A Study to Evaluate the Safety and Efficacy of Abatacept in Patients With Diffuse Systemic Sclerosis (Scleroderma)
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Purpose
Systemic sclerosis (scleroderma) is an autoimmune connective tissue disease that involves the skin and other internal organs for which there are few effective treatment options. We hypothesize that treatment with abatacept, a new therapy recently approved for the treatment of rheumatoid arthritis, may reduce the progression of skin thickening and fibrosis in people with scleroderma.
| Condition | Intervention | Phase |
|---|---|---|
|
Scleroderma, Diffuse Scleroderma, Systemic |
Drug: Abatacept |
Phase 1 Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Investigator, Outcomes Assessor) Primary Purpose: Treatment |
| Official Title: | A Pilot Study to Evaluate the Safety and Efficacy of Abatacept in Patients With Systemic Sclerosis |
- Change in modified Rodnan Skin Score [ Time Frame: 6 months ] [ Designated as safety issue: No ]
- Change in oral aperture and hand extension [ Time Frame: 6 months ] [ Designated as safety issue: No ]
- Change in Pulmonary Function Tests [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
- Change in digital ulcerations [ Time Frame: 6 months ] [ Designated as safety issue: No ]
- Scleroderma Health Assessment Questionnaire [ Time Frame: 6 months ] [ Designated as safety issue: No ]
- Change in serum autoantibody profile [ Time Frame: 6 months ] [ Designated as safety issue: No ]
- Change in serum cytokine profile [ Time Frame: 6 months ] [ Designated as safety issue: No ]
| Enrollment: | 10 |
| Study Start Date: | November 2008 |
| Study Completion Date: | June 2011 |
| Primary Completion Date: | June 2011 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: Abatacept
Study drug, Abatacept, will be administered in a double-blind fashion to the active arm of the study. It will be administered intravenously.
|
Drug: Abatacept
Active drug, abatacept, will be administered intravenously in a blinded fashion
Drug: Abatacept
Abatacept will be administered, dosed based upon weight, intravenously on days 1, 15, 30 and monthly thereafter for a total of 7 doses.
|
|
Placebo Comparator: IV fluid
The placebo arm will receive matched intravenous fluids.
|
Drug: Abatacept
Abatacept will be administered, dosed based upon weight, intravenously on days 1, 15, 30 and monthly thereafter for a total of 7 doses.
|
Detailed Description:
Systemic sclerosis is an autoimmune connective tissue disease of unknown etiology characterized by progressive fibrosis of the skin and internal organs, vascular damage, and autoantibody production. Although the disease is relatively rare, it is associated with considerable morbidity and mortality. There have been improvements in survival over the past few decades; however, this has been related to better management of vascular manifestations of disease including renal crisis, pulmonary hypertension, gastroesophageal reflux disease, and Raynaud's phenomenon. Clinical studies of disease modifying therapies for cutaneous disease to date have been relatively unsuccessful.
Although the etiology of the disease remains unknown, several observations support the role of activated T cells in both the blood and skin of affected patients. Abatacept, a recombinant fusion protein that blocks T cell activation, has recently been approved by the FDA for rheumatoid arthritis. We hypothesize that inhibition of T cell activation with abatacept may be efficacious in the treatment of patients with diffuse systemic sclerosis. This is a randomized, double-blinded, placebo-controlled clinical trial of abatacept versus placebo in patients with diffuse systemic sclerosis. Changes in validated measures of skin thickness and disease activity over 6-months of treatment will be compared between patients receiving abatacept and those receiving placebo. Patients will be randomized 2:1 to receive abatacept.
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Diagnosis of diffuse systemic sclerosis
- age 18 years or older
- Adequate renal, pulmonary, and cardiovascular function
- Willingness to use effective contraception for the duration of the study if subject is of childbearing potential

Exclusion Criteria:
- Other connective tissues diseases or overlap syndromes including MCTD, SLE, RA, eosinophilic fasciitis, and limited systemic sclerosis or morphea
- Use of disease modifying agents including methotrexate, cyclosporine,azathioprine, mycophenolate mofetil, minocycline, doxycycline, minocycline, thalidomide, penicillamine, tamoxifen, colchicine, or investigational agent within 90 days of screening visit
- HIV, Hepatitis B or Hepatitis C infection
- use of prednisone greater than 10mg daily for 28 days prior to screening visit
- women who are breastfeeding or pregnant
Contacts and Locations| United States, California | |
| Stanford University School of Medicine | |
| Stanford, California, United States, 94305 | |
| Principal Investigator: | Eliza Farmer Chakravarty | Stanford University |
More Information
No publications provided
| Responsible Party: | Eliza Chakravarty, Stanford University |
| ClinicalTrials.gov Identifier: | NCT00442611 History of Changes |
| Other Study ID Numbers: | 100 186 |
| Study First Received: | March 1, 2007 |
| Last Updated: | July 20, 2011 |
| Health Authority: | United States: Food and Drug Administration |
Additional relevant MeSH terms:
|
Scleroderma, Systemic Scleroderma, Diffuse Scleroderma, Localized Sclerosis Connective Tissue Diseases Skin Diseases Pathologic Processes |
Abatacept Antirheumatic Agents Therapeutic Uses Pharmacologic Actions Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs |
ClinicalTrials.gov processed this record on May 22, 2013