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

Sponsor:	Stanford University
Information provided by:	Stanford University
ClinicalTrials.gov Identifier:	NCT00725361

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 I

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
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:

MedlinePlus related topics: Scleroderma

Drug Information available for: Ambrisentan

U.S. FDA Resources

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
Estimated Primary Completion Date:	June 2009 (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

Please refer to this study by its ClinicalTrials.gov identifier: NCT00725361

Contacts

Contact: Lorinda Chung, MD

(650) 493-5000 ext 62042

shauwei@stanford.edu

Locations

United States, California
Stanford University School of Medicine	Recruiting
Stanford, California, United States, 94305
Contact: Lorinda Chung, MD 650-493-5000 ext 62042 shauwei@stanford.edu
Contact: Kait Arefiev, MD (650) 721-7144 karefiev@stanford.edu
Principal Investigator: Lorinda S Chung
Sub-Investigator: Katharine Lucy Arefiev
Sub-Investigator: David Franklin Fiorentino

Sponsors and Collaborators

Stanford University

Investigators

Principal Investigator:

Lorinda S Chung

Stanford University

More Information

No publications provided

Responsible Party:	Stanford University ( Lorinda Chung, MD )
Study ID Numbers:	SU-07222008-1265, IRB Protocol # 14409
Study First Received:	July 28, 2008
Last Updated:	February 26, 2009
ClinicalTrials.gov Identifier:	NCT00725361 History of Changes
Health Authority:	United States: Food and Drug Administration; United States: Institutional Review Board

Additional relevant MeSH terms:

Scleroderma, Diffuse
Pathologic Processes
Skin Diseases

Ulcer
Scleroderma, Systemic
Connective Tissue Diseases

ClinicalTrials.gov processed this record on February 04, 2010