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

Primary Purpose

Systemic Sclerosis

Status
Terminated
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
treprostinil diethanolamine
Sponsored by
United Therapeutics
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Systemic Sclerosis focused on measuring systemic sclerosis, scleroderma, digital ulcers, vasculopathy

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • University of Alabama - Birmingham - Arthritis Clinical Intervention Program
  • Mayo Clinic Scottsdale
  • UCLA
  • Barbara Davis Centre
  • University of Connecticut Health Center
  • Georgetown University - Dept. of Medicine/Rheumatology
  • Northwestern University - Feinberg School of Medicine
  • Johns Hopkins University - Division of Rheumatology
  • Boston University School of Medicine
  • University of Michigan - Scleroderma Program
  • University of Minnesota
  • UMDNJ Clinical Research Center
  • North Shore - LIJ Health System
  • The Hospital for Special Surgery
  • The Cleveland Clinic Foundation
  • University of Toledo
  • University of Pittsburgh
  • Medical University of South Carolina
  • University of Texas - Houston
  • University of Utah
  • Virginia Mason Medical
  • Medical College of Wisconsin
  • St Joseph's Health Care
  • Jewish General Hospital
  • Royal Free Hospital - Center for Rheumatology
  • Salford Royal Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Treprostinil

Arm Description

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).

Outcomes

Primary Outcome Measures

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.
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.
Formation of New Ulcers
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
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
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.

Full Information

First Posted
February 16, 2009
Last Updated
January 8, 2014
Sponsor
United Therapeutics
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1. Study Identification

Unique Protocol Identification Number
NCT00848107
Brief Title
Open-Label Study of Oral Treprostinil in Digital Ulcers
Acronym
DISTOL-EXT
Official Title
Digital Ischemic Lesions in Scleroderma Treated With Oral Treprostinil Diethanolamine: An Open-label Multicenter Extension Study
Study Type
Interventional

2. Study Status

Record Verification Date
January 2014
Overall Recruitment Status
Terminated
Why Stopped
The TDE-DU-202 extension study was discontinued after the randomized, placebo-controlled TDE-DU-201 study did not meet its primary efficacy objective.
Study Start Date
September 2009 (undefined)
Primary Completion Date
September 2011 (Actual)
Study Completion Date
September 2011 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
United Therapeutics

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Systemic Sclerosis
Keywords
systemic sclerosis, scleroderma, digital ulcers, vasculopathy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
115 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Treprostinil
Arm Type
Experimental
Arm Description
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).
Intervention Type
Drug
Intervention Name(s)
treprostinil diethanolamine
Intervention Description
sustained release tablet; BID dosing; up to 16 mg BID
Primary Outcome Measure Information:
Title
Net Ulcer Burden- Mean Change From Time of Study Entry for Each Scheduled Visit Assessment
Description
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
Title
Total Ulcer Number- Mean Change From Time of Study Entry for Each Scheduled Visit Assessment
Description
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
Title
Formation of New Ulcers
Description
The number and percentage of subjects who developed new ulcers during the study were summarized.
Time Frame
18 months (or last study visit)
Secondary Outcome Measure Information:
Title
Patient Function and QOL Measure: Scleroderma Health Assessments Questionnaire (SHAQ)- Mean Change From Study Entry in SHAQ Component Scores at Each Scheduled Assessment
Description
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).
Time Frame
Baseline and Months 1, 3, 6, and 12
Title
Patient Function and Quality of Life Measure: Cochin Hand Function Scale (CHFS)-Mean Change From Study Entry in CHFS Score at Each Scheduled Assessment
Description
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
James Seibold, MD
Organizational Affiliation
Scleroderma Research Consultants LLC
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Alabama - Birmingham - Arthritis Clinical Intervention Program
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35294-7201
Country
United States
Facility Name
Mayo Clinic Scottsdale
City
Scottsdale
State/Province
Arizona
ZIP/Postal Code
85259
Country
United States
Facility Name
UCLA
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095-1670
Country
United States
Facility Name
Barbara Davis Centre
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
University of Connecticut Health Center
City
Farmington
State/Province
Connecticut
ZIP/Postal Code
06030-1310
Country
United States
Facility Name
Georgetown University - Dept. of Medicine/Rheumatology
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20007
Country
United States
Facility Name
Northwestern University - Feinberg School of Medicine
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Johns Hopkins University - Division of Rheumatology
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21224
Country
United States
Facility Name
Boston University School of Medicine
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02118-2526
Country
United States
Facility Name
University of Michigan - Scleroderma Program
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48106
Country
United States
Facility Name
University of Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Facility Name
UMDNJ Clinical Research Center
City
New Brunswick
State/Province
New Jersey
ZIP/Postal Code
08903
Country
United States
Facility Name
North Shore - LIJ Health System
City
Lake Success
State/Province
New York
ZIP/Postal Code
11042
Country
United States
Facility Name
The Hospital for Special Surgery
City
New York
State/Province
New York
ZIP/Postal Code
10021
Country
United States
Facility Name
The Cleveland Clinic Foundation
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Facility Name
University of Toledo
City
Toledo
State/Province
Ohio
ZIP/Postal Code
43614
Country
United States
Facility Name
University of Pittsburgh
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15261
Country
United States
Facility Name
Medical University of South Carolina
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425-8900
Country
United States
Facility Name
University of Texas - Houston
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
University of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Facility Name
Virginia Mason Medical
City
Seattle
State/Province
Washington
ZIP/Postal Code
98101
Country
United States
Facility Name
Medical College of Wisconsin
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States
Facility Name
St Joseph's Health Care
City
London
State/Province
Ontario
ZIP/Postal Code
N6A 4V2
Country
Canada
Facility Name
Jewish General Hospital
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H3T 1E2
Country
Canada
Facility Name
Royal Free Hospital - Center for Rheumatology
City
London
ZIP/Postal Code
NW3 2QG
Country
United Kingdom
Facility Name
Salford Royal Hospital
City
Manchester
ZIP/Postal Code
M13 9PT
Country
United Kingdom

12. IPD Sharing Statement

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Open-Label Study of Oral Treprostinil in Digital Ulcers

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