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

NCT00442611 | Completed Phase 1 Results

• 1 other identifier: 100 186
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

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.

Conditions

Scleroderma, Diffuse; Scleroderma, Systemic

Outcome Measures

Primary Outcomes (1)

• Change in Modified Rodnan Skin Score

  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).

  6 months

Secondary Outcomes (5)

• Oral Aperture at Baseline and Month 6

  Baseline; Month 6

• Hand Extension at Baseline and Month 6

  Baseline; Month 6

• Digital Ulcerations at Baseline and Month 6

  Baseline; Month 6

• Change in Pulmonary Function Tests

  6 months

• Change in Scleroderma Health Assessment Questionnaire

  6 months

Study Arms (2)

Abatacept

EXPERIMENTAL

Abatacept (dosed based upon weight) administered intravenously (IV) on days 1, 15, 30 and monthly thereafter for a total of 7 doses.

Drug: Abatacept

IV fluid

PLACEBO COMPARATOR

Placebo to match abatacept (IV fluid) administered on days 1, 15, 30 and monthly thereafter for a total of 7 doses.

Drug: Placebo

Interventions

Abatacept DRUG

Abatacept

Placebo DRUG

IV fluid

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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\


You may not qualify if:

• 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

Sponsors & Collaborators

• Stanford University: lead
• Bristol-Myers Squibb: collaborator

Study Sites (1)

Stanford University School of Medicine

Stanford, California, 94305, United States

Location

Related Publications (1)

• Chakravarty EF, Martyanov V, Fiorentino D, Wood TA, Haddon DJ, Jarrell JA, Utz PJ, Genovese MC, Whitfield ML, Chung L. Gene expression changes reflect clinical response in a placebo-controlled randomized trial of abatacept in patients with diffuse cutaneous systemic sclerosis. Arthritis Res Ther. 2015 Jun 13;17(1):159. doi: 10.1186/s13075-015-0669-3.

  PMID: 26071192 DERIVED

MeSH Terms

Conditions

Scleroderma, Diffuse; Scleroderma, Systemic

Interventions

Abatacept

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Intervention Hierarchy (Ancestors)

Immunoconjugates; Antibodies; Immunoglobulins; Serum Globulins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Globulins

Results Point of Contact

• Title: Dr. Lorinda Chung
• Organization: Stanford University

shauwai@stanford.edu

(650)493-5000

Study Officials

• Eliza Farmer Chakravarty

  Stanford University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principle Investigator

Study Record Dates

• First Submitted: March 1, 2007
• First Posted: March 2, 2007
• Study Start: November 1, 2008
• Primary Completion: June 1, 2011
• Study Completion: June 1, 2011
• Last Updated: October 31, 2017
• Results First Posted: April 24, 2015

Record last verified: 2017-09

Locations

US (1)