Trial · NCT00406393

Sirolimus/Tacrolimus Versus Tacrolimus/Methotrexate for Preventing Graft-Versus-Host Disease (GVHD) (BMT CTN 0402)

NCT00406393, Phase 3, Completed, Medical College of Wisconsin; results first posted 2016-03-07

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Sirolimus/Tacrolimus Versus Tacrolimus/Methotrexate for Preventing Graft-Versus-Host Disease (GVHD) (BMT CTN 0402). Trials, record NCT00406393. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00406393

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Sirolimus/Tacrolimus Versus Tacrolimus/Methotrexate for Preventing Graft-Versus-Host Disease (GVHD) (BMT CTN 0402)
NCT number: NCT00406393
Status: Completed
Phases: Phase 3
Enrollment: 304 participants, actual
Lead sponsor: Medical College of Wisconsin
Conditions: Leukemia, Myelocytic, Acute, Leukemia, Lymphocytic, Acute, Leukemia, Myeloid, Chronic, Myelodysplastic Syndromes
Interventions: Tacrolimus, Methotrexate, Sirolimus
First posted: 2006-12-04
Last updated: 2023-01-04
Results first posted: 2016-03-07

Source ClinicalTrials.gov

Registered outcomes12

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Rate of Grades II-IV Acute GVHD-free Survival	Day 114	The primary objective is to compare rates of 114-day Grades II-IV acute GVHD-free survival post randomization for HLA-matched, related donor allogeneic peripheral blood stem cell transplantation using two different GVHD prophylaxis regimens. Participants are graded on a scale of 1 to 4 according to their symptoms and organs involved, where 4 represents a worse grade.
Secondary	Incidence of Acute GVHD	Measured at Day 100	Cumulative incidence of acute GVHD (grade II-IV) occurring 100 days from transplantation.
Secondary	Time to Neutrophil and Platelet Engraftment	Measured through Day 100	Neutrophil engraftment is defined as achieving an Absolute Neutrophil Count (ANC) \> 500/mcL for three consecutive measurements on different days. Platelet engraftment is defined as a platelet count \> 20,000/mcL for three consecutive measurements over three or more days.
Secondary	Mucositis Severity	Measured at Day 21	Mucositis severity will be scored per the modified Oral Mucositis Assessment Scale (OMAS) scoring system on a scale of 0 - 4, where 0 equals normal mucosa and 4 represents severe mucosa.
Secondary	Rate of Veno-occlusive Disease (VOD)	Measured through Day 100	VOD will be defined as the occurrence of VOD (based on the Baltimore Criteria for the diagnosis of VOD) in conjunction with other end-organ dysfunction.
Secondary	Thrombotic Microangiopathy (TMA) Infection	Measured through Day 100	The occurrence of TMA within the first 100 days after stem cell transplantation will be recorded. The first day of onset will be used for reporting purposes.
Secondary	Reactivation of Cytomegalovirus (CMV) Infection	Measured at Year 2
Secondary	Treatment-related Mortality	Measured at Day 100 and Year 2
Secondary	Malignant Disease Relapse	Measured at Year 2	Testing for recurrent malignancy in the blood, marrow or other sites will be used to assess relapse after transplantation. For the purpose of this study, relapse is defined by either morphological or cytogenetic evidence of AML, ALL, CML, MDS or CMML consistent with pre-transplant features.
Secondary	Overall Survival	Measured at Year 2

1 to 10 of 12Source ClinicalTrials.gov

See all 12 registered outcomes

Outcomes12

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Units	Measured as
PRIMARY	Rate of Grades II-IV Acute GVHD-free Survival	Day 114	percentage of participants	NUMBER
SECONDARY	Incidence of Acute GVHD	Measured at Day 100	percentage of participants	NUMBER
SECONDARY	Time to Neutrophil and Platelet Engraftment	Measured through Day 100	days	MEDIAN
SECONDARY	Mucositis Severity	Measured at Day 21	units on a scale	MEAN
SECONDARY	Rate of Veno-occlusive Disease (VOD)	Measured through Day 100	percentage of participants	NUMBER
SECONDARY	Thrombotic Microangiopathy (TMA) Infection	Measured through Day 100	percentage of participants	NUMBER
SECONDARY	Reactivation of Cytomegalovirus (CMV) Infection	Measured at Year 2	percentage of participants	NUMBER
SECONDARY	Treatment-related Mortality	Measured at Day 100 and Year 2	percentage of participants	NUMBER
SECONDARY	Malignant Disease Relapse	Measured at Year 2	percentage of participants	NUMBER
SECONDARY	Overall Survival	Measured at Year 2	percentage of participants	NUMBER

1 to 10 of 12Source ClinicalTrials.gov

See all 12 outcomes

Results by group24

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Rate of Grades II-IV Acute GVHD-free Survival	Tacrolimus/Sirolimus		67	95% Confidence Interval 59 to 74	percentage of participants
Rate of Grades II-IV Acute GVHD-free Survival	Tacrolimus/Methotrexate		62	95% Confidence Interval 54 to 70	percentage of participants
Incidence of Acute GVHD	Tacrolimus/Sirolimus		26	95% Confidence Interval 19 to 34	percentage of participants
Incidence of Acute GVHD	Tacrolimus/Methotrexate		34	95% Confidence Interval 26 to 41	percentage of participants
Time to Neutrophil and Platelet Engraftment	Tacrolimus/Sirolimus	Neutrophil Engraftment	14	Full Range 8 to 27	days
Time to Neutrophil and Platelet Engraftment	Tacrolimus/Methotrexate	Neutrophil Engraftment	16	Full Range 10 to 68	days
Time to Neutrophil and Platelet Engraftment	Tacrolimus/Sirolimus	Platelet Engraftment	16	Full Range 7 to 100	days
Time to Neutrophil and Platelet Engraftment	Tacrolimus/Methotrexate	Platelet Engraftment	19	Full Range 9 to 100	days
Mucositis Severity	Tacrolimus/Sirolimus		0.31	Standard Deviation 0.28	units on a scale
Mucositis Severity	Tacrolimus/Methotrexate		0.47	Standard Deviation 0.40	units on a scale

1 to 10 of 24Source ClinicalTrials.gov

See all 24 results by group

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Tacrolimus/Sirolimus	151
Overall Study	STARTED	Tacrolimus/Methotrexate	153
Overall Study	COMPLETED	Tacrolimus/Sirolimus	149
Overall Study	COMPLETED	Tacrolimus/Methotrexate	152
Overall Study	NOT COMPLETED	Tacrolimus/Sirolimus	2
Overall Study	NOT COMPLETED	Tacrolimus/Methotrexate	1

Source ClinicalTrials.gov

Adverse events42

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
serious	Mental status changes	Psychiatric disorders	Tacrolimus/Sirolimus	1	149	1
serious	Mental status changes	Psychiatric disorders	Tacrolimus/Methotrexate	0	152
serious	Death	General disorders	Tacrolimus/Sirolimus	1	149	1
serious	Death	General disorders	Tacrolimus/Methotrexate	0	152
serious	Embolism	Vascular disorders	Tacrolimus/Sirolimus	1	149	1
serious	Embolism	Vascular disorders	Tacrolimus/Methotrexate	0	152
serious	Aphasia	Nervous system disorders	Tacrolimus/Sirolimus	1	149	1
serious	Aphasia	Nervous system disorders	Tacrolimus/Methotrexate	0	152
serious	Thoracotomy	Surgical and medical procedures	Tacrolimus/Sirolimus	1	149	1
serious	Thoracotomy	Surgical and medical procedures	Tacrolimus/Methotrexate	0	152

1 to 10 of 42Source ClinicalTrials.gov

See all 42 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Tacrolimus/Sirolimus
Total, all-cause mortality	Tacrolimus/Methotrexate
Total, other adverse events	Tacrolimus/Sirolimus	0	149
Total, other adverse events	Tacrolimus/Methotrexate	0	152
Total, serious adverse events	Tacrolimus/Sirolimus	9	149
Total, serious adverse events	Tacrolimus/Methotrexate	12	152

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
24982504	RESULT

Source PubMed

Grants1

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

Project	Title	Principal investigators	Organization	Funder	Fiscal years
U01HL069294	BMT Clinical Research Network Data Coordinating Center	HOROWITZ, MARY MARESCA	MEDICAL COLLEGE OF WISCONSIN	NATIONAL HEART, LUNG, AND BLOOD INSTITUTE	2001 to 2010

Source NIH RePORTER

Trial comparisons1

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison
Tacrolimus/Sirolimus vs Tacrolimus/Methotrexate: serious adverse events

Source Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT00406393

GET https://conductscience.org/api/trials/v1/NCT00406393/results?page=2

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