Trial Outcomes & Findings for Mycophenolate Mofetil (MMF) for Treatment of Chronic Graft-versus-host Disease (GVHD) (NCT NCT00089141)

NCT ID: NCT00089141

Last Updated: 2013-05-03

Results Overview

Withdrawal of all systemic immunosuppressive treatment after resolution of chronic GVHD, before death or onset of recurrent malignancy

• Recruitment status: TERMINATED
• Study phase: PHASE3
• Target enrollment: 151 participants
• Primary outcome timeframe: 2 years
• Results posted on: 2013-05-03

Participant Flow

Clinic patients were recruited from May 2004 through June 2008.

Participant milestones

Measure	Mycophenolate Mofetil Patients receive oral mycophenolate mofetil 1000 mg twice daily.	Placebo Patients receive oral placebo twice daily
Overall Study STARTED	74	77
Overall Study COMPLETED	56	53
Overall Study NOT COMPLETED	18	24

Reasons for withdrawal

Measure	Mycophenolate Mofetil Patients receive oral mycophenolate mofetil 1000 mg twice daily.	Placebo Patients receive oral placebo twice daily
Overall Study Termination of trial	18	24

Baseline Characteristics

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

Baseline characteristics by cohort

Measure	Mycophenolate Mofetil n=74 Participants Patients receive oral mycophenolate mofetil 1000 mg twice daily.	Placebo n=77 Participants Patients receive oral placebo twice daily	Total n=151 Participants Total of all reporting groups
Age, Categorical <=18 years	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	68 Participants n=5 Participants	71 Participants n=7 Participants	139 Participants n=5 Participants
Age, Categorical >=65 years	5 Participants n=5 Participants	5 Participants n=7 Participants	10 Participants n=5 Participants
Age Continuous	48.3 years STANDARD_DEVIATION 13.3 • n=5 Participants	47.9 years STANDARD_DEVIATION 13.3 • n=7 Participants	48.1 years STANDARD_DEVIATION 13.3 • n=5 Participants
Sex: Female, Male Female	33 Participants n=5 Participants	37 Participants n=7 Participants	70 Participants n=5 Participants
Sex: Female, Male Male	41 Participants n=5 Participants	40 Participants n=7 Participants	81 Participants n=5 Participants
Region of Enrollment United States	73 participants n=5 Participants	75 participants n=7 Participants	148 participants n=5 Participants
Region of Enrollment Canada	1 participants n=5 Participants	2 participants n=7 Participants	3 participants n=5 Participants

PRIMARY outcome

Timeframe: 2 years

Withdrawal of all systemic immunosuppressive treatment after resolution of chronic GVHD, before death or onset of recurrent malignancy

Outcome measures

Measure	Mycophenolate Mofetil n=74 Participants Patients receive oral mycophenolate mofetil 1000 mg twice daily.	Placebo n=77 Participants Patients receive oral placebo twice daily
Cure of Chronic GVHD Without Resorting to Secondary Systemic Therapy	11 participants	10 participants

SECONDARY outcome

Timeframe: 2 years

Administration of secondary systemic therapy for chronic GVHD, death during primary therapy, or onset of recurrent malignancy or bronchiolitis obliterans during primary therapy

Outcome measures

Measure	Mycophenolate Mofetil n=74 Participants Patients receive oral mycophenolate mofetil 1000 mg twice daily.	Placebo n=77 Participants Patients receive oral placebo twice daily
Definitive Absence of Efficacy Success	45 participants	40 participants

SECONDARY outcome

Timeframe: 2 years

Administration of any systemic therapy other than the immunosuppressive agents used for initial treatment, because of persistent or progressive chronic graft-versus-host disease

Outcome measures

Measure	Mycophenolate Mofetil n=74 Participants Patients receive oral mycophenolate mofetil 1000 mg twice daily.	Placebo n=77 Participants Patients receive oral placebo twice daily
Open Label Systemic Treatment Because of Inadequate Response to Primary Therapy	24 participants	25 participants

SECONDARY outcome

Timeframe: within 4 years

Development of bronchiolitis obliterans during treatment

Outcome measures

Measure	Mycophenolate Mofetil n=74 Participants Patients receive oral mycophenolate mofetil 1000 mg twice daily.	Placebo n=77 Participants Patients receive oral placebo twice daily
Bronchiolitis Obliterans	5 participants	4 participants

SECONDARY outcome

Timeframe: within 4 years

Development of recurrent malignancy after enrollment in the study

Outcome measures

Measure	Mycophenolate Mofetil n=74 Participants Patients receive oral mycophenolate mofetil 1000 mg twice daily.	Placebo n=77 Participants Patients receive oral placebo twice daily
Recurrent Malignancy	17 participants	10 participants

SECONDARY outcome

Timeframe: within 4 years

Death without prior development of recurrent malignancy

Outcome measures

Measure	Mycophenolate Mofetil n=74 Participants Patients receive oral mycophenolate mofetil 1000 mg twice daily.	Placebo n=77 Participants Patients receive oral placebo twice daily
Non-relapse Mortality	8 participants	5 participants

SECONDARY outcome

Timeframe: within 4 years

Death due to any cause or development of recurrent malignancy at any time after enrollment

Outcome measures

Measure	Mycophenolate Mofetil n=74 Participants Patients receive oral mycophenolate mofetil 1000 mg twice daily.	Placebo n=77 Participants Patients receive oral placebo twice daily
Death or Recurrent Malignancy	25 participants	15 participants

SECONDARY outcome

Timeframe: within 4 years

Death from any cause after enrollment in the study

Outcome measures

Measure	Mycophenolate Mofetil n=74 Participants Patients receive oral mycophenolate mofetil 1000 mg twice daily.	Placebo n=77 Participants Patients receive oral placebo twice daily
Death	19 participants	10 participants

SECONDARY outcome

Timeframe: within 4 years

Withdrawal of treatment with prednisone after improvement or resolution of chronic GVHD

Outcome measures

Measure	Mycophenolate Mofetil n=74 Participants Patients receive oral mycophenolate mofetil 1000 mg twice daily.	Placebo n=77 Participants Patients receive oral placebo twice daily
Withdrawal of Prednisone	30 participants	33 participants

SECONDARY outcome

Timeframe: within 4 years

Withdrawal of all immunosuppressive treatment without recurrent malignancy

Outcome measures

Measure	Mycophenolate Mofetil n=74 Participants Patients receive oral mycophenolate mofetil 1000 mg twice daily.	Placebo n=77 Participants Patients receive oral placebo twice daily
End of Systemic Treatment	15 participants	15 participants

Adverse Events

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Paul J. Martin, M.D.

Fred Hutchinson Cancer Research Center (FHCRC)

Phone: 206-667-4798

Email: pmartin@fhcrc.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place