A Study to Evaluate the Safety and Efficacy of Abatacept in Patients With Diffuse Systemic Sclerosis (Scleroderma)
Information source: Stanford University
ClinicalTrials.gov processed this data on August 23, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Scleroderma, Diffuse; Scleroderma, Systemic
Intervention: Abatacept (Drug); Abatacept (Drug)
Phase: Phase 1/Phase 2
Status: Completed
Sponsored by: Stanford University Official(s) and/or principal investigator(s): Eliza Farmer Chakravarty, Principal Investigator, Affiliation: Stanford University
Summary
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.
Clinical Details
Official title: A Pilot Study to Evaluate the Safety and Efficacy of Abatacept in Patients With Systemic Sclerosis
Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator, Outcomes Assessor), Primary Purpose: Treatment
Primary outcome: Change in Modified Rodnan Skin Score
Secondary outcome: Change in Oral Aperture and Hand ExtensionChange in Pulmonary Function Tests Change in Digital Ulcerations Change in Scleroderma Health Assessment Questionnaire Change in Serum Autoantibody Profile Change in Serum Cytokine Profile
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
Minimum age: 18 Years.
Maximum age: N/A.
Gender(s): Both.
Criteria:
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
Locations and Contacts
Stanford University School of Medicine, Stanford, California 94305, United States
Additional Information
Starting date: November 2008
Last updated: April 23, 2015
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