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

Scleroderma, Systemic Clinical Trial

Official title:

A Pilot Study to Evaluate the Efficacy of Ambrisentan in the Treatment and Prevention of Digital Ulcers in Patients With Systemic Sclerosis.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00725361
• Other study ID #: SU-07222008-1265
• Secondary ID: IRB Protocol # 1
• Status: Completed
• Phase: N/A
• First received: July 28, 2008
• Last updated: November 2, 2016
• Start date: June 2008
• Est. completion date: December 2010

Study information

• Verified date: November 2016
• Source: Stanford University
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationUnited States: Institutional Review Board
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 20
• Est. completion date: December 2010
• Est. primary completion date: December 2010
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• n Ulcer
• Scleroderma, Diffuse
• Scleroderma, Systemic
• Sclerosis
• Ulcer

Intervention

Drug:
• Ambrisentan

Locations

Country	Name	City	State
United States	Stanford University School of Medicine	Stanford	California

Sponsors (1)

Lead Sponsor	Collaborator
Stanford University

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The number of new digital ulcers (DU) that have developed in the preceding 4 weeks assessed at week 24 from baseline.		4 weeks	No
Secondary	1. The number of new DU that have developed in the preceding 4 weeks assessed at week 12 from baseline.		4 weeks	No
Secondary	2. The percentage of subjects experiencing complete (total reepithelialization) healing of all baseline DU at weeks 12 and 24.		24 weeks	No
Secondary	3. The percentage of subjects experiencing complete healing of > 50% of the number of baseline DU at weeks 12 and 24.		12 and 24 weeks	No
Secondary	4. Change in the Scleroderma Health Assessment Questionnaire (SHAQ) at weeks 12 and 24.		12 and 24 weeks	No
Secondary	5. Change in physician global assessment of DU severity by visual analogue scale (VAS) at weeks 12 and 24.		12 and 24 weeks	No
Secondary	6. Change in the Cochin Hand Functional Scale (CHFS) at weeks 12 and 24.		12 and 24 weeks	No
Secondary	7. Change in SF-36 assessment of health status at weeks 12 and 24.		12 and 24 weeks	No
Secondary	8. Change in MRSS at weeks 12 and 24.		12 and 24 weeks	No