A Study to Evaluate the Efficacy of an Oral Medication in the Treatment and Prevention of Digital Ulcers in Patients With Systemic Sclerosis (Scleroderma).

This study has been completed.
Sponsor:
Information provided by:
Stanford University
ClinicalTrials.gov Identifier:
NCT00725361
First received: July 28, 2008
Last updated: March 11, 2011
Last verified: March 2011
  Purpose

This is a research study of an investigational drug called ambrisentan (Letairis) in the treatment and prevention of digital ulcers in patients with systemic sclerosis.


Condition Intervention Phase
n Ulcer
Scleroderma, Systemic
Drug: Ambrisentan
Phase 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Pilot Study to Evaluate the Efficacy of Ambrisentan in the Treatment and Prevention of Digital Ulcers in Patients With Systemic Sclerosis.

Resource links provided by NLM:


Further study details as provided by Stanford University:

Primary Outcome Measures:
  • The number of new digital ulcers (DU) that have developed in the preceding 4 weeks assessed at week 24 from baseline.

Secondary Outcome Measures:
  • 1. The number of new DU that have developed in the preceding 4 weeks assessed at week 12 from baseline.
  • 2. The percentage of subjects experiencing complete (total reepithelialization) healing of all baseline DU at weeks 12 and 24.
  • 3. The percentage of subjects experiencing complete healing of > 50% of the number of baseline DU at weeks 12 and 24.
  • 4. Change in the Scleroderma Health Assessment Questionnaire (SHAQ) at weeks 12 and 24.
  • 5. Change in physician global assessment of DU severity by visual analogue scale (VAS) at weeks 12 and 24.
  • 6. Change in the Cochin Hand Functional Scale (CHFS) at weeks 12 and 24.
  • 7. Change in SF-36 assessment of health status at weeks 12 and 24.
  • 8. Change in MRSS at weeks 12 and 24.

Estimated Enrollment: 20
Study Start Date: June 2008
Primary Completion Date: December 2010 (Final data collection date for primary outcome measure)
Detailed Description:

Ambrisentan is an endothelin blocker approved for the treatment of pulmonary arterial hypertension. Patients with systemic sclerosis can have damage to their blood vessels, resulting in increased levels of endothelin in their bloodstream that then causes decreased blood flow to the digits. This can result in very painful digital ulcers. We hope to learn whether blocking the action of endothelin with ambrisentan will be helpful in the treatment and prevention of digital ulcers in patients with systemic sclerosis. This therapy is not approved for the treatment of systemic sclerosis, but this study will help us learn whether ambrisentan is safe and effective in the treatment and prevention of digital ulcers in patients with systemic sclerosis.

  Eligibility

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

Inclusion Criteria:

  • Diagnosis of limited or diffuse systemic sclerosis (SSc) according to American College of Rheumatology or LeRoy criteria
  • Age greater than 18 years of age
  • At least one digital ulcer located on the volar or lateral surface at or distal to the proximal interphalangeal joints
  • At least one new DU that developed within 12 weeks prior to screening
  • Vasodilator therapies, including calcium channel blockers, alpha-1-antagonists, ACE-inhibitors, nitroglycerin, and angiotensin receptor blockers, are permitted as long as the doses are stable for 2 weeks prior to screening and throughout the study
  • Treatment with omeprazole or other proton pump inhibitors must be stable for 2 weeks prior to screening and throughout the study

Exclusion Criteria:

  • Patients with pulmonary arterial hypertension, NYHA Class III or IV
  • Patients who are hemodynamically unstable, or have acute renal, cardiac or pulmonary failure
  • Concurrent malignancy except non-melanoma skin cancers
  • Patients who have required systemic antibiotics for infected digital ulcers within 2 weeks of screening
  • Patients receiving phosphodiesterase-5 inhibitors, endothelin receptor antagonists, or prostanoids within 4 weeks of screening
  • Patients receiving cyclosporine within 6 weeks of screening
  • Patients who have participated in any investigational study within 30 days of screening
  • Pregnant or nursing women
  • Patients with a history of drug or alcohol abuse within 6 months of screening
  • History of hepatitis B, hepatitis C, or HIV infection
  • Any medical condition that, in the opinion of the investigator, might interfere with the subject's participation in the study or poses an added risk for the subject
  • Inability to comply with study and follow-up procedures
  • Transaminase elevation > 3X the upper limit of normal at screening
  • Hemoglobin less than 8.5 g/dL
  • Platelet count less than 100 X 109/L
  • White blood cell count less than 3.0 X 109/L
  • Serum creatinine less than 2.0 mg/dL
  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: NCT00725361

Locations
United States, California
Stanford University School of Medicine
Stanford, California, United States, 94305
Sponsors and Collaborators
Stanford University
Investigators
Principal Investigator: Lorinda S Chung Stanford University
  More Information

No publications provided

Responsible Party: Lorinda Chung, MD, Stanford University
ClinicalTrials.gov Identifier: NCT00725361     History of Changes
Other Study ID Numbers: SU-07222008-1265, IRB Protocol # 14409
Study First Received: July 28, 2008
Last Updated: March 11, 2011
Health Authority: United States: Food and Drug Administration
United States: Institutional Review Board

Additional relevant MeSH terms:
Scleroderma, Systemic
Scleroderma, Diffuse
Sclerosis
Ulcer
Connective Tissue Diseases
Skin Diseases
Pathologic Processes

ClinicalTrials.gov processed this record on July 28, 2014