Back to results

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

Primary Purpose

n Ulcer, Scleroderma, Systemic

Status

Completed

Phase

Not Applicable

Locations

United States

Study Type

Interventional

Intervention

Ambrisentan

About this trial

This is an interventional treatment trial for n Ulcer

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Stanford University School of Medicine

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Active

Arm Description

Ambrisentan

Outcomes

Primary Outcome Measures

New Digital Ulcers (DU) 4 Weeks Prior to Week 24

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

Secondary Outcome Measures

New DU 4 Weeks Prior to Week 12

The number of new DU that have developed in the preceding 4 weeks assessed at week 12 from baseline.

Subjects Experiencing Complete (Total Reepithelialization) Healing of All Baseline DU at Week 12.

Subjects Experiencing Complete Healing of > 50% of the Number of Baseline DU at Week 12.

Change in the Scleroderma Health Assessment Questionnaire (SHAQ) at Week 24 Compared With Baseline.

Range is 0-3 with 0 meaning the least amount of disability and 3 the greatest of disability.

Full Information

NCT ID

NCT00725361

First Posted

July 28, 2008

Last Updated

March 1, 2017

Sponsor

Stanford University

1. Study Identification

Unique Protocol Identification Number

NCT00725361

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

March 2017

Overall Recruitment Status

Completed

Study Start Date

June 2008 (undefined)

Primary Completion Date

December 2010 (Actual)

Study Completion Date

December 2010 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Stanford University

4. Oversight

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

n Ulcer, Scleroderma, Systemic

7. Study Design

Primary Purpose

Treatment

Study Phase

Not Applicable

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

20 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Active

Arm Type

Experimental

Arm Description

Ambrisentan

Intervention Type

Drug

Intervention Name(s)

Ambrisentan

Primary Outcome Measure Information:

Title

New Digital Ulcers (DU) 4 Weeks Prior to Week 24

Description

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

Time Frame

4 weeks prior to week 24

Secondary Outcome Measure Information:

Title

New DU 4 Weeks Prior to Week 12

Description

The number of new DU that have developed in the preceding 4 weeks assessed at week 12 from baseline.

Time Frame

4 weeks prior to week 12

Title

Subjects Experiencing Complete (Total Reepithelialization) Healing of All Baseline DU at Week 12.

Time Frame

12 weeks

Title

Subjects Experiencing Complete Healing of > 50% of the Number of Baseline DU at Week 12.

Time Frame

12 weeks

Title

Change in the Scleroderma Health Assessment Questionnaire (SHAQ) at Week 24 Compared With Baseline.

Description

Range is 0-3 with 0 meaning the least amount of disability and 3 the greatest of disability.

Time Frame

24 weeks

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Lorinda S Chung

Organizational Affiliation

Stanford University

Official's Role

Principal Investigator

Facility Information:

Facility Name

Stanford University School of Medicine

City

Stanford

State/Province

California

ZIP/Postal Code

94305

Country

United States

12. IPD Sharing Statement

Learn more about this trial

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

We'll reach out to this number within 24 hrs