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Trial record 96 of 1164 for:    MYCOPHENOLIC ACID

Mycophenolate Mofetil (MMF) for Treatment of Chronic Graft-versus-host Disease (GVHD)

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ClinicalTrials.gov Identifier: NCT00089141
Recruitment Status : Terminated (Low probability of positive outcome)
First Posted : August 5, 2004
Results First Posted : August 26, 2009
Last Update Posted : May 3, 2013
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Martin, Paul, Fred Hutchinson Cancer Research Center

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Cancer
Interventions Drug: mycophenolate mofetil
Drug: placebo
Enrollment 151
Recruitment Details Clinic patients were recruited from May 2004 through June 2008.
Pre-assignment Details  
Arm/Group Title Mycophenolate Mofetil Placebo
Hide Arm/Group Description Patients receive oral mycophenolate mofetil 1000 mg twice daily. Patients receive oral placebo twice daily
Period Title: Overall Study
Started 74 77
Completed 56 53
Not Completed 18 24
Reason Not Completed
Termination of trial             18             24
Arm/Group Title Mycophenolate Mofetil Placebo Total
Hide Arm/Group Description Patients receive oral mycophenolate mofetil 1000 mg twice daily. Patients receive oral placebo twice daily Total of all reporting groups
Overall Number of Baseline Participants 74 77 151
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 74 participants 77 participants 151 participants
<=18 years
1
   1.4%
1
   1.3%
2
   1.3%
Between 18 and 65 years
68
  91.9%
71
  92.2%
139
  92.1%
>=65 years
5
   6.8%
5
   6.5%
10
   6.6%
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 74 participants 77 participants 151 participants
48.3  (13.3) 47.9  (13.3) 48.1  (13.3)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 74 participants 77 participants 151 participants
Female
33
  44.6%
37
  48.1%
70
  46.4%
Male
41
  55.4%
40
  51.9%
81
  53.6%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 74 participants 77 participants 151 participants
United States 73 75 148
Canada 1 2 3
1.Primary Outcome
Title Cure of Chronic GVHD Without Resorting to Secondary Systemic Therapy
Hide Description Withdrawal of all systemic immunosuppressive treatment after resolution of chronic GVHD, before death or onset of recurrent malignancy
Time Frame 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Mycophenolate Mofetil Placebo
Hide Arm/Group Description:
Patients receive oral mycophenolate mofetil 1000 mg twice daily.
Patients receive oral placebo twice daily
Overall Number of Participants Analyzed 74 77
Measure Type: Number
Unit of Measure: participants
11 10
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Mycophenolate Mofetil, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value .22
Comments P values are 2 sided and are based on likelihood ratio statistics.
Method Regression, Cox
Comments Analysis for all endpoints was stratified by number of affected organs and type of conditioning regimen.
Method of Estimation Estimation Parameter Hazard Ratio (HR)
Estimated Value 1.66
Confidence Interval 95%
0.7 to 3.7
Estimation Comments For all analyses, MMF arm in numerator, and placebo arm in denominator. Hazard ratio estimate includes 3 efficacy success events that occurred after two years in the placebo arm.
2.Secondary Outcome
Title Definitive Absence of Efficacy Success
Hide Description Administration of secondary systemic therapy for chronic GVHD, death during primary therapy, or onset of recurrent malignancy or bronchiolitis obliterans during primary therapy
Time Frame 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Mycophenolate Mofetil Placebo
Hide Arm/Group Description:
Patients receive oral mycophenolate mofetil 1000 mg twice daily.
Patients receive oral placebo twice daily
Overall Number of Participants Analyzed 74 77
Measure Type: Number
Unit of Measure: participants
45 40
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Mycophenolate Mofetil, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value .03
Comments [Not Specified]
Method Regression, Cox
Comments Statistical analysis did not count treatment continuing beyond 2 years as efficacy failure (n = 2 in each arm).
Method of Estimation Estimation Parameter Hazard Ratio (HR)
Estimated Value 1.65
Confidence Interval 95%
1.1 to 2.6
Estimation Comments [Not Specified]
3.Secondary Outcome
Title Open Label Systemic Treatment Because of Inadequate Response to Primary Therapy
Hide Description Administration of any systemic therapy other than the immunosuppressive agents used for initial treatment, because of persistent or progressive chronic graft-versus-host disease
Time Frame 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Mycophenolate Mofetil Placebo
Hide Arm/Group Description:
Patients receive oral mycophenolate mofetil 1000 mg twice daily.
Patients receive oral placebo twice daily
Overall Number of Participants Analyzed 74 77
Measure Type: Number
Unit of Measure: participants
24 25
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Mycophenolate Mofetil, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value .55
Comments [Not Specified]
Method Regression, Cox
Comments [Not Specified]
Method of Estimation Estimation Parameter Hazard Ratio (HR)
Estimated Value 1.19
Confidence Interval 95%
0.7 to 2.1
Estimation Comments [Not Specified]
4.Secondary Outcome
Title Bronchiolitis Obliterans
Hide Description Development of bronchiolitis obliterans during treatment
Time Frame within 4 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Mycophenolate Mofetil Placebo
Hide Arm/Group Description:
Patients receive oral mycophenolate mofetil 1000 mg twice daily.
Patients receive oral placebo twice daily
Overall Number of Participants Analyzed 74 77
Measure Type: Number
Unit of Measure: participants
5 4
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Mycophenolate Mofetil, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value .48
Comments [Not Specified]
Method Regression, Cox
Comments [Not Specified]
Method of Estimation Estimation Parameter Hazard Ratio (HR)
Estimated Value 1.61
Confidence Interval 95%
0.4 to 6.0
Estimation Comments [Not Specified]
5.Secondary Outcome
Title Recurrent Malignancy
Hide Description Development of recurrent malignancy after enrollment in the study
Time Frame within 4 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Mycophenolate Mofetil Placebo
Hide Arm/Group Description:
Patients receive oral mycophenolate mofetil 1000 mg twice daily.
Patients receive oral placebo twice daily
Overall Number of Participants Analyzed 74 77
Measure Type: Number
Unit of Measure: participants
17 10
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Mycophenolate Mofetil, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value .17
Comments [Not Specified]
Method Regression, Cox
Comments adjusted for risk category
Method of Estimation Estimation Parameter Hazard Ratio (HR)
Estimated Value 1.74
Confidence Interval 95%
0.8 to 3.9
Estimation Comments [Not Specified]
6.Secondary Outcome
Title Non-relapse Mortality
Hide Description Death without prior development of recurrent malignancy
Time Frame within 4 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Mycophenolate Mofetil Placebo
Hide Arm/Group Description:
Patients receive oral mycophenolate mofetil 1000 mg twice daily.
Patients receive oral placebo twice daily
Overall Number of Participants Analyzed 74 77
Measure Type: Number
Unit of Measure: participants
8 5
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Mycophenolate Mofetil, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value .41
Comments [Not Specified]
Method Regression, Cox
Comments [Not Specified]
Method of Estimation Estimation Parameter Hazard Ratio (HR)
Estimated Value 1.62
Confidence Interval 95%
0.5 to 5.0
Estimation Comments [Not Specified]
7.Secondary Outcome
Title Death or Recurrent Malignancy
Hide Description Death due to any cause or development of recurrent malignancy at any time after enrollment
Time Frame within 4 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Mycophenolate Mofetil Placebo
Hide Arm/Group Description:
Patients receive oral mycophenolate mofetil 1000 mg twice daily.
Patients receive oral placebo twice daily
Overall Number of Participants Analyzed 74 77
Measure Type: Number
Unit of Measure: participants
25 15
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Mycophenolate Mofetil, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value .14
Comments [Not Specified]
Method Regression, Cox
Comments [Not Specified]
Method of Estimation Estimation Parameter Hazard Ratio (HR)
Estimated Value 1.69
Confidence Interval 95%
0.9 to 3.2
Estimation Comments [Not Specified]
8.Secondary Outcome
Title Death
Hide Description Death from any cause after enrollment in the study
Time Frame within 4 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Mycophenolate Mofetil Placebo
Hide Arm/Group Description:
Patients receive oral mycophenolate mofetil 1000 mg twice daily.
Patients receive oral placebo twice daily
Overall Number of Participants Analyzed 74 77
Measure Type: Number
Unit of Measure: participants
19 10
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Mycophenolate Mofetil, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value .10
Comments [Not Specified]
Method Regression, Cox
Comments [Not Specified]
Method of Estimation Estimation Parameter Hazard Ratio (HR)
Estimated Value 1.99
Confidence Interval 95%
0.9 to 4.3
Estimation Comments [Not Specified]
9.Secondary Outcome
Title Withdrawal of Prednisone
Hide Description Withdrawal of treatment with prednisone after improvement or resolution of chronic GVHD
Time Frame within 4 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Mycophenolate Mofetil Placebo
Hide Arm/Group Description:
Patients receive oral mycophenolate mofetil 1000 mg twice daily.
Patients receive oral placebo twice daily
Overall Number of Participants Analyzed 74 77
Measure Type: Number
Unit of Measure: participants
30 33
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Mycophenolate Mofetil, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value .34
Comments [Not Specified]
Method Regression, Cox
Comments [Not Specified]
Method of Estimation Estimation Parameter Hazard Ratio (HR)
Estimated Value 1.28
Confidence Interval 95%
.08 to 2.1
Estimation Comments [Not Specified]
10.Secondary Outcome
Title End of Systemic Treatment
Hide Description Withdrawal of all immunosuppressive treatment without recurrent malignancy
Time Frame within 4 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Mycophenolate Mofetil Placebo
Hide Arm/Group Description:
Patients receive oral mycophenolate mofetil 1000 mg twice daily.
Patients receive oral placebo twice daily
Overall Number of Participants Analyzed 74 77
Measure Type: Number
Unit of Measure: participants
15 15
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Mycophenolate Mofetil, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value .28
Comments [Not Specified]
Method Regression, Cox
Comments [Not Specified]
Method of Estimation Estimation Parameter Hazard Ratio (HR)
Estimated Value 1.51
Confidence Interval 95%
0.7 to 3.2
Estimation Comments [Not Specified]
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
Name/Title: Paul J. Martin, M.D.
Organization: Fred Hutchinson Cancer Research Center (FHCRC)
Phone: 206-667-4798
Responsible Party: Martin, Paul, Fred Hutchinson Cancer Research Center
ClinicalTrials.gov Identifier: NCT00089141     History of Changes
Other Study ID Numbers: 1697.00
FHCRC-1697.00
ROCHE-FHCRC-1697.00
UMN-2004UC007
CDR0000378054 ( Registry Identifier: PDQ )
First Submitted: August 4, 2004
First Posted: August 5, 2004
Results First Submitted: July 15, 2009
Results First Posted: August 26, 2009
Last Update Posted: May 3, 2013