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Pulmonary Involvement in Scleroderma: A Clinical Study of the Safety and Efficacy of Mycophenolate Mofetil in Scleroderma Patients With Lung Involvement

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ClinicalTrials.gov Identifier: NCT00333437
Recruitment Status : Completed
First Posted : June 5, 2006
Results First Posted : May 17, 2013
Last Update Posted : November 13, 2013
Sponsor:
Collaborator:
Roche Pharma AG
Information provided by (Responsible Party):
University of California, San Francisco

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Scleroderma, Systemic
Intervention Drug: Mycophenolate mofetil
Enrollment 7
Recruitment Details 8 patients were screened at one clinical site (UCSF) in the United States
Pre-assignment Details One patient screened for enrollment did not meet eligibility criteria
Arm/Group Title Single-group Study Treatment
Hide Arm/Group Description Study subjects receive standard mycophenolate dosing 2 grams/day.
Period Title: Overall Study
Started 7
Completed 7
Not Completed 0
Arm/Group Title Treatment
Hide Arm/Group Description Study subjects receive standard mycophenolate dosing.
Overall Number of Baseline Participants 7
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants
<=18 years
0
   0.0%
Between 18 and 65 years
7
 100.0%
>=65 years
0
   0.0%
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 7 participants
52.57  (7.52)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants
Female
5
  71.4%
Male
2
  28.6%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 7 participants
7
1.Primary Outcome
Title Mean Change From Baseline in Forced Vital Capacity (FVC)
Hide Description compare pre- and post-therapy FVC (post- minus pre-). Forced vital capacity (FVC) is the volume of air (liters) that can forcibly be blown out after full inspiration.
Time Frame Baseline, 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment
Hide Arm/Group Description:
Study subjects receive standard mycophenolate dosing.
Overall Number of Participants Analyzed 7
Mean (Standard Deviation)
Unit of Measure: Liters
0.1786  (0.1613)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Treatment
Comments H(0): post-pre FVC (liters) = 0
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.026
Comments Not adjusted
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Change from Baseline
Estimated Value 0.1786
Confidence Interval (2-Sided) 95%
0.0294 to 0.3277
Parameter Dispersion
Type: Standard Deviation
Value: 0.1613
Estimation Comments [Not Specified]
2.Secondary Outcome
Title Mean Change in Bronchoalveolar Lavage (BAL) Components (Neutrophils, Eosinophils)
Hide Description BAL samples were colleected from the affected lobe (as determined by lung CT scans) before beginning and after completing study therapy.
Time Frame Baseline, 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment
Hide Arm/Group Description:
Study subjects receive standard mycophenolate dosing 2 grams/day.
Overall Number of Participants Analyzed 7
Mean (Standard Deviation)
Unit of Measure: Cells/uL
Mean change in neutrophil count -3  (8.1)
Mean change in eosinophil count -6.3  (16.4)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Treatment
Comments H(0): Post-pre neutrophil count = 0
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3652
Comments significant p<0.05
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value -3
Confidence Interval (2-Sided) 95%
-10.49 to 4.4945
Parameter Dispersion
Type: Standard Deviation
Value: 8.1035
Estimation Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Treatment
Comments H(0): post-pre eosinophil count = 0
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3485
Comments significant p<0.05
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value -6.286
Confidence Interval (2-Sided) 95%
-21.41 to 8.8426
Parameter Dispersion
Type: Standard Deviation
Value: 16.358
Estimation Comments [Not Specified]
3.Secondary Outcome
Title Change in Shortness of Breath (Self-reported)
Hide Description Participants reported frequency of shortness of breath experienced with exertion
Time Frame Baseline, 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment
Hide Arm/Group Description:
Study subjects receive standard mycophenolate dosing.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: participants
Patient-reported less shortness of breath 6
Patient-reported no change in shortness of breath 1
Patient-reported increased shortness of breath 0
4.Secondary Outcome
Title Mean Change in Six Minute Walk Distance
Hide Description Comparison of 6-minute walk distance before beginning and after completing study therapy
Time Frame 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment
Hide Arm/Group Description:
Study subjects receive standard mycophenolate dosing.
Overall Number of Participants Analyzed 7
Mean (Standard Deviation)
Unit of Measure: Feet
264.3  (194.7)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Treatment
Comments H(0): post-pre walk distance = 0
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0115
Comments Significant p<0.05
Method t-test, 2 sided
Comments Not adjusted
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 264.26
Confidence Interval (2-Sided) 95%
84.256 to 444.32
Parameter Dispersion
Type: Standard Deviation
Value: 194.66
Estimation Comments [Not Specified]
5.Secondary Outcome
Title Mean Change in Diffusion Capacity of the Lung for Carbon Monoxide (DLCO)
Hide Description DLCO was measured before beginning and after completion of study therapy
Time Frame 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment
Hide Arm/Group Description:
Study subjects receive standard mycophenolate dosing.
Overall Number of Participants Analyzed 7
Mean (Standard Deviation)
Unit of Measure: Liters
1.86  (1.50)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Treatment
Comments H(0): Post-pre DLCO = 0
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0167
Comments significant p<0.05
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 1.8643
Confidence Interval (2-Sided) 95%
0.4758 to 3.2527
Parameter Dispersion
Type: Standard Deviation
Value: 1.5013
Estimation Comments [Not Specified]
Time Frame 12 months
Adverse Event Reporting Description Appropriate safety and serological studies were obtained prior to starting study medication and on a regular basis throughout the study. Patients were asked to keep a diary of significant medical events occurring between visits.
 
Arm/Group Title Treatment
Hide Arm/Group Description Study subjects receive standard mycophenolate dosing.
All-Cause Mortality
Treatment
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Treatment
Affected / at Risk (%) # Events
Total   1/7 (14.29%)    
Blood and lymphatic system disorders   
Leukopenia  [1]  1/7 (14.29%)  7
Indicates events were collected by systematic assessment
[1]
Patient was hospitalized
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Treatment
Affected / at Risk (%) # Events
Total   1/7 (14.29%)    
Gastrointestinal disorders   
Gastrointestinal distress  [1]  1/7 (14.29%)  1
Indicates events were collected by systematic assessment
[1]
Patient was unable to tolerate full dose of study drug because of GI distress
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Jeffrey A. Golden, MD
Organization: University of California San Francisco
Phone: 415-353-2935
Responsible Party: University of California, San Francisco
ClinicalTrials.gov Identifier: NCT00333437     History of Changes
Other Study ID Numbers: CEL371
First Submitted: June 2, 2006
First Posted: June 5, 2006
Results First Submitted: May 6, 2013
Results First Posted: May 17, 2013
Last Update Posted: November 13, 2013