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

NCT ID: NCT00725361

Last Updated: 2017-03-31

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 20 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2008-06-30
• Study Completion Date: 2010-12-31

Brief Summary

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.

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.

Conditions

n Ulcer; Scleroderma, Systemic

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Active

Ambrisentan

Group Type: EXPERIMENTAL

Ambrisentan

Intervention Type: DRUG

Interventions

Ambrisentan

Intervention Type: DRUG

Eligibility 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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Stanford University

OTHER

Sponsor Role: lead

Responsible Party

Lorinda S Chung

Associate Professor of Medicine

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Lorinda S Chung

Role: PRINCIPAL_INVESTIGATOR

Stanford University

Locations

Stanford University School of Medicine

Stanford, California, United States

Countries

United States

Other Identifiers

IRB Protocol # 14409

Identifier Type: -

Identifier Source: secondary_id

SU-07222008-1265

Identifier Type: -

Identifier Source: org_study_id