Trial · NCT02345850
Calcineurin Inhibitor-Free Interventions BMT CTN 1301 for Prevention of Graft-versus-Host Disease (BMT CTN 1301)
NCT02345850, Phase 3, Completed, National Heart, Lung, and Blood Institute (NHLBI); results first posted 2021-12-21
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Calcineurin Inhibitor-Free Interventions BMT CTN 1301 for Prevention of Graft-versus-Host Disease (BMT CTN 1301). Trials, record NCT02345850. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02345850
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Calcineurin Inhibitor-Free Interventions BMT CTN 1301 for Prevention of Graft-versus-Host Disease (BMT CTN 1301)
- NCT number
- NCT02345850
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 346 participants, actual
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- Conditions
- Acute Leukemia, Myelodysplasia
- Interventions
Unmanipulated Bone Marrow Graft with Tacrolimus/Methotrexate, Mobilized CD34-selected Peripheral Blood Stem Cell graft, Unmanipulated Bone Marrow Graft with Cyclophosphamide, Cyclophosphamide, Tacrolimus, Methotrexate
- First posted
- 2015-01-26
- Last updated
- 2023-03-07
- Results first posted
- 2021-12-21
Source ClinicalTrials.gov
Registered outcomes21
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Chronic GVHD-free, Relapse-free Survival (CRFS) Probability | 2 years | The primary endpoint of the trial is Chronic GVHD/Relapse-Free Survival (CRFS), treated as a time to event variable. An event for this time to event outcome is defined as moderate to severe chronic GVHD, disease relapse, or death by any cause. Participant will be censored if lost to follow up prior to 2 years. Time is from randomization to the event of moderate to severe chronic GVHD, disease relapse, death, last follow up, or 2 years, whichever comes first. The primary analysis is performed using the intent-to-treat principle (ITT) so that all randomized patients are included in the analysis. |
| Secondary | Percentage of Participants With Overall Survival (OS) | 2 Years | OS is a key secondary endpoint, with explicit control of the type I error rate through a gatekeeper approach. Formal significance testing of OS between a CNI-free strategy and the control will be conducted if the corresponding CRFS comparison is significant. This OS comparison will be done using a Bonferroni adjusted significance level of 0.05/3 to account for three potential CNI-free comparisons to the control. Otherwise, survival analyses will be considered exploratory. Death from any cause is considered as event for this endpoint. Participant is censored if lost to follow up. |
| Secondary | Percentage of Participants With Relapse-free Survival | 2 Years | The events for this endpoint RFS are death and relapse of the underlying malignancy. The analyses of this endpoint use the transplanted populations and time is from transplant to the event of disease relapse or death, or last follow up, whichever comes first. |
| Secondary | Percentage of Participants With Treatment-related Mortality | 2 Years | The events for this endpoint TRM are deaths prior to relapse of the underlying malignancy. The analyses of this endpoint will use the transplanted populations, and time will be from transplant to the first of disease relapse, death, or last follow up. TRM are evaluated using the cumulative incidence function. Deaths without relapse are the events for this endpoint and relapse is a competing risk for this endpoint. |
| Secondary | Participants With Immunosuppression-free Survival | 1 Year | Patients who are alive, relapse-free, and do not need ongoing immune suppression to control GVHD at one year post HSCT are considered successes for this endpoint. Immune suppression is defined as any systemic agents used to control or suppress GVHD. |
| Secondary | Percentage of Participants With Disease Relapse | 2 Years | Relapse is defined by either morphological evidence of acute leukemia or MDS consistent with pre-transplant features, or radiologic evidence of lymphoma, documented or not by biopsy. The event is defined as increase in size of prior sites of disease or evidence of new sites of disease, documented or not by biopsy. Relapse is adjudicated by ERC. Disease relapse is analyzed using cumulative incidence function with death as a competing risk. The analyses of this endpoint use the transplanted populations, and the time will be measured from transplant to the earliest of death, relapse/progression, or last follow up. |
| Secondary | Percentage of Participants With Neutrophil Engraftment | Day 28 | Neutrophil recovery is defined as achieving an absolute neutrophil count (ANC) ≥ 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. |
| Secondary | Percentage of Participants With Platelet Recovery | Day 60 | Platelet recovery is defined as the first day of a sustained platelet count \>20,000/mm\^3 with no platelet transfusion in the preceding seven days. The first day of sustained platelet count above this threshold will be designated the day of platelet engraftment. The competing event is death without platelet recovery. |
| Secondary | Participants With Primary Graft Failure | Day 28 | Primary graft failure is defined as no neutrophil recovery to \> 500 cells/µL by Day 28 post HSCT. |
| Secondary | Percentage of Participants With Secondary Graft Failure | 2 Years | Secondary graft failure will be assessed according to neutrophil count after initial hematologic recovery. Secondary graft failure is defined as initial neutrophil engraftment followed by subsequent decline in absolute neutrophil counts \< 500 cells/µL, unresponsive to growth factor therapy, but cannot be explained by disease relapse or medications. Secondary graft failure will be analyzed using cumulative incidence function with death as a competing risk. |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 registered outcomesOutcomes21
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Chronic GVHD-free, Relapse-free Survival (CRFS) Probability | 2 years | All randomized patients are analyzed for this endpoint. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Overall Survival (OS) | 2 Years | The randomized or transplanted participants are included in the analyses. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Relapse-free Survival | 2 Years | The analyses of this endpoint will use the transplanted population. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Treatment-related Mortality | 2 Years | The analyses of this endpoint will use the transplanted populations. | percentage of participants | NUMBER |
| SECONDARY | Participants With Immunosuppression-free Survival | 1 Year | The analyses of this endpoint will use the transplanted populations. Two participant of CD34 Selected Graft arm and one participants of Post-Transplant Cyclophosphamide arm were lost to follow-up while alive and not relapsed, and they are considered as not evaluable for this endpoint. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Percentage of Participants With Disease Relapse | 2 Years | The analyses of this endpoint use the transplanted populations. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Neutrophil Engraftment | Day 28 | The analyses of the endpoint use the transplanted populations. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Platelet Recovery | Day 60 | The analyses of the endpoint use the transplanted populations. Three transplanted participants (one from the CD34 arm and two from the PTCy arm) are missing platelet data and are not included in the analyses. | percentage of participants | NUMBER |
| SECONDARY | Participants With Primary Graft Failure | Day 28 | The analyses of the endpoint use the transplanted populations. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Percentage of Participants With Secondary Graft Failure | 2 Years | The analyses of the endpoint use the transplanted populations. | percentage of participants | NUMBER |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 outcomesResults by group287
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Chronic GVHD-free, Relapse-free Survival (CRFS) Probability | CD34 Selected Graft | 1 year Post Randomization | 60.2 | 95% Confidence Interval 50.3 to 68.7 | percentage of participants |
| Chronic GVHD-free, Relapse-free Survival (CRFS) Probability | Post-Transplant Cyclophosphamide | 1 year Post Randomization | 60.3 | 95% Confidence Interval 50.5 to 68.7 | percentage of participants |
| Chronic GVHD-free, Relapse-free Survival (CRFS) Probability | Tacrolimus/Methotrexate Control | 1 year Post Randomization | 52.6 | 95% Confidence Interval 43.1 to 61.3 | percentage of participants |
| Chronic GVHD-free, Relapse-free Survival (CRFS) Probability | CD34 Selected Graft | 2 years Post Randomization | 50.6 | 95% Confidence Interval 40.8 to 59.6 | percentage of participants |
| Chronic GVHD-free, Relapse-free Survival (CRFS) Probability | Post-Transplant Cyclophosphamide | 2 years Post Randomization | 48.1 | 95% Confidence Interval 38.5 to 57.1 | percentage of participants |
| Chronic GVHD-free, Relapse-free Survival (CRFS) Probability | Tacrolimus/Methotrexate Control | 2 years Post Randomization | 41.0 | 95% Confidence Interval 32.0 to 49.9 | percentage of participants |
| Percentage of Participants With Overall Survival (OS) | CD34 Selected Graft | 1 year post-randomization | 75.7 | 95% Confidence Interval 66.4 to 82.8 | percentage of participants |
| Percentage of Participants With Overall Survival (OS) | Post-Transplant Cyclophosphamide | 1 year post-randomization | 84.6 | 95% Confidence Interval 76.4 to 90.1 | percentage of participants |
| Percentage of Participants With Overall Survival (OS) | Tacrolimus/Methotrexate Control | 1 year post-randomization | 84.2 | 95% Confidence Interval 76.1 to 89.7 | percentage of participants |
| Percentage of Participants With Overall Survival (OS) | CD34 Selected Graft | 2 year post-randomization | 60.1 | 95% Confidence Interval 50.1 to 68.8 | percentage of participants |
1 to 10 of 287Source ClinicalTrials.gov
See all 287 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | CD34 Selected Graft | 114 | |
| Overall Study | STARTED | Post-Transplant Cyclophosphamide | 114 | |
| Overall Study | STARTED | Tacrolimus/Methotrexate Control | 118 | |
| Overall Study | COMPLETED | CD34 Selected Graft | 101 | |
| Overall Study | COMPLETED | Post-Transplant Cyclophosphamide | 106 | |
| Overall Study | COMPLETED | Tacrolimus/Methotrexate Control | 112 | |
| Overall Study | NOT COMPLETED | CD34 Selected Graft | 13 | |
| Overall Study | NOT COMPLETED | Post-Transplant Cyclophosphamide | 8 | |
| Overall Study | NOT COMPLETED | Tacrolimus/Methotrexate Control | 6 |
Source ClinicalTrials.gov
Adverse events81
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 | FOLLICULAR LYMPHOMA | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | CD34 Selected Graft | 0 | 114 | 0 |
| other | FOLLICULAR LYMPHOMA | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Post-Transplant Cyclophosphamide | 0 | 114 | 0 |
| other | FOLLICULAR LYMPHOMA | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Tacrolimus/Methotrexate Control | 1 | 118 | 1 |
| other | RESPIRATORY FAILURE | Respiratory, thoracic and mediastinal disorders | CD34 Selected Graft | 1 | 114 | 1 |
| other | RESPIRATORY FAILURE | Respiratory, thoracic and mediastinal disorders | Post-Transplant Cyclophosphamide | 0 | 114 | 0 |
| other | RESPIRATORY FAILURE | Respiratory, thoracic and mediastinal disorders | Tacrolimus/Methotrexate Control | 0 | 118 | 0 |
| other | ARTHRALGIA | Musculoskeletal and connective tissue disorders | CD34 Selected Graft | 0 | 114 | 0 |
| other | ARTHRALGIA | Musculoskeletal and connective tissue disorders | Post-Transplant Cyclophosphamide | 0 | 114 | 0 |
| other | ARTHRALGIA | Musculoskeletal and connective tissue disorders | Tacrolimus/Methotrexate Control | 1 | 118 | 1 |
| other | ELEVATED ENDOTOXIN LEVEL | Product Issues | CD34 Selected Graft | 1 | 114 | 1 |
1 to 10 of 81Source ClinicalTrials.gov
See all 81 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | CD34 Selected Graft | 42 | 114 |
| Total, all-cause mortality | Post-Transplant Cyclophosphamide | 27 | 114 |
| Total, all-cause mortality | Tacrolimus/Methotrexate Control | 30 | 118 |
| Total, other adverse events | CD34 Selected Graft | 3 | 114 |
| Total, other adverse events | Post-Transplant Cyclophosphamide | 1 | 114 |
| Total, other adverse events | Tacrolimus/Methotrexate Control | 2 | 118 |
| Total, serious adverse events | CD34 Selected Graft | 9 | 114 |
| Total, serious adverse events | Post-Transplant Cyclophosphamide | 7 | 114 |
| Total, serious adverse events | Tacrolimus/Methotrexate Control | 7 | 118 |
Source ClinicalTrials.gov
Papers2
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 |
|---|---|---|---|---|---|
| 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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02345850
GET https://conductscience.org/api/trials/v1/NCT02345850/registered?page=2