Open-Label Study of Oral Treprostinil in Digital Ulcers

Systemic Sclerosis Clinical Trial

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Official title:

Digital Ischemic Lesions in Scleroderma Treated With Oral Treprostinil Diethanolamine: An Open-label Multicenter Extension Study

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT00848107
• Other study ID #: TDE-DU-202
• Status: Terminated
• Phase: Phase 2
• First received: February 16, 2009
• Last updated: January 8, 2014
• Start date: September 2009
• Est. completion date: September 2011

Study information

• Verified date: January 2014
• Source: United Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationUnited Kingdom: Research Ethics CommitteeUnited Kingdom: Medicines and Healthcare Products Regulatory Agency
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 115
• Est. completion date: September 2011
• Est. primary completion date: September 2011
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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.

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Scleroderma, Diffuse
• Scleroderma, Systemic
• Systemic Sclerosis

Intervention

Drug:
• treprostinil diethanolamine
sustained release tablet; BID dosing; up to 16 mg BID

Locations

Country	Name	City	State
Canada	St Joseph's Health Care	London	Ontario
Canada	Jewish General Hospital	Montreal	Quebec
United Kingdom	Royal Free Hospital - Center for Rheumatology	London
United Kingdom	Salford Royal Hospital	Manchester
United States	University of Michigan - Scleroderma Program	Ann Arbor	Michigan
United States	Barbara Davis Centre	Aurora	Colorado
United States	Johns Hopkins University - Division of Rheumatology	Baltimore	Maryland
United States	University of Alabama - Birmingham - Arthritis Clinical Intervention Program	Birmingham	Alabama
United States	Boston University School of Medicine	Boston	Massachusetts
United States	Medical University of South Carolina	Charleston	South Carolina
United States	Northwestern University - Feinberg School of Medicine	Chicago	Illinois
United States	The Cleveland Clinic Foundation	Cleveland	Ohio
United States	University of Connecticut Health Center	Farmington	Connecticut
United States	University of Texas - Houston	Houston	Texas
United States	North Shore - LIJ Health System	Lake Success	New York
United States	UCLA	Los Angeles	California
United States	Medical College of Wisconsin	Milwaukee	Wisconsin
United States	University of Minnesota	Minneapolis	Minnesota
United States	UMDNJ Clinical Research Center	New Brunswick	New Jersey
United States	The Hospital for Special Surgery	New York	New York
United States	University of Pittsburgh	Pittsburgh	Pennsylvania
United States	University of Utah	Salt Lake City	Utah
United States	Mayo Clinic Scottsdale	Scottsdale	Arizona
United States	Virginia Mason Medical	Seattle	Washington
United States	University of Toledo	Toledo	Ohio
United States	Georgetown University - Dept. of Medicine/Rheumatology	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
United Therapeutics

Countries where clinical trial is conducted

United States,  Canada,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	Baseline and Months 1, 3, 6, 9, 12, and 18	No
Primary	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.	Baseline and Months 1, 3, 6, 9, 12, and 18	No
Primary	Formation of New Ulcers	The number and percentage of subjects who developed new ulcers during the study were summarized.	18 months (or last study visit)	No
Secondary	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).	Baseline and Months 1, 3, 6, and 12	No
Secondary	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.	Baseline and Months 1, 3, 6, and 12	No