Trial Outcomes & Findings for Pharmacokinetics-based Mycophenolate Mofetil Dosing for GVHD Prevention (NCT NCT01487577)

NCT ID: NCT01487577

Last Updated: 2016-06-21

Results Overview

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 19 participants
• Primary outcome timeframe: 100 days
• Results posted on: 2016-06-21

Participant Flow

Candidates are identified through the investigators' clinical practice.

Participant milestones

Measure	Mycophenolate Mofetil Pharmacokinetics-based targeting of Mycophenolate mofetil
Overall Study STARTED	19
Overall Study COMPLETED	18
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Measure	Mycophenolate Mofetil Pharmacokinetics-based targeting of Mycophenolate mofetil
Overall Study Became ineligible after signing consent	1

Baseline Characteristics

Pharmacokinetics-based Mycophenolate Mofetil Dosing for GVHD Prevention

Baseline characteristics by cohort

Measure	Mycophenolate Mofetil n=19 Participants Pharmacokinetics-based targeting of mycophenolate mofetil Mycophenolate mofetil: Based on individual pharmacokinetics data, mycophenolate mofetil will be administered by continuous infusion to target a desired AUC exposure
Age, Categorical <=18 years	16 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	3 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	10 Participants n=5 Participants
Sex: Female, Male Male	9 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	2 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants
Race (NIH/OMB) White	15 Participants n=5 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	19 participants n=5 Participants

PRIMARY outcome

Timeframe: 100 days

Outcome measures

Measure	Mycophenolate Mofetil n=18 Participants Pharmacokinetics-based targeting of mycophenolate mofetil Mycophenolate mofetil: Based on individual pharmacokinetics data, mycophenolate mofetil will be administered by continuous infusion to target a desired AUC exposure
Number of Participants With Grade ≥3 Toxicities Scored According to the CTCAE Version 4.0.	0 participants

PRIMARY outcome

Timeframe: 1 year

Acute GVHD will be graded according to the Modified Glucksberg Staging Criteria. Chronic GVHD will be graded according to NIH Chronic GVHD Consensus Guidelines.

Outcome measures

Measure	Mycophenolate Mofetil n=18 Participants Pharmacokinetics-based targeting of mycophenolate mofetil Mycophenolate mofetil: Based on individual pharmacokinetics data, mycophenolate mofetil will be administered by continuous infusion to target a desired AUC exposure
Number of Participants With Acute Grade II-IV GVHD, Acute Grade III-IV GVHD, and Chronic GVHD. Acute grade II-IV GVHD	6 participants
Number of Participants With Acute Grade II-IV GVHD, Acute Grade III-IV GVHD, and Chronic GVHD. Acute grade III-IV GVHD	4 participants
Number of Participants With Acute Grade II-IV GVHD, Acute Grade III-IV GVHD, and Chronic GVHD. Chronic GVHD	2 participants

SECONDARY outcome

Timeframe: 100 days

Neutrophil and platelet engraftment definitions as defined by the CIBMTR Data Management Manual.

Outcome measures

Measure	Mycophenolate Mofetil n=18 Participants Pharmacokinetics-based targeting of mycophenolate mofetil Mycophenolate mofetil: Based on individual pharmacokinetics data, mycophenolate mofetil will be administered by continuous infusion to target a desired AUC exposure
Number of Participants With Neutrophil and Platelet Engraftment. Incidence of neutrophil engraftment	18 participants
Number of Participants With Neutrophil and Platelet Engraftment. Incidence of platelet engraftment	15 participants

SECONDARY outcome

Timeframe: 1 year

Outcome measures

Measure	Mycophenolate Mofetil n=18 Participants Pharmacokinetics-based targeting of mycophenolate mofetil Mycophenolate mofetil: Based on individual pharmacokinetics data, mycophenolate mofetil will be administered by continuous infusion to target a desired AUC exposure
Number of Participants Who Experienced Relapse.	4 participants

SECONDARY outcome

Timeframe: 1 year

Outcome measures

Measure	Mycophenolate Mofetil n=18 Participants Pharmacokinetics-based targeting of mycophenolate mofetil Mycophenolate mofetil: Based on individual pharmacokinetics data, mycophenolate mofetil will be administered by continuous infusion to target a desired AUC exposure
Number of Participants Who Experienced Nonrelapse Mortality.	0 participants

SECONDARY outcome

Timeframe: 1 year

Outcome measures

Measure	Mycophenolate Mofetil n=18 Participants Pharmacokinetics-based targeting of mycophenolate mofetil Mycophenolate mofetil: Based on individual pharmacokinetics data, mycophenolate mofetil will be administered by continuous infusion to target a desired AUC exposure
Number of Participants in Overall Survival.	15 participants

SECONDARY outcome

Timeframe: 100 days

Pharmacokinetic analysis includes, but is not limited to, area under the plasma concentration versus time curve (AUC).

Outcome measures

Measure	Mycophenolate Mofetil n=18 Participants Pharmacokinetics-based targeting of mycophenolate mofetil Mycophenolate mofetil: Based on individual pharmacokinetics data, mycophenolate mofetil will be administered by continuous infusion to target a desired AUC exposure
Pharmacokinetic Analysis of MMF AUC to Evaluate MMF Dose Relationships to Drug Exposure. AUC, Intermittent IV dosing	46.5 mcg*hr/mL Interval 16.7 to 65.9
Pharmacokinetic Analysis of MMF AUC to Evaluate MMF Dose Relationships to Drug Exposure. AUC, Continuous infusion	40.1 mcg*hr/mL Interval 20.6 to 63.8
Pharmacokinetic Analysis of MMF AUC to Evaluate MMF Dose Relationships to Drug Exposure. AUC, Intermittent PO dosing	36.1 mcg*hr/mL Interval 11.9 to 95.9

SECONDARY outcome

Timeframe: 100 days

Pharmacokinetic analysis includes, but is not limited to, clearance.

Outcome measures

Measure	Mycophenolate Mofetil n=18 Participants Pharmacokinetics-based targeting of mycophenolate mofetil Mycophenolate mofetil: Based on individual pharmacokinetics data, mycophenolate mofetil will be administered by continuous infusion to target a desired AUC exposure
Pharmacokinetic Analysis of MMF Clearance to Evaluate MMF Dose Relationships to Drug Exposure. Clearance, Intermittent IV dosing	10.2 mL/min/kg Interval 6.8 to 30.8
Pharmacokinetic Analysis of MMF Clearance to Evaluate MMF Dose Relationships to Drug Exposure. Clearance, Continuous infusion	15.1 mL/min/kg Interval 7.2 to 73.0
Pharmacokinetic Analysis of MMF Clearance to Evaluate MMF Dose Relationships to Drug Exposure. Clearance, Intermittent PO dosing	10.9 mL/min/kg Interval 4.9 to 26.6

SECONDARY outcome

Timeframe: 100 days

Pharmacokinetic analysis includes, but is not limited to, steady-state concentrations.

Outcome measures

Measure	Mycophenolate Mofetil n=18 Participants Pharmacokinetics-based targeting of mycophenolate mofetil Mycophenolate mofetil: Based on individual pharmacokinetics data, mycophenolate mofetil will be administered by continuous infusion to target a desired AUC exposure
Pharmacokinetic Analysis of MMF Steady State Concentration to Evaluate MMF Dose Relationships to Drug Exposure. Steady-state concentration, Intermittent IV dosing	1.9 mcg/mL Interval 0.6 to 2.7
Pharmacokinetic Analysis of MMF Steady State Concentration to Evaluate MMF Dose Relationships to Drug Exposure. Steady-state concentration, Continuous infusion	1.7 mcg/mL Interval 0.9 to 2.7
Pharmacokinetic Analysis of MMF Steady State Concentration to Evaluate MMF Dose Relationships to Drug Exposure. Steady-state concentration, Intermittent PO dosing	1.5 mcg/mL Interval 0.5 to 4.0

Adverse Events

Mycophenolate Mofetil

Serious events: 4 serious events

Other events: 8 other events

Deaths: 0 deaths

Serious adverse events

Measure	Mycophenolate Mofetil n=18 participants at risk Pharmacokinetics-based targeting of mycophenolate mofetil Mycophenolate mofetil: Based on individual pharmacokinetics data, mycophenolate mofetil will be administered by continuous infusion to target a desired AUC exposure
Infections and infestations Tracheitis	5.6% 1/18 • Number of events 1 • 1 year post-enrollment
Hepatobiliary disorders Hepatobiliary disorders, other	11.1% 2/18 • Number of events 2 • 1 year post-enrollment
Cardiac disorders Pericardial effusion	5.6% 1/18 • Number of events 1 • 1 year post-enrollment

Other adverse events

Measure	Mycophenolate Mofetil n=18 participants at risk Pharmacokinetics-based targeting of mycophenolate mofetil Mycophenolate mofetil: Based on individual pharmacokinetics data, mycophenolate mofetil will be administered by continuous infusion to target a desired AUC exposure
Immune system disorders Acute GVHD	44.4% 8/18 • Number of events 8 • 1 year post-enrollment
Immune system disorders Chronic GVHD	11.1% 2/18 • Number of events 2 • 1 year post-enrollment

Additional Information

Randy M. Windreich, MD

Children's Hospital of Pittsburgh of UPMC

Phone: 412-692-5225

Email: randy.windreich@chp.edu

Results disclosure agreements

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