Scottsdale, Arizona, United States
Georgetown University - Dept. of Medicine/Rheumatology
🇺🇸Washington, District of Columbia, United States
Jewish General Hospital
🇨🇦Montreal, Quebec, Canada
North Shore - LIJ Health System
🇺🇸Lake Success, New York, United States
Salford Royal Hospital
🇬🇧Manchester, United Kingdom
University of Toledo
🇺🇸Toledo, Ohio, United States
University of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
St Joseph's Health Care
🇨🇦London, Ontario, Canada
University of Alabama - Birmingham - Arthritis Clinical Intervention Program
🇺🇸Birmingham, Alabama, United States
University of Utah
🇺🇸Salt Lake City, Utah, United States
Barbara Davis Centre
🇺🇸Aurora, Colorado, United States
University of Minnesota
🇺🇸Minneapolis, Minnesota, United States
University of Michigan - Scleroderma Program
🇺🇸Ann Arbor, Michigan, United States
Medical University of South Carolina
🇺🇸Charleston, South Carolina, United States
University of Texas - Houston
🇺🇸Houston, Texas, United States
Medical College of Wisconsin
🇺🇸Milwaukee, Wisconsin, United States