Open-Label Study of Oral Treprostinil in Digital Ulcers (DISTOL-EXT)

This study has been terminated.
(The TDE-DU-202 extension study was discontinued after the randomized, placebo-controlled TDE-DU-201 study did not meet its primary efficacy objective.)
Sponsor:
Information provided by (Responsible Party):
United Therapeutics
ClinicalTrials.gov Identifier:
NCT00848107
First received: February 16, 2009
Last updated: January 8, 2014
Last verified: January 2014
  Purpose

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.


Condition Intervention Phase
Systemic Sclerosis
Drug: treprostinil diethanolamine
Phase 2

Study Type: Interventional
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

Resource links provided by NLM:


Further study details as provided by United Therapeutics:

Primary Outcome Measures:
  • 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.


Secondary Outcome Measures:
  • 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.


Enrollment: 115
Study Start Date: September 2009
Study Completion Date: September 2011
Primary Completion Date: September 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Treprostinil
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

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00848107

  Show 26 Study Locations
Sponsors and Collaborators
United Therapeutics
Investigators
Principal Investigator: James Seibold, MD Scleroderma Research Consultants LLC
  More Information

No publications provided

Responsible Party: United Therapeutics
ClinicalTrials.gov Identifier: NCT00848107     History of Changes
Other Study ID Numbers: TDE-DU-202
Study First Received: February 16, 2009
Results First Received: July 12, 2013
Last Updated: January 8, 2014
Health Authority: United States: Food and Drug Administration
United Kingdom: Research Ethics Committee
United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by United Therapeutics:
systemic sclerosis
scleroderma
digital ulcers
vasculopathy

Additional relevant MeSH terms:
Scleroderma, Diffuse
Scleroderma, Systemic
Sclerosis
Connective Tissue Diseases
Pathologic Processes
Skin Diseases
Treprostinil
Antihypertensive Agents
Cardiovascular Agents
Pharmacologic Actions
Therapeutic Uses

ClinicalTrials.gov processed this record on October 23, 2014