Trial · NCT02913261
Safety and Efficacy of Ruxolitinib Versus Best Available Therapy in Patients With Corticosteroid-refractory Acute Graft vs. Host Disease After Allogeneic Stem Cell Transplantation
NCT02913261, Phase 3, Completed, Novartis Pharmaceuticals; results first posted 2023-02-08
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Safety and Efficacy of Ruxolitinib Versus Best Available Therapy in Patients With Corticosteroid-refractory Acute Graft vs. Host Disease After Allogeneic Stem Cell Transplantation. Trials, record NCT02913261. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02913261
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety and Efficacy of Ruxolitinib Versus Best Available Therapy in Patients With Corticosteroid-refractory Acute Graft vs. Host Disease After Allogeneic Stem Cell Transplantation
- NCT number
- NCT02913261
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 310 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Corticosteroid Refractory Acute Graft vs Host Disease
- Interventions
- Ruxolitinib (RUX), Best Available Therapy (BAT)
- First posted
- 2016-09-23
- Last updated
- 2023-02-08
- Other registries' IDs
- eudract:2016-002584-33
- Results first posted
- 2023-02-08
Source ClinicalTrials.gov
Registered outcomes26
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Overall Response Rate (ORR) at Day 28 | Day 28 | Overall response rate at Day 28 after randomization was defined as the percentage participants in each arm demonstrating a complete response (CR) or partial response (PR), based on investigator assessment \& according to standard criteria, without requirement for additional systemic therapies for an earlier progression, mixed response or non-response. Scoring of response was relative to the organ stage at the time of randomization. CR was defined as a score of 0 for the aGvHD grading in all evaluable organs that indicates complete resolution of all signs \& symptoms of aGvHD in all evaluable organs without administration of additional systemic therapies for any earlier progression, mixed response or non-response of aGvHD. PR was defined as improvement of 1 stage in 1 or more organs involved with aGvHD signs or symptoms without progression in other organs or sites without administration of additional systemic therapies for an earlier progression, mixed response or non-response of aGvHD. |
| Secondary | Durable Overall Response Rate (DORR) (Key Secondary Endpoint) at Day 56 | Day 56 | Percentage of all participants in each arm who achieved a complete response (CR) or partial response (PR) at Day 28 (primary endpoint) AND maintained a CR or PR at Day 56 based on investigator assessment and according to standard criteria. CR was defined as a score of 0 for the aGvHD grading in all evaluable organs that indicates complete resolution of all signs and symptoms of aGvHD in all evaluable organs without administration of additional systemic therapies for any earlier progression, mixed response or non-response of aGvHD. PR was defined as improvement of 1 stage in 1 or more organs involved with aGvHD signs or symptoms without progression in other organs or sites without administration of additional systemic therapies for an earlier progression, mixed response or non-response of aGvHD. |
| Secondary | Overall Response Rate (ORR) at Day 14 | Day 14 | ORR at Da4 14 is the percentage of participants who achieved overall response (CR+PR) at Day 14 based on investigator assessment and according to standard criteria. CR was defined as a score of 0 for the aGvHD grading in all evaluable organs that indicates complete resolution of all signs and symptoms of aGvHD in all evaluable organs without administration of additional systemic therapies for any earlier progression, mixed response or non-response of aGvHD. PR was defined as improvement of 1 stage in 1 or more organs involved with aGvHD signs or symptoms without progression in other organs or sites without administration of additional systemic therapies for an earlier progression, mixed response or non-response of aGvHD. |
| Secondary | Duration of Response (DOR) | Up to 24 months | Duration of response was defined for patients who had a CR or PR at Day 28. This was the interval between the date of first documented response of CR or PR (i.e., the start date of response), till the date of progression or addition of systemic therapies for aGvHD on or after Day 28. Death without prior observation of aGvHD progression and onset of chronic GvHD were considered. Duration of response was censored at the last response assessment prior to or at the analysis cut-off date, if no events/competing risk occurred before or at the cut-off date. |
| Secondary | Cumulative Steroid Dosing Until Day 56 | up to Day 56 | Weekly cumulative steroid dose for each participant up to Day 56 or discontinuation of randomized treatment. Participants should have undergone tapering of steroids if it had been required. Tapering the immunosuppression therapy was performed in 2 steps: Taper of corticosteroids: initiated not earlier than Day 7, and performed as per institutional guidelines. Only patients with assessments done at each time point are reported. This is the reason that the overall number per treatment is higher and the number of participants with responses vary over time. |
| Secondary | Kaplan Meier Estimates of Probability of Overall Survival (OS) by Time Interval | 1, 2, 6, 12, 18 & 24 months | OS was defined as the time from the date of randomization to date of death due to any cause. If a patient was not known to have died, then OS was censored at the latest date the patient was known to be alive (on or before the cut-off date). Results are based on Kaplan Meier (KM) estimates. |
| Secondary | Kaplan Meier Estimates of Probability of Event-free Survival (EFS) by Time Interval | 1, 2, 6, 12, 18 & 24 months | Event-free survival was defined as the time from the date of randomization to the date of hematologic disease relapse/progression, graft failure, or death due to any cause. If a patient was not known to have any event, then EFS was censored at the latest date the patient was known to be alive (on or before the cut-off date). Results are based on Kaplan Meier (KM) estimates. |
| Secondary | Cumulative Incidence Rate of Failure-Free Survival (FFS) | 1, 2, 6, 12, 18, & 24 Months | FFS was defined as the time from the date of randomization to date of hematologic disease relapse/progression, non-relapse mortality, or addition of new systemic aGvHD treatment. Probability of FFS with 95% CIs are presented for each treatment group, accounting for onset of chronic GvHD as the competing risk. |
| Secondary | Cumulative Probability of Non Relapse Mortality (NRM) | 1, 2, 6, 12, 18 & 24 months | NRM was defined as the time from date of randomization to date of death not preceded by hematologic disease relapse/progression. Hematologic disease relapse/progression was considered a competing risk for NRM with the date of hematologic disease relapse/progression being the earlier of documented hematologic disease relapse/progression or institution of therapy to treat potential hematologic disease relapse/progression. If a patient was not known to have died or to have relapsed/progressed, then NRM was censored at the latest date the patient was known to be alive (on or before the cut-off date). Data is provided based on cumulative probability of hematologic disease relapse/progression. |
| Secondary | Cumulative Probability of Malignancy Relapse/Progression (MR) | 1, 2, 6, 12 , 18 & 24 months | MR was defined as the time from date of randomization to hematologic malignancy relapse/progression. Deaths not preceded by hematologic malignancy relapse/progression were considered competing risks. If a patient was not known to have event or competing risks, then MR was censored at the latest date the patient was known to be alive (on or before the cut-off date). Calculated for patients with underlying hematologic malignant disease. |
1 to 10 of 26Source ClinicalTrials.gov
See all 26 registered outcomesOutcomes27
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Overall Response Rate (ORR) at Day 28 | Day 28 | The Full Analysis Set (FAS) comprised all patients to whom study treatment was assigned by randomization. | Percentage of participants | NUMBER |
| SECONDARY | Durable Overall Response Rate (DORR) (Key Secondary Endpoint) at Day 56 | Day 56 | The Full Analysis Set (FAS) comprised all patients to whom study treatment was assigned by randomization. | Percentage of participants | NUMBER |
| SECONDARY | Overall Response Rate (ORR) at Day 14 | Day 14 | The Full Analysis Set (FAS) comprised all patients to whom study treatment was assigned by randomization. | Percentage of participants | NUMBER |
| SECONDARY | Duration of Response (DOR) | Up to 24 months | The Full Analysis Set (FAS) comprised all patients to whom study treatment was assigned by randomization. These were patients to whom study treatment was assigned by randomization and whose overall response was complete response (CR) or partial response (PR). | Days | MEDIAN |
| SECONDARY | Cumulative Steroid Dosing Until Day 56 | up to Day 56 | The Full Analysis Set (FAS) comprised all patients to whom study treatment was assigned by randomization. | mg | MEDIAN |
| SECONDARY | Kaplan Meier Estimates of Probability of Overall Survival (OS) by Time Interval | 1, 2, 6, 12, 18 & 24 months | The Full Analysis Set (FAS) comprised all patients to whom study treatment was assigned by randomization. | Percentage of participants | NUMBER |
| SECONDARY | Kaplan Meier Estimates of Probability of Event-free Survival (EFS) by Time Interval | 1, 2, 6, 12, 18 & 24 months | The Full Analysis Set (FAS) comprised all patients to whom study treatment was assigned by randomization. | Percentage of participants | NUMBER |
| SECONDARY | Cumulative Incidence Rate of Failure-Free Survival (FFS) | 1, 2, 6, 12, 18, & 24 Months | The Full Analysis Set (FAS) comprised all patients to whom study treatment was assigned by randomization. | Cumulative probability of FFS | NUMBER |
| SECONDARY | Cumulative Probability of Non Relapse Mortality (NRM) | 1, 2, 6, 12, 18 & 24 months | The Full Analysis Set (FAS) comprised all patients to whom study treatment was assigned by randomization. | Cumulative probability of NRM | NUMBER |
| SECONDARY | Cumulative Probability of Malignancy Relapse/Progression (MR) | 1, 2, 6, 12 , 18 & 24 months | The Full Analysis Set (FAS) comprised all patients to whom study treatment was assigned by randomization. Calculated for patients with underlying hematologic malignant disease. | Cumulative probability of MR | NUMBER |
1 to 10 of 27Source ClinicalTrials.gov
See all 27 outcomesResults by group139
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Overall Response Rate (ORR) at Day 28 | Ruxolitinib (RUX) | 62.3 | 95% Confidence Interval 54.2 to 70.0 | Percentage of participants | |
| Overall Response Rate (ORR) at Day 28 | Best Available Therapy (BAT) | 39.4 | 95% Confidence Interval 31.6 to 47.5 | Percentage of participants | |
| Durable Overall Response Rate (DORR) (Key Secondary Endpoint) at Day 56 | Ruxolitinib (RUX) | 39.6 | 95% Confidence Interval 31.8 to 47.8 | Percentage of participants | |
| Durable Overall Response Rate (DORR) (Key Secondary Endpoint) at Day 56 | Best Available Therapy (BAT) | 21.9 | 95% Confidence Interval 15.7 to 29.3 | Percentage of participants | |
| Overall Response Rate (ORR) at Day 14 | Ruxolitinib (RUX) | 63.0 | 95% Confidence Interval 54.8 to 70.6 | Percentage of participants | |
| Overall Response Rate (ORR) at Day 14 | Best Available Therapy (BAT) | 47.1 | 95% Confidence Interval 39.0 to 55.3 | Percentage of participants | |
| Duration of Response (DOR) | Ruxolitinib (RUX) | 167.0 | Full Range 22.0 to 677.0 | Days | |
| Duration of Response (DOR) | Best Available Therapy (BAT) | 106.0 | Full Range 10.0 to 526.0 | Days | |
| Cumulative Steroid Dosing Until Day 56 | Ruxolitinib (RUX) | By Week 1 | 962.5 | Full Range 140.0 to 2337.5 | mg |
| Cumulative Steroid Dosing Until Day 56 | Best Available Therapy (BAT) | By Week 1 | 923.6 | Full Range 140.0 to 6093.8 | mg |
1 to 10 of 139Source ClinicalTrials.gov
See all 139 results by groupParticipant flow16
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Ruxolitinib (RUX) | 154 | |
| Overall Study | STARTED | Best Available Therapy (BAT) | 155 | |
| Overall Study | Not Treated | Ruxolitinib (RUX) | 2 | |
| Overall Study | Not Treated | Best Available Therapy (BAT) | 5 | |
| Overall Study | Crossover Treatment at End of Randomized Treatment | Ruxolitinib (RUX) | 0 | |
| Overall Study | Crossover Treatment at End of Randomized Treatment | Best Available Therapy (BAT) | 49 | |
| Overall Study | Entered Long-term Follow-up | Ruxolitinib (RUX) | 102 | |
| Overall Study | Entered Long-term Follow-up | Best Available Therapy (BAT) | 51 | |
| Overall Study | Completed Screening & Randomized | Ruxolitinib (RUX) | 154 | |
| Overall Study | Completed Screening & Randomized | Best Available Therapy (BAT) | 155 |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 participant flowAdverse events846
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 | Anaemia | Blood and lymphatic system disorders | Ruxolitinib (Rux) | 61 | 152 | |
| other | Anaemia | Blood and lymphatic system disorders | Best Available Therapy (BAT) | 47 | 150 | |
| other | Anaemia | Blood and lymphatic system disorders | Cross-Over | 16 | 49 | |
| other | Leukopenia | Blood and lymphatic system disorders | Ruxolitinib (Rux) | 14 | 152 | |
| other | Leukopenia | Blood and lymphatic system disorders | Best Available Therapy (BAT) | 2 | 150 | |
| other | Leukopenia | Blood and lymphatic system disorders | Cross-Over | 3 | 49 | |
| other | Neutropenia | Blood and lymphatic system disorders | Ruxolitinib (Rux) | 36 | 152 | |
| other | Neutropenia | Blood and lymphatic system disorders | Best Available Therapy (BAT) | 19 | 150 | |
| other | Neutropenia | Blood and lymphatic system disorders | Cross-Over | 10 | 49 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Ruxolitinib (Rux) | 56 | 152 |
1 to 10 of 846Source ClinicalTrials.gov
See all 846 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Ruxolitinib (Rux) | 89 | 152 |
| Total, all-cause mortality | Best Available Therapy (BAT) | 89 | 150 |
| Total, all-cause mortality | Cross-Over | 29 | 49 |
| Total, other adverse events | Ruxolitinib (Rux) | 144 | 152 |
| Total, other adverse events | Best Available Therapy (BAT) | 136 | 150 |
| Total, other adverse events | Cross-Over | 43 | 49 |
| Total, serious adverse events | Ruxolitinib (Rux) | 101 | 152 |
| Total, serious adverse events | Best Available Therapy (BAT) | 80 | 150 |
| Total, serious adverse events | Cross-Over | 38 | 49 |
Source ClinicalTrials.gov
Papers6
The papers the trial lists on ClinicalTrials.gov.
Grants0
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.
None in this release.
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.
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