Open-Label Study of Oral Treprostinil in Digital Ulcers (DISTOL-EXT)
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 Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Digital Ischemic Lesions in Scleroderma Treated With Oral Treprostinil Diethanolamine: An Open-label Multicenter Extension Study|
- Net Ulcer Burden- Mean Change From Time of Study Entry for Each Scheduled Visit Assessment [ Time Frame: Baseline and Months 1, 3, 6, 9, 12, and 18 ] [ Designated as safety issue: No ]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 [ Time Frame: Baseline and Months 1, 3, 6, 9, 12, and 18 ] [ Designated as safety issue: No ]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.
- Formation of New Ulcers [ Time Frame: 18 months (or last study visit) ] [ Designated as safety issue: No ]The number and percentage of subjects who developed new ulcers during the study were summarized.
- Patient Function and QOL Measure: Scleroderma Health Assessments Questionnaire (SHAQ)- Mean Change From Study Entry in SHAQ Component Scores at Each Scheduled Assessment [ Time Frame: Baseline and Months 1, 3, 6, and 12 ] [ Designated as safety issue: No ]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).
- Patient Function and Quality of Life Measure: Cochin Hand Function Scale (CHFS)-Mean Change From Study Entry in CHFS Score at Each Scheduled Assessment [ Time Frame: Baseline and Months 1, 3, 6, and 12 ] [ Designated as safety issue: No ]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.
|Study Start Date:||September 2009|
|Study Completion Date:||September 2011|
|Primary Completion Date:||September 2011 (Final data collection date for primary outcome measure)|
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).
Drug: treprostinil diethanolamine
sustained release tablet; BID dosing; up to 16 mg BID
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|Principal Investigator:||James Seibold, MD||Scleroderma Research Consultants LLC|