Digital Ischemic Lesions in Scleroderma Treated With Oral Treprostinil Diethanolamine (DISTOL-1)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
United Therapeutics
ClinicalTrials.gov Identifier:
NCT00775463
First received: October 17, 2008
Last updated: January 31, 2014
Last verified: January 2014
Results First Received: January 31, 2014  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Investigator);   Primary Purpose: Treatment
Conditions: Systemic Sclerosis
Scleroderma
Interventions: Drug: treprostinil diethanolamine
Drug: placebo

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Subjects were recruited across 32 clinical trial sites in the US, Canada and UK experienced in the treatment of systemic sclerosis (SSc) and capable of conducting the trial according to ICH GCP guidance. Subjects were recruited between April 2009 and October 2010.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Placebo All subjects who recieved at least one dose of study drug.
Treprostinil Diethanolamine All subejcts who recieved at least one dose of study drug. One subject in the treprostinil diethanolamine treatment group was randomized, withdrew consent and exited the study prior to taking any study medication and is not included in the analysis summary.

Participant Flow:   Overall Study
    Placebo     Treprostinil Diethanolamine  
STARTED     76     72  
COMPLETED     65     59  
NOT COMPLETED     11     13  
Withdrawal by Subject                 0                 1  
Adverse Event                 7                 9  
Protocol Violation                 2                 2  
Lost to Follow-up                 1                 0  
Lack of Efficacy                 1                 1  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Baseline demographics and characteristics were summarized for subjects who received at least one dose of study drug. One subject in the treprostinil diethanolamine treatment group was randomized, withdrew consent and exited the study prior to taking any study medication and is not included in the summary.

Reporting Groups
  Description
Placebo intent to treat population
Treprostinil Diethanolamine intent to treat population
Total Total of all reporting groups

Baseline Measures
    Placebo     Treprostinil Diethanolamine     Total  
Number of Participants  
[units: participants]
  76     71     147  
Age  
[units: years]
Mean ( Full Range )
  47.8  
  ( 20 to 74 )  
  49.8  
  ( 19 to 82 )  
  48.8  
  ( 19 to 82 )  
Gender  
[units: participants]
     
Female     55     54     109  
Male     21     17     38  
Race/Ethnicity, Customized [1]
[units: participants]
     
Asian     3     4     7  
Native Hawaiian or Other Pacific Islander     0     1     1  
Black or African American     10     7     17  
White     62     59     121  
Unknown or Not Reported     1     1     2  
SSc Classification  
[units: participants]
     
Limited     55     40     95  
Diffuse     21     31     52  
Years since SSc diagnosis  
[units: years]
Mean ( Full Range )
  10.7  
  ( 0 to 30 )  
  10.3  
  ( 0 to 35 )  
  10.5  
  ( 0 to 35 )  
[1] Note: A Subject can be included in more than one race category.



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Net Ulcer Burden   [ Time Frame: Week 20 ]

2.  Secondary:   Digital Ulcer Pain VAS   [ Time Frame: Week 20 ]

3.  Secondary:   Patient Global Assessment of Digital Ulcer Severity VAS   [ Time Frame: Week 20 ]

4.  Secondary:   Physician Global Assessment of Digital Ulcer Severity VAS   [ Time Frame: Week 20 ]

5.  Secondary:   Cochin Hand Function Scale (CHFS)   [ Time Frame: Week 20 ]

6.  Secondary:   Scleroderma Health Assessment Questionnaire (SHAQ)   [ Time Frame: Week 20 ]

7.  Secondary:   Modified Rodnan Skin Score (mRSS)   [ Time Frame: Week 20 ]

8.  Secondary:   Short-Form McGill Pain Questionnaire   [ Time Frame: Week 20 ]

9.  Secondary:   Patient Impression of Change (PIC) Questionnaire   [ Time Frame: Week 20 ]

10.  Secondary:   Short Form 36   [ Time Frame: Week 20 ]

11.  Secondary:   Time to Ulcer Healing- Percentage of Subjects With Complete Healing   [ Time Frame: Week 20 ]

12.  Secondary:   Time to Ulcer Healing   [ Time Frame: Week 20 ]


  Serious Adverse Events


  Other Adverse Events


  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Limitations and Caveats
Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.  


Results Point of Contact:  
Name/Title: Oral Treprostinil Program Head
Organization: United Therapeutics Corporation
phone: 919-485-8350
e-mail: klaliberte@unither.com


No publications provided


Responsible Party: United Therapeutics
ClinicalTrials.gov Identifier: NCT00775463     History of Changes
Other Study ID Numbers: TDE-DU-201
Study First Received: October 17, 2008
Results First Received: January 31, 2014
Last Updated: January 31, 2014
Health Authority: United States: Food and Drug Administration
United States: Institutional Review Board
United Kingdom: Medicines and Healthcare Products Regulatory Agency
United Kingdom: Research Ethics Committee