Systemic SclerosisPhase 2DISTOL-EXTNCT00848107
Open-Label Study of Oral Treprostinil in Digital Ulcers
A Phase 2 study testing treprostinil diethanolamine in people with Systemic Sclerosis, run by United Therapeutics at 26 sites in 3 countries.
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Systemic Sclerosis
- What is being tested?
- treprostinil diethanolamine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 115 participants
- Where
- 26 sitesin 3 countries
- Study dates
- Sep 2009to Sep 2011
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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).
Who cannot join
- 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.
Trial locations and contacts
26 sites in 3 countries.
26 sites in 3 countries
United States22 sites
University of Michigan - Scleroderma Program
Ann Arbor, Michigan, 48106
Barbara Davis Centre
Aurora, Colorado, 80045
Johns Hopkins University - Division of Rheumatology
Baltimore, Maryland, 21224
University of Alabama - Birmingham - Arthritis Clinical Intervention Program
Birmingham, Alabama, 35294-7201
Boston University School of Medicine
Boston, Massachusetts, 02118-2526
Medical University of South Carolina
Charleston, South Carolina, 29425-8900
Northwestern University - Feinberg School of Medicine
Chicago, Illinois, 60611
The Cleveland Clinic Foundation
Cleveland, Ohio, 44195
University of Connecticut Health Center
Farmington, Connecticut, 06030-1310
University of Texas - Houston
Houston, Texas, 77030
North Shore - LIJ Health System
Lake Success, New York, 11042
UCLA
Los Angeles, California, 90095-1670
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
University of Minnesota
Minneapolis, Minnesota, 55455
UMDNJ Clinical Research Center
New Brunswick, New Jersey, 08903
The Hospital for Special Surgery
New York, New York, 10021
University of Pittsburgh
Pittsburgh, Pennsylvania, 15261
University of Utah
Salt Lake City, Utah, 84132
Mayo Clinic Scottsdale
Scottsdale, Arizona, 85259
Virginia Mason Medical
Seattle, Washington, 98101
University of Toledo
Toledo, Ohio, 43614
Georgetown University - Dept. of Medicine/Rheumatology
Washington D.C., District of Columbia, 20007
Canada2 sites
St Joseph's Health Care
London, Ontario, N6A 4V2
Jewish General Hospital
Montreal, Quebec, H3T 1E2
United Kingdom2 sites
Royal Free Hospital - Center for Rheumatology
London, NW3 2QG
Salford Royal Hospital
Manchester, M13 9PT
About this study
Official title: Digital Ischemic Lesions in Scleroderma Treated With Oral Treprostinil Diethanolamine: An Open-label Multicenter Extension Study
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.
- Conditions
- Systemic Sclerosis
- Being tested
- treprostinil diethanolamine
- Keywords
- systemic sclerosis, scleroderma, digital ulcers, vasculopathy
- Medical subject headings
- Scleroderma, Systemic, Scleroderma, Diffuse, digital ulcers, Vascular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
treprostinil diethanolamineDrug
sustained release tablet; BID dosing; up to 16 mg BID
| Study group | Role | What they receive |
|---|---|---|
| Treprostinil | Experimental | 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). Receives: Drug: treprostinil diethanolamine |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline and Months 1, 3, 6, 9, 12, and 18
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.
Measured: Baseline and Months 1, 3, 6, 9, 12, and 18
Formation of New Ulcers
The number and percentage of subjects who developed new ulcers during the study were summarized.
Measured: 18 months (or last study visit)
Secondary outcomes (2)
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).
Measured: Baseline and Months 1, 3, 6, and 12
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.
Measured: Baseline and Months 1, 3, 6, and 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Feb 2009 (estimated)
- Study startSep 2009
- Primary completionSep 2011
- Study completionSep 2011
Why it 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.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Systemic SclerosisStudies of the same condition, open and under way.
- More trials run by United TherapeuticsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 115 participants (actual)
Sponsor and investigators
- Lead sponsor
- United Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- James Seibold, MD, Principal investigator, Scleroderma Research Consultants LLC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00848107
- Sponsor's protocol ID
- TDE-DU-202
- Acronym
- DISTOL-EXT
- Status last verified
- Jan 2014
- First submitted
- 16 Feb 2009
- Last updated
- 18 Jun 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Digital Ischemic Lesions in Scleroderma Treated With Oral Treprostinil Diethanolamine: An Open-label Multicenter Extension Study
- ClinicalTrials.gov ID
- NCT00848107
- Acronym / sponsor ID
- DISTOL-EXT / TDE-DU-202
- Conditions
- Systemic Sclerosis
- Keywords
- systemic sclerosis, scleroderma, digital ulcers, vasculopathy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason 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.
- Enrolment
- 115 participants (actual)
- Lead sponsor
- United TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- Sep 2009
- Primary completion
- Sep 2011 (actual)
- Study completion
- Sep 2011 (actual)
- First posted
- 20 Feb 2009 (estimated)
- Results
- Posted 7 Feb 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treprostinil | Experimental | Drug: treprostinil diethanolamineTreprostinil 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). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- 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).
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| treprostinil diethanolaminesustained release tablet; BID dosing; up to 16 mg BID | Drug | Treprostinil |
Primary outcome
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
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
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)
The number and percentage of subjects who developed new ulcers during the study were summarized.
2 secondary outcomes
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
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
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jun 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
James Seibold, MD principal investigator
Scleroderma Research Consultants LLC
26 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing treprostinil diethanolamine in people with Systemic Sclerosis, run by United Therapeutics at 26 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 18 Jun 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 26 sites in United States, Canada, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 7 Feb 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Jun 2024.