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

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ClinicalTrials.gov Identifier: NCT00442611
Recruitment Status : Completed
First Posted : March 2, 2007
Results First Posted : April 24, 2015
Last Update Posted : October 31, 2017
Sponsor:
Collaborator:
Bristol-Myers Squibb
Information provided by (Responsible Party):
Lorinda S Chung, Stanford University

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Triple (Participant, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions Scleroderma, Diffuse
Scleroderma, Systemic
Interventions Drug: Abatacept
Drug: Placebo
Enrollment 10
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Abatacept IV Fluid
Hide Arm/Group Description Abatacept (dosed based upon weight) administered intravenously (IV) on days 1, 15, 30 and monthly thereafter for a total of 7 doses. Placebo to match abatacept (IV fluid) administered on days 1, 15, 30 and monthly thereafter for a total of 7 doses.
Period Title: Overall Study
Started 7 3
Completed 6 3
Not Completed 1 0
Arm/Group Title Abatacept IV Fluid Total
Hide Arm/Group Description Abatacept (dosed based upon weight) administered intravenously (IV) on days 1, 15, 30 and monthly thereafter for a total of 7 doses. Placebo to match abatacept (IV fluid) administered on days 1, 15, 30 and monthly thereafter for a total of 7 doses. Total of all reporting groups
Overall Number of Baseline Participants 7 3 10
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 7 participants 3 participants 10 participants
39.8  (11.4) 48.6  (13.9) 44.2  (12.5)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants 3 participants 10 participants
Female
5
  71.4%
3
 100.0%
8
  80.0%
Male
2
  28.6%
0
   0.0%
2
  20.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 7 participants 3 participants 10 participants
7 3 10
1.Primary Outcome
Title Change in Modified Rodnan Skin Score
Hide Description 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).
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Abatacept IV Fluid
Hide Arm/Group Description:
Abatacept (dosed based upon weight) administered intravenously (IV) on days 1, 15, 30 and monthly thereafter for a total of 7 doses.
Placebo to match abatacept (IV fluid) administered on days 1, 15, 30 and monthly thereafter for a total of 7 doses.
Overall Number of Participants Analyzed 7 3
Mean (Standard Deviation)
Unit of Measure: MRSS score
-8.6  (7.5) -2.3  (15)
2.Secondary Outcome
Title Oral Aperture at Baseline and Month 6
Hide Description [Not Specified]
Time Frame Baseline; Month 6
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Abatacept IV Fluid
Hide Arm/Group Description:
Abatacept (dosed based upon weight) administered intravenously (IV) on days 1, 15, 30 and monthly thereafter for a total of 7 doses.
Placebo to match abatacept (IV fluid) administered on days 1, 15, 30 and monthly thereafter for a total of 7 doses.
Overall Number of Participants Analyzed 7 3
Mean (Standard Deviation)
Unit of Measure: mm
Right side baseline 31.1  (6.51) 21.0  (4.32)
Right side month 6 29.4  (4.98) 21.0  (1.41)
Left side baseline 30.9  (7.18) 20.0  (2.45)
Left side month 6 29.3  (4.65) 20.0  (4.08)
3.Secondary Outcome
Title Hand Extension at Baseline and Month 6
Hide Description [Not Specified]
Time Frame Baseline; Month 6
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Abatacept IV Fluid
Hide Arm/Group Description:
Abatacept (dosed based upon weight) administered intravenously (IV) on days 1, 15, 30 and monthly thereafter for a total of 7 doses.
Placebo to match abatacept (IV fluid) administered on days 1, 15, 30 and monthly thereafter for a total of 7 doses.
Overall Number of Participants Analyzed 7 3
Mean (Standard Deviation)
Unit of Measure: mm
Right hand baseline 177.7  (28.11) 98.3  (20.95)
Right hand month 6 178.9  (31.38) 103.3  (24.94)
Left hand baseline 183.7  (23.96) 110.7  (35.98)
Left hand month 6 184.3  (25.23) 116.3  (40.58)
4.Secondary Outcome
Title Digital Ulcerations at Baseline and Month 6
Hide Description [Not Specified]
Time Frame Baseline; Month 6
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Abatacept IV Fluid
Hide Arm/Group Description:
Abatacept (dosed based upon weight) administered intravenously (IV) on days 1, 15, 30 and monthly thereafter for a total of 7 doses.
Placebo to match abatacept (IV fluid) administered on days 1, 15, 30 and monthly thereafter for a total of 7 doses.
Overall Number of Participants Analyzed 7 3
Mean (Standard Deviation)
Unit of Measure: ulcers
Right hand baseline 0.3  (0.70) 0.7  (0.47)
Right hand month 6 0.1  (0.35) 1.3  (0.47)
Left hand baseline 0.0  (0.00) 0.7  (0.47)
Left hand month 6 0.0  (0.00) 1.0  (0.82)
5.Secondary Outcome
Title Change in Pulmonary Function Tests
Hide Description 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.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Participants with available data were analyzed.
Arm/Group Title Abatacept IV Fluid
Hide Arm/Group Description:
Abatacept (dosed based upon weight) administered intravenously (IV) on days 1, 15, 30 and monthly thereafter for a total of 7 doses.
Placebo to match abatacept (IV fluid) administered on days 1, 15, 30 and monthly thereafter for a total of 7 doses.
Overall Number of Participants Analyzed 6 3
Mean (Standard Deviation)
Unit of Measure: % Predicted
FVC % Predicted 1.3  (8.5) .3  (8.5)
DLCO % Predicted 2.0  (6.3) -7.4  (10.7)
6.Secondary Outcome
Title Change in Scleroderma Health Assessment Questionnaire
Hide Description

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).

Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Participants with available data were analyzed.
Arm/Group Title Abatacept IV Fluid
Hide Arm/Group Description:
Abatacept (dosed based upon weight) administered intravenously (IV) on days 1, 15, 30 and monthly thereafter for a total of 7 doses.
Placebo to match abatacept (IV fluid) administered on days 1, 15, 30 and monthly thereafter for a total of 7 doses.
Overall Number of Participants Analyzed 6 3
Mean (Standard Deviation)
Unit of Measure: HAQ-DI score
-0.04  (.24) .25  (.25)
Time Frame 6 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Abatacept IV Fluid
Hide Arm/Group Description Abatacept (dosed based upon weight) administered intravenously (IV) on days 1, 15, 30 and monthly thereafter for a total of 7 doses. Placebo to match abatacept (IV fluid) administered on days 1, 15, 30 and monthly thereafter for a total of 7 doses.
All-Cause Mortality
Abatacept IV Fluid
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Abatacept IV Fluid
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/7 (14.29%)      0/3 (0.00%)    
Vascular disorders     
Infection  [1]  1/7 (14.29%)  1 0/3 (0.00%)  0
Indicates events were collected by systematic assessment
[1]
Infected toe digital ulcer
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Abatacept IV Fluid
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   6/7 (85.71%)      3/3 (100.00%)    
Gastrointestinal disorders     
nausea   0/1 (0.00%)  0 1/3 (33.33%)  1
fever   1/7 (14.29%)  1 0/3 (0.00%)  0
General disorders     
headache   1/7 (14.29%)  1 0/3 (0.00%)  0
dry mouth   0/7 (0.00%)  0 1/3 (33.33%)  1
Respiratory, thoracic and mediastinal disorders     
infection   2/7 (28.57%)  2 1/3 (33.33%)  1
Skin and subcutaneous tissue disorders     
pruritus   2/7 (28.57%)  2 0/3 (0.00%)  0
Vascular disorders     
lower extremity edema   0/7 (0.00%)  0 1/3 (33.33%)  1
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. Lorinda Chung
Organization: Stanford University
Phone: (650)493-5000 ext 6242
EMail: shauwai@stanford.edu
Layout table for additonal information
Responsible Party: Lorinda S Chung, Stanford University
ClinicalTrials.gov Identifier: NCT00442611     History of Changes
Other Study ID Numbers: 100 186
First Submitted: March 1, 2007
First Posted: March 2, 2007
Results First Submitted: January 26, 2015
Results First Posted: April 24, 2015
Last Update Posted: October 31, 2017