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Digital Ischemic Lesions in Scleroderma Treated With Oral Treprostinil Diethanolamine (DISTOL-1)
This study is currently recruiting participants.
Verified by United Therapeutics, August 2009
First Received: October 17, 2008   Last Updated: August 12, 2009   History of Changes
Sponsor: United Therapeutics
Information provided by: United Therapeutics
ClinicalTrials.gov Identifier: NCT00775463
  Purpose

This study will evaluate the effect of treprostinil diethanolamine (UT-15C) sustained release tablets(compared to placebo) on digital ulcers in patients with scleroderma. Treprostinil diethanolamine is an analog of prostacyclin. Prostacyclin is a naturally occuring substance produced by the cells of blood vessels that inhibits platelet aggregation, induces vasodilation, and suppresses smooth muscle proliferation. Improvement in blood flow in lower limbs and fingers would be anticipated to result in a reduction in ischemic pain, Raynaud's phenomenon and promote healing of digital ulcers and other ischemic wounds.


Condition Intervention Phase
Systemic Sclerosis
Scleroderma
Drug: treprostinil diethanolamine
Drug: placebo
Phase II

Study Type: Interventional
Study Design: Treatment, Randomized, Double Blind (Subject, Investigator), Placebo Control, Parallel Assignment, Efficacy Study
Official Title: DISTOL-1: Digital Ischemic Lesions in Scleroderma Treated With Oral Treprostinil Diethanolamine: A Randomized, Double-blind, Placebo-controlled, Multicenter Study

Resource links provided by NLM:


Further study details as provided by United Therapeutics:

Primary Outcome Measures:
  • Reduction in net ulcer burden [ Time Frame: Through week 20 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Scleroderma Health Assessment Questionnaire (SHAQ) and other functional and quality of life scales [ Time Frame: Through week 20 ] [ Designated as safety issue: No ]
  • modified Rodnan Skin Score (mRSS) [ Time Frame: Baseline and week 20 ] [ Designated as safety issue: No ]
  • serum biomarkers [ Time Frame: Baseline and week 20 ] [ Designated as safety issue: No ]
  • adverse events [ Time Frame: Throughout week 20 ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 150
Study Start Date: May 2009
Arms Assigned Interventions
treprostinil diethanolamine: Experimental Drug: treprostinil diethanolamine
oral sustained release tablet. Maximum tolerable dose not exceeding 16 mg twice daily (BID)
placebo (sugar pill): Placebo Comparator Drug: placebo

  Eligibility

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

Inclusion Criteria:

  • Subject gives voluntary written informed consent to participate in the study.
  • Diagnosis of systemic sclerosis (SSc) as defined by American College of Rheumatology (ACR) criteria.
  • Males and females age greater than 18 years
  • Presence of at least one active digital ulcer (meets protocol defined qualifications for active digital ulcer)
  • Females of childbearing potential must be willing to use a reliable form of medically acceptable contraception and have a negative pregnancy test
  • Able to communicate effectively with study personnel and willing to comply with protocol requirements.

Exclusion Criteria:

  • Diagnosis of pulmonary arterial hypertension (PAH).
  • Body weight less than 40 kg
  • History of postural hypotension, unexplained syncope, a blood pressure that is less than 85 mmHg systolic or 50 mmHg diastolic at Screening or Baseline.
  • Hemoglobin concentration less than 75% of the lower limit of the normal range
  • Moderate to severe hepatic impairment, i.e., Child-Pugh Class B or C.
  • Intractable diarrhea, or severe malabsorption, defined as greater than 15% unintentional loss of body weight in the last 6 months prior to Screening; any severe organ failure (e.g., lung, kidney), bleeding diathesis or platelet disorder, or any life-threatening condition.
  • Pregnancy or breast-feeding.
  • Overlap with another connective tissue disease that could affect rest pain and hand function (e.g. diabetes mellitus, rheumatoid arthritis).
  • Sympathectomy of the upper limb performed within 12 months of Baseline.
  • Receipt of prostanoid treatment (epoprostenol, treprostinil sodium, or other prostacyclin analog) within the previous 3 months of Baseline for conditions including Reynaud's phenomenon, rest pain and / or digital ulcers.
  • Required systemic antibiotics for infected digital ulcers within 2 weeks of Screening.
  • Local injection of botulinum toxin in an affected finger within 1 month prior to Baseline.
  • Treatment with endothelin receptor antagonists within 1 month prior to Baseline.
  • Treatment with phosphodiesterase inhibitors such as sildenafil, except for intermittent treatment of male erectile dysfunction.
  • Treatment with statin within 1 month prior to Screening, unless for management of hyperlipidemia.
  • Received an investigational product within 1 month preceding Screening.
  • Known hypersensitivity to treprostinil diethanolamine or any of the excipients.
  • Tobacco use at any level within the past 6 months prior to Screening.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00775463

Contacts
Contact: Kristan Rollins, PharmD 919-425-8167 krollins@unither.com

  Show 26 Study Locations
Sponsors and Collaborators
United Therapeutics
Investigators
Principal Investigator: James Seibold, MD University of Michigan Scleroderma Program
  More Information

No publications provided

Responsible Party: United Therapeutics ( Kristan Rollins, PharmD Sr Clinical Research Scientist )
Study ID Numbers: TDE-DU-201
Study First Received: October 17, 2008
Last Updated: August 12, 2009
ClinicalTrials.gov Identifier: NCT00775463     History of Changes
Health Authority: United States: Food and Drug Administration;   United Kingdom: Research Ethics Committee

Keywords provided by United Therapeutics:
Scleroderma, Diffuse
Scleroderma, Limited
Scleroderma, Systemic
Ulcer
prostacyclin

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

ClinicalTrials.gov processed this record on February 08, 2010