Trial Outcomes & Findings for Tacrolimus and Mycophenolate Mofetil (MMF) in GVHD Prophylactic Regimen Compared to Tacrolimus and Methotrexate (MTX (NCT NCT00360685)

NCT ID: NCT00360685

Last Updated: 2013-05-03

Results Overview

Mucositis was assessed prospectively daily while the patient was hospitalized and graded retrospectively based on nurse and clinician assessments according to the clinical criteria set forth in the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE; version 3.0). Severe mucositis as defined as grade 3 or grade 4.

• Recruitment status: COMPLETED
• Study phase: NA
• Target enrollment: 89 participants
• Primary outcome timeframe: 2 year
• Results posted on: 2013-05-03

Participant Flow

Participant milestones

Measure	Tacrolimus And Methotrexate All patients will receive TAC 0.03 mg/kg/24h as a continuous IV infusion beginning day -3 (day 0 being the anticipated day of hematopoietic stem cell infusion). Tacrolimus dosing should be based on ideal body weight. TAC levels should be measured on day 0 then at least twice weekly for the first month and the dose will be adjusted to maintain whole blood levels of 5-15ng/mL. When the patient is able to tolerate oral medications, the total daily dose of IV TAC will be converted to oral dosing at a 1:3 (IV:PO) ratio. The daily oral dose will be divided into twice daily dosing. Full dose TAC will be given until day +60 (+7) when tapering will begin in the absence of GVHD. Provided no GVHD develops, TAC should be discontinued by day +180 (+14). MTX 15 mg/m2 IV day +1 then 10 mg/m2 IV on days +3, +6, and +11.	Tacrolimus And Mycophenolate Mofetil All patients will receive TAC 0.03 mg/kg/24h as a continuous IV infusion beginning day -3. TAC levels should be measured on day 0 then at least twice weekly for the first month and the dose will be adjusted to maintain whole blood levels of 5-15ng/mL. When the patient is able to tolerate oral medications, the total daily dose of IV TAC will be converted to oral dosing. Full dose TAC will be given until day +60 (+7) when tapering will begin in the absence of GVHD. Provided no GVHD develops, TAC should be discontinued by day +180 (+14). MMF 30 mg/kg/day IV in 2 divided doses beginning day 0. Dosing should be based on the lesser of adjusted ideal body weight or actual body weight. The IV dose will be converted to the oral formulation when spatient is able to tolerate oral medications. Oral dosing may be capped at 1 gram twice daily. In the absence of GVHD a tapering schedule will begin on day +240 (+14) and be completed on day +360 (+14).
Overall Study STARTED	47	42
Overall Study COMPLETED	47	42
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Tacrolimus and Mycophenolate Mofetil (MMF) in GVHD Prophylactic Regimen Compared to Tacrolimus and Methotrexate (MTX

Baseline characteristics by cohort

Measure	Tacrolimus And Methotrexate n=47 Participants Tacrolimus (TAC) And Methotrexate (MTX)	Tacrolimus And Mycophenolate Mofetil n=42 Participants Tacrolimus (TAC) And Mycophenolate Mofetil (MMF)	Total n=89 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	45 Participants n=5 Participants	39 Participants n=7 Participants	84 Participants n=5 Participants
Age, Categorical >=65 years	2 Participants n=5 Participants	3 Participants n=7 Participants	5 Participants n=5 Participants
Sex: Female, Male Female	18 Participants n=5 Participants	21 Participants n=7 Participants	39 Participants n=5 Participants
Sex: Female, Male Male	29 Participants n=5 Participants	21 Participants n=7 Participants	50 Participants n=5 Participants
Region of Enrollment United States	47 participants n=5 Participants	42 participants n=7 Participants	89 participants n=5 Participants

PRIMARY outcome

Timeframe: 2 year

Population: Intent to treat

Mucositis was assessed prospectively daily while the patient was hospitalized and graded retrospectively based on nurse and clinician assessments according to the clinical criteria set forth in the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE; version 3.0). Severe mucositis as defined as grade 3 or grade 4.

Outcome measures

Measure	Tacrolimus And Methotrexate n=47 Participants Tacrolimus And Methotrexate	Tacrolimus And Mycophenolate Mofetil n=42 Participants Tacrolimus (TAC) And Mycophenolate Mofetil (MMF)
Incidence of Severe Mucositis	25 participants	14 participants

SECONDARY outcome

Timeframe: 100 days post transplant

Population: intent to treat

incidence of aGVHD (grades 2 - 4) 100 days post allogeneic hematopoietic cell transplantation

Outcome measures

Measure	Tacrolimus And Methotrexate n=47 Participants Tacrolimus And Methotrexate	Tacrolimus And Mycophenolate Mofetil n=42 Participants Tacrolimus (TAC) And Mycophenolate Mofetil (MMF)
Incidence of Acute Graft-vs-host Disease (aGVHD)	45 participants	33 participants

SECONDARY outcome

Timeframe: 1 year

Population: intent to treat

number of participants alive at one year

Outcome measures

Measure	Tacrolimus And Methotrexate n=47 Participants Tacrolimus And Methotrexate	Tacrolimus And Mycophenolate Mofetil n=42 Participants Tacrolimus (TAC) And Mycophenolate Mofetil (MMF)
Overall Survival	28 participants	27 participants

Adverse Events

Tacrolimus And Methotrexate

Serious events: 25 serious events

Other events: 0 other events

Deaths: 0 deaths

Tacrolimus And Mycophenolate Mofetil

Serious events: 19 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Measure	Tacrolimus And Methotrexate n=47 participants at risk Tacrolimus (TAC) And Methotrexate (MTX)	Tacrolimus And Mycophenolate Mofetil n=42 participants at risk Tacrolimus (TAC) And Mycophenolate Mofetil (MMF)
General disorders Death	53.2% 25/47 • Number of events 25 • 2 years	45.2% 19/42 • Number of events 19 • 2 years

Other adverse events

Adverse event data not reported

Additional Information

Janelle Perkins, PharmD

Moffitt Cancer Center

Phone: 813-745-7226

Email: Janelle.Perkins@moffit.org

Results disclosure agreements

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