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Trial record 2 of 2 for:    Abatacept | Scleroderma

A Study to Evaluate the Safety and Efficacy of Abatacept in Patients With Diffuse Systemic Sclerosis (Scleroderma)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00442611
Recruitment Status : Completed
First Posted : March 2, 2007
Results First Posted : April 24, 2015
Last Update Posted : October 31, 2017
Bristol-Myers Squibb
Information provided by (Responsible Party):
Lorinda S Chung, Stanford University

Brief 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.

Condition or disease Intervention/treatment Phase
Scleroderma, Diffuse Scleroderma, Systemic Drug: Abatacept Drug: Placebo Phase 1 Phase 2

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.

The protocol was amended during the study and the outcome measures "Change in Serum Autoantibody Profile" and "Change in Serum Cytokine Profile" were changed to exploratory outcomes.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Pilot Study to Evaluate the Safety and Efficacy of Abatacept in Patients With Systemic Sclerosis
Study Start Date : November 2008
Actual Primary Completion Date : June 2011
Actual Study Completion Date : June 2011

Arm Intervention/treatment
Experimental: Abatacept
Abatacept (dosed based upon weight) administered intravenously (IV) on days 1, 15, 30 and monthly thereafter for a total of 7 doses.
Drug: Abatacept
Placebo Comparator: IV fluid
Placebo to match abatacept (IV fluid) administered on days 1, 15, 30 and monthly thereafter for a total of 7 doses.
Drug: Placebo

Primary Outcome Measures :
  1. Change in Modified Rodnan Skin Score [ Time Frame: 6 months ]
    Modified Rodnan Skin Score measures skin thickness and is the sum of scores from 17 surface anatomic areas rated on a 0-3 scale (0=normal skin; 1=mild thickness; 2=moderate thickness; 3=severe thickness with inability to pinch the skin into a fold). Total modified Rodnan Skin Score ranges from 0 (best possible outcome) to 51 (worst possible outcome).

Secondary Outcome Measures :
  1. Oral Aperture at Baseline and Month 6 [ Time Frame: Baseline; Month 6 ]
  2. Hand Extension at Baseline and Month 6 [ Time Frame: Baseline; Month 6 ]
  3. Digital Ulcerations at Baseline and Month 6 [ Time Frame: Baseline; Month 6 ]
  4. Change in Pulmonary Function Tests [ Time Frame: 6 months ]
    FVC (Forced Vital Capacity) is the amount of air that can be forcibly exhaled from the lungs after taking the deepest possible breath. DLCO (Diffusing capacity of the lung for carbon monoxide) is the extent to which oxygen passes from the lungs to the blood.

  5. Change in Scleroderma Health Assessment Questionnaire [ Time Frame: 6 months ]

    The HAQ Disability Index (HAQ-DI) includes 20 items in 8 functional domains (dressing/grooming, arising, eating, walking, hygiene, reach, grip, and activities) assessing the patient's usual abilities in the past seven days. Each item is scored on a 0-3 scale (0=without any difficulty; 1=with some difficulty; 2=with much difficulty; 3=unable to do). The use of assistive devices for any domain increases the domain score by 1 point to a maximum of 3. The overall score is calculated by summing the highest item score in each of the domains and dividing the sum by 8, with an overall score of 0 indicating no disability, and a score of 3 indicating severe disability.

    The time points compared were 6 months to baseline (6 months minus baseline).

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00442611

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United States, California
Stanford University School of Medicine
Stanford, California, United States, 94305
Sponsors and Collaborators
Stanford University
Bristol-Myers Squibb
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Principal Investigator: Eliza Farmer Chakravarty Stanford University
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Lorinda S Chung, Principle Investigator, Stanford University Identifier: NCT00442611    
Other Study ID Numbers: 100 186
First Posted: March 2, 2007    Key Record Dates
Results First Posted: April 24, 2015
Last Update Posted: October 31, 2017
Last Verified: September 2017
Additional relevant MeSH terms:
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Scleroderma, Systemic
Scleroderma, Diffuse
Scleroderma, Localized
Connective Tissue Diseases
Skin Diseases
Immune Checkpoint Inhibitors
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents