Trial · NCT03959241
TAC/MTX vs. TAC/MMF/PTCY for Prevention of Graft-versus-Host Disease and Microbiome and Immune Reconstitution Study (BMT CTN 1703/1801)
NCT03959241, Phase 3, Completed, Medical College of Wisconsin; results first posted 2024-01-12
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. TAC/MTX vs. TAC/MMF/PTCY for Prevention of Graft-versus-Host Disease and Microbiome and Immune Reconstitution Study (BMT CTN 1703/1801). Trials, record NCT03959241. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03959241
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- TAC/MTX vs. TAC/MMF/PTCY for Prevention of Graft-versus-Host Disease and Microbiome and Immune Reconstitution Study (BMT CTN 1703/1801)
- NCT number
- NCT03959241
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 431 participants, actual
- Lead sponsor
- Medical College of Wisconsin
- Conditions
- Acute Leukemia, Chronic Myelogenous Leukemia (CML), Myelodysplasia, Lymphoma
- Interventions
Mobilized Peripheral Blood Stem Cell graft with Tacrolimus/Methotrexate, Tacrolimus, Methotrexate, Mobilized Peripheral Blood Stem Cell graft with Tacrolimus/Mycophenolate Mofetil/Post-Transplant Cyclophosphamide, Tacrolimus, Mycophenolate Mofetil, Cyclophosphamide
- First posted
- 2019-05-22
- Last updated
- 2024-04-04
- Results first posted
- 2024-01-12
Source ClinicalTrials.gov
Registered outcomes24
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants With GVHD/Relapse or Progression-free Survival (GRFS) at One Year | 1 year post randomization | The primary endpoint is GRFS as a time to event endpoint from randomization. All randomized patients will be followed for one year; however, the primary endpoint will be analyzed as a time to event endpoint. The primary analysis will be done using the randomized population. An event for this time to event outcome is defined as grade III-IV acute GVHD, chronic GVHD requiring systemic immune suppression, disease relapse or progression, or death by any cause, whichever comes first. Time to event analyses were conducted. An unadjusted Kaplan-Meier analysis was done. Additionally, a multivariate Cox regression model adjusting for the following pre-specified covariates: age group (\< 65 vs. 65+), disease risk index (low, intermediate, high/very high), planned RIC conditioning regimen (Fludarabine/Busulfan, Fludarabine/Melphalan, Other), donor type/HLA matching score (related 6/6, unrelated 7/8, unrelated 8/8), and planned use of post-transplant maintenance therapy (Yes vs. no). |
| Secondary | Percentage of Participants With Grades II-IV and III-IV Acute GVHD at Day 100 Post-transplant | Days 100 post-transplant | Acute GVHD was graded according to Mount Sinai aGVHD International Consortium (MAGIC) Criteria (Harris et al. 2016) with higher grade indicating worse outcomes. Grade I aGVHD is defined as Skin stage 1-2 and stage 0 for both GI and liver. Grade II is stage 3 skin, stage 1 GI, or stage 1 liver. Grade III is stage 2-3 GI or stage 2-3 liver. Grade IV is stage 4 skin or stage 4 liver. The cumulative incidence of acute GVHD grade II-IV and III-IV at Day 100 was estimated using the Aalen-Johansen estimator with 95% confidence intervals, treating death prior to aGVHD as a competing event. The cumulative incidences of Minnesota standard and high risk aGVHD at Day 100 were determined with 95% confidence intervals. Time to aGVHD is defined as time from transplant until onset of grades II-IV and III-IV aGVHD, respectively. A multivariate Cox regression model was used to compare the treatment groups, with adjustment for same baseline characteristics as for the primary endpoint. |
| Secondary | Participants With Maximum Acute GVHD at One Year Post-transplant | One year post-transplant | Acute GVHD were graded according to Mount Sinai Acute GVHD International Consortium (MAGIC) Criteria (Harris et al. Biology of Blood and Marrow Transplantation 2016; 22:4-10). The higher acute GVHD grade indicates worse outcomes. Grade 0 is no acute GVHD of any organ. Grade I acute GVHD is defined as Skin stage of 1-2 and stage 0 for both GI and liver organs. Grade II is stage 3 of skin, or stage 1 of GI, or stage 1 of liver. Grade III is stage 2-3 for GI, or stage 2-3 of liver. Grade IV is stage 4 of skin, or stage 4 of liver. Max acute GVHD by one-year post-transplant was computed. |
| Secondary | Participants With Maximum Stage of Skin, Lower GI, and Liver at Day 100 Post-transplant | Day 100 post-transplant | Organ stages were graded according to Mount Sinai Acute GVHD International Consortium (MAGIC) Criteria (Harris et al. 2016). Higher stage in any organ indicates worse outcomes. For skin, stage 0-no GVHD rash; 1-Maculopapular Rash \<25% BSA; 2-Maculopapular Rash 25-50% BSA; 3-Maculopapular Rash \>50% BSA; 4-Generalized Erythroderma Plus Bullous Formation and Desquamation \>5% BSA. For Lower GI, 0-No Diarrhea or Diarrhea-Adult: \<500 mL/day, \<3 Episodes/day; Child: \<10 mL/kg/day, \<4 Episodes/day; 1-Diarrhea-Adult: 500-999 mL/day, 3-4 Episodes/day; Child: 10-19.9 mL/kg/day, 4-6 Episodes/day; 2-Diarrhea-Adult: 1000-1500 mL/day, 5-7 Episodes/day; Child: 20-30 mL/kg/day, 7-10 Episodes/day; 3-Diarrhea-Adult: \>1500 mL/day, \>7 Episodes/day; Child: \>30 mL/kg/day, \>10 Episodes/day; 4-Severe Abdominal Pain With or Without Ileus or Grossly Bloody Stool. For liver, 0-Bilirubin \<2.0 mg/dL; 1-Bilirubin 2.0-3.0 mg/dL; 2-Bilirubin 3.1-6.0 mg/dL; 3-Bilirubin 6.1-15.0 mg/dL; 4-Bilirubin \>15.0 mg/dL |
| Secondary | Participants With Maximum Stage of Upper GI at Day 100 Post-transplant | Day 100 post-transplant | Organ stages were graded according to Mount Sinai Acute GVHD International Consortium (MAGIC) Criteria (Harris et al. 2016). Higher stage in any organ indicates worse outcomes. For upper GI, stage 0 is no or intermittent nausea, vomiting, or anorexia; stage 1 is persistent nausea, vomiting, or anorexia. The maximum stages of Upper GI at patient level were summarized at Day 100. |
| Secondary | Percentage of Participants With Chronic GVHD Post-transplant | 6, 12 months post-transplant | Percentage of participants with chronic GVHD (cGVHD) at one-year will be estimated with 95% confidence intervals for each treatment group using the cumulative incidence estimate with the complementary log-log transformation, treating death prior to cGVHD as a competing event. Chronic GVHD is based on NIH Consensus Criteria (2014 NIH Consensus Criteria) and includes mild, moderate and severe chronic GVHD. Eight organs were scored on a 0-3 scale to reflect degree of cGVHD involvement. Liver and pulmonary function test results and use of systemic therapy for treatment of cGVHD were also recorded. Assessment of cGVHD occurred up to one-year post-transplant. This endpoint considers any cGVHD onset. A multivariate Cox regression model for the cause-specific hazard of cGVHD was used to compare the treatment groups, after adjustment for baseline characteristics as described for the primary endpoint. |
| Secondary | Number of Participants Experiencing Chronic GVHD With Maximum Severity at 12 Months Post-transplant | 12 months post-transplant | Chronic GVHD data were collected directly from providers and chart review as defined by the NIH Consensus Conference Criteria. Eight organs were scored on a 0-3 scale to reflect degree of chronic GVHD involvement per the NIH global severity scores of mild, moderate and severe chronic GVHD. The maximum severity of chronic GVHD through 12 months post-transplant will be tabulated by treatment arm. |
| Secondary | Number of Participants With Immunosuppression-Free Survival (ISFS) at 1 Year Post-transplant | 1 year post-transplant | Participants who are alive, relapse-free, and do not need ongoing immune suppression (IS) to control GVHD at one year post-transplant are considered successes for the endpoint immunosuppression-free survival (ISFS). Immune suppression is defined as any systemic agents used to control or suppress GVHD. Corticosteroid doses greater than 10 mg were considered active systemic immune suppression treatment. Participants who discontinued immune suppression within 15 days or less prior to the 1-year time point was considered to be on immune suppression for this endpoint. |
| Secondary | Percentage of Participants With Immunosuppression-Free Survival (ISFS) at 1 Year Post-transplant | 1 year post-transplant | Participants who are alive, relapse-free, and do not need ongoing immune suppression to control GVHD at one year post-transplant are considered successes for the endpoint immunosuppression-free survival (ISFS). Percentage of participants alive, relapse free, and off immune suppression at 1 year post-transplant were described for each treatment group, along with 95% Clopper-Pearson confidence intervals. |
| Secondary | Percentage of Participants With Neutrophil Recovery Post-transplant | Days 28 and day 100 post-transplant | Neutrophil recovery is defined as achieving an absolute neutrophil count (ANC) greater than or equal to 500/mm\^3 for three consecutive measurements on three different days. The first of the three days will be designated the day of neutrophil recovery. The competing event is death without neutrophil recovery. For participants who never drop ANC below 500/mm\^3, the date of neutrophil recovery will be Day +1 post-transplant. The cumulative incidence of neutrophil recovery by Day 28 and Day 100 was described for each treatment group with point estimates and 95% confidence intervals using the complementary log-log transformation and treating death as a competing event. |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 registered outcomesOutcomes24
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants With GVHD/Relapse or Progression-free Survival (GRFS) at One Year | 1 year post randomization | Analysis Population includes all randomized participants | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Grades II-IV and III-IV Acute GVHD at Day 100 Post-transplant | Days 100 post-transplant | Analysis Population includes transplanted participants. | percentage of participants | NUMBER |
| SECONDARY | Participants With Maximum Acute GVHD at One Year Post-transplant | One year post-transplant | Analysis Population includes transplanted participants | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Participants With Maximum Stage of Skin, Lower GI, and Liver at Day 100 Post-transplant | Day 100 post-transplant | Analysis Population includes transplanted participants | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Participants With Maximum Stage of Upper GI at Day 100 Post-transplant | Day 100 post-transplant | Analysis Population includes transplanted participants | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Percentage of Participants With Chronic GVHD Post-transplant | 6, 12 months post-transplant | Analysis Population includes transplanted participants | percentage of participants | NUMBER |
| SECONDARY | Number of Participants Experiencing Chronic GVHD With Maximum Severity at 12 Months Post-transplant | 12 months post-transplant | Analysis Population includes transplanted participants | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Immunosuppression-Free Survival (ISFS) at 1 Year Post-transplant | 1 year post-transplant | Analysis Population includes transplanted and evaluable participants for ISFS | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Percentage of Participants With Immunosuppression-Free Survival (ISFS) at 1 Year Post-transplant | 1 year post-transplant | Analysis Population includes transplanted and evaluable participants for ISFS. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Neutrophil Recovery Post-transplant | Days 28 and day 100 post-transplant | Analysis Population includes transplanted participants | percentage of participants | NUMBER |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 outcomesResults by group220
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants With GVHD/Relapse or Progression-free Survival (GRFS) at One Year | PTCY/Tacrolimus/MMF | 52.3 | 95% Confidence Interval 45.2 to 58.8 | percentage of participants | |
| Percentage of Participants With GVHD/Relapse or Progression-free Survival (GRFS) at One Year | Tacrolimus/Methotrexate | 35.5 | 95% Confidence Interval 29.1 to 42.0 | percentage of participants | |
| Percentage of Participants With Grades II-IV and III-IV Acute GVHD at Day 100 Post-transplant | PTCY/Tacrolimus/MMF | MAGIC aGVHD Grade II-IV | 53.8 | 95% Confidence Interval 46.7 to 60.4 | percentage of participants |
| Percentage of Participants With Grades II-IV and III-IV Acute GVHD at Day 100 Post-transplant | Tacrolimus/Methotrexate | MAGIC aGVHD Grade II-IV | 51.9 | 95% Confidence Interval 44.9 to 58.4 | percentage of participants |
| Percentage of Participants With Grades II-IV and III-IV Acute GVHD at Day 100 Post-transplant | PTCY/Tacrolimus/MMF | MAGIC aGVHD Grade III-IV | 6.3 | 95% Confidence Interval 3.5 to 10.2 | percentage of participants |
| Percentage of Participants With Grades II-IV and III-IV Acute GVHD at Day 100 Post-transplant | Tacrolimus/Methotrexate | MAGIC aGVHD Grade III-IV | 14.7 | 95% Confidence Interval 10.3 to 19.8 | percentage of participants |
| Percentage of Participants With Grades II-IV and III-IV Acute GVHD at Day 100 Post-transplant | PTCY/Tacrolimus/MMF | Standard risk aGVHD | 64.1 | 95% Confidence Interval 57.1 to 70.3 | percentage of participants |
| Percentage of Participants With Grades II-IV and III-IV Acute GVHD at Day 100 Post-transplant | Tacrolimus/Methotrexate | Standard risk aGVHD | 64.3 | 95% Confidence Interval 57.4 to 70.4 | percentage of participants |
| Percentage of Participants With Grades II-IV and III-IV Acute GVHD at Day 100 Post-transplant | PTCY/Tacrolimus/MMF | High risk aGVHD | 13.1 | 95% Confidence Interval 8.9 to 18.1 | percentage of participants |
| Percentage of Participants With Grades II-IV and III-IV Acute GVHD at Day 100 Post-transplant | Tacrolimus/Methotrexate | High risk aGVHD | 18.5 | 95% Confidence Interval 13.6 to 24.0 | percentage of participants |
1 to 10 of 220Source ClinicalTrials.gov
See all 220 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | PTCY/Tacrolimus/MMF | 214 | |
| Overall Study | STARTED | Tacrolimus/Methotrexate | 217 | |
| Overall Study | COMPLETED | PTCY/Tacrolimus/MMF | 203 | |
| Overall Study | COMPLETED | Tacrolimus/Methotrexate | 208 | |
| Overall Study | NOT COMPLETED | PTCY/Tacrolimus/MMF | 11 | |
| Overall Study | NOT COMPLETED | Tacrolimus/Methotrexate | 9 |
Source ClinicalTrials.gov
Adverse events76
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 |
|---|---|---|---|---|---|---|
| other | Grades 3-5 General Disorders | General disorders | PTCY/Tacrolimus/MMF | 51 | 208 | 68 |
| other | Grades 3-5 General Disorders | General disorders | Tacrolimus/Methotrexate | 48 | 212 | 61 |
| other | Grades 3-5 Immune System Disorders | Immune system disorders | PTCY/Tacrolimus/MMF | 5 | 208 | 5 |
| other | Grades 3-5 Immune System Disorders | Immune system disorders | Tacrolimus/Methotrexate | 6 | 212 | 6 |
| other | Grades 3-5 GI Disorders | Gastrointestinal disorders | PTCY/Tacrolimus/MMF | 57 | 208 | 73 |
| other | Grades 3-5 GI Disorders | Gastrointestinal disorders | Tacrolimus/Methotrexate | 77 | 212 | 116 |
| other | Grades 3-5 Renal Disorders | Renal and urinary disorders | PTCY/Tacrolimus/MMF | 33 | 208 | 46 |
| other | Grades 3-5 Renal Disorders | Renal and urinary disorders | Tacrolimus/Methotrexate | 30 | 212 | 42 |
| other | Grades 3-5 Hemorrhagic Disorders | Vascular disorders | PTCY/Tacrolimus/MMF | 12 | 208 | 13 |
| other | Grades 3-5 Hemorrhagic Disorders | Vascular disorders | Tacrolimus/Methotrexate | 11 | 212 | 11 |
1 to 10 of 76Source ClinicalTrials.gov
See all 76 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | PTCY/Tacrolimus/MMF | 58 | 214 |
| Total, all-cause mortality | Tacrolimus/Methotrexate | 70 | 217 |
| Total, other adverse events | PTCY/Tacrolimus/MMF | 142 | 208 |
| Total, other adverse events | Tacrolimus/Methotrexate | 149 | 212 |
| Total, serious adverse events | PTCY/Tacrolimus/MMF | 7 | 208 |
| Total, serious adverse events | Tacrolimus/Methotrexate | 14 | 212 |
Source ClinicalTrials.gov
Papers3
The papers the trial lists on ClinicalTrials.gov.
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 |
|---|---|---|---|---|---|
| U10HL069294 | Blood & Marrow Transplant Clinical Trials Network Data Coordinating Center | HOROWITZ, MARY MARESCA | MEDICAL COLLEGE OF WISCONSIN | NATIONAL HEART, LUNG, AND BLOOD INSTITUTE | 2011 to 2016 |
Source NIH RePORTER
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| PTCY/Tacrolimus/MMF vs Tacrolimus/Methotrexate: deaths from any cause | Trial comparisons |
| PTCY/Tacrolimus/MMF vs Tacrolimus/Methotrexate: serious adverse events | 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.
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.
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