Trial · NCT02806947
A Study to Evaluate Steroid-free Treatment for Standard-Risk aGVHD (BMT CTN 1501)
NCT02806947, Phase 2, Completed, Medical College of Wisconsin; results first posted 2019-12-17
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study to Evaluate Steroid-free Treatment for Standard-Risk aGVHD (BMT CTN 1501). Trials, record NCT02806947. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02806947
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Evaluate Steroid-free Treatment for Standard-Risk aGVHD (BMT CTN 1501)
- NCT number
- NCT02806947
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 127 participants, actual
- Lead sponsor
- Medical College of Wisconsin
- Conditions
- Acute GVHD
- Interventions
- Sirolimus, Prednisone
- First posted
- 2016-06-21
- Last updated
- 2021-11-01
- Results first posted
- 2019-12-17
Source ClinicalTrials.gov
Registered outcomes12
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants With Complete or Partial Response (CR/PR) to Acute GVHD Treatment | Days 28 and 56 Post-randomization | Scoring of CR/PR is in comparison to the participant's acute GVHD status at randomization. Complete response (CR) is defined as staging of 0 for in all target organs for GVHD - skin, GI tract, and liver. Partial response (PR) is defined as improvement in some target organ(s) without worsening in others. Death and initiation of systemic acute GVHD treatment beyond randomized treatment are considered failures for this endpoint. Organ staging is defined below: Skin stage: 0: No rash 1. Rash \<25% of body surface area (BSA) 2. Rash on 25-50% of BSA 3. Rash on \>50% of BSA 4. Generalized erythroderma with bullous formation Liver stage (based on bilirubin level): 0: \<2 mg/dL 1. 2-3 mg/dL 2. 3.01-6 mg/dL 3. 6.01-15.0 mg/dL 4. \>15 mg/dL GI stage: 0: No diarrhea or diarrhea \<500 mL/day 1. Diarrhea 500-999 mL/day or persistent nausea with histologic evidence of GVHD 2. Diarrhea 1000-1499 mL/day 3. Diarrhea \>1500 mL/day 4. Severe abdominal pain with or without ileus |
| Secondary | Percentage of Participants With Complete or Partial Response (CR/PR) and Steroid Dose Less Than 0.25 mg/kg Per Day | Day 28 Post-randomization | The proportion of patients with CR/PR and on a prednisone-equivalent steroid dose of 0.25 mg/kg/day or less is evaluated. CR/PR scoring is in comparison to acute GVHD status at randomization. CR is defined as staging of 0 in all target organs. PR is defined as improvement in some organ(s) without worsening in others. Death and initiation of steroid-free, systemic acute GVHD treatment beyond randomized therapy are considered failures for this endpoint. Organ staging is defined as: Skin stage: 0: No rash 1. Rash \<25% of body surface area (BSA) 2. Rash 25-50% of BSA 3. Rash \>50% of BSA 4. Generalized erythroderma with bullous formation Liver stage (based on bilirubin level in mg/dL): 0: \<2 1. 2-3 2. 3.01-6 3. 6.01-15.0 4. \>15 mg/dL GI stage: 0: No diarrhea or diarrhea \<500 mL/day 1. Diarrhea 500-999 mL/day or persistent nausea with histologic evidence of GVHD 2. Diarrhea 1000-1499 mL/day 3. Diarrhea \>1500 mL/day 4. Severe abdominal pain with or without ileus |
| Secondary | Acute GVHD Response | Days 28 and 56 Post-randomization | Acute GVHD response is classified as CR, PR, mixed response (MR), no response (NR), and progression and scored by comparison to acute GVHD status at randomization. MR is defined as improvement in some organ(s) with worsening in another, progression as worsening in some organ(s) without improvement in others, and NR as absence of any improvement or worsening. Death and initiation of systemic acute GVHD treatment beyond randomized treatment are classified as NR. Organ staging is defined as: Skin stage: 0: No rash 1. Rash \<25% of body surface area (BSA) 2. Rash 25-50% of BSA 3. Rash \>50% of BSA 4. Generalized erythroderma with bullous formation Liver stage (based on bilirubin level in mg/dL): 0: \<2 1. 2-3 2. 3.01-6 3. 6.01-15.0 4. \>15 GI stage: 0: No diarrhea or diarrhea \<500 mL/day 1. Diarrhea 500-999 mL/day or persistent nausea with histologic evidence of GVHD 2. Diarrhea 1000-1499 mL/day 3. Diarrhea \>1500 mL/day 4. Severe abdominal pain with or without ileus |
| Secondary | Percentage of Participants With Treatment Failure | Days 28 and 56 Post-randomization | Treatment failure is defined as either no response (NR) or progression and scored by comparison to acute GVHD status at randomization. Progression is defined as worsening in some target organ(s) without improvement in others and NR is defined as absence of any improvement or worsening in target organs. Death and initiation of systemic acute GVHD treatment beyond randomized treatment are classified as NR. Organ staging is defined as: Skin stage: 0: No rash 1. Rash \<25% of body surface area (BSA) 2. Rash 25-50% of BSA 3. Rash \>50% of BSA 4. Generalized erythroderma with bullous formation Liver stage (based on bilirubin level in mg/dL): 0: \<2 1. 2-3 2. 3.01-6 3. 6.01-15.0 4. \>15 GI stage: 0: No diarrhea or diarrhea \<500 mL/day 1. Diarrhea 500-999 mL/day or persistent nausea with histologic evidence of GVHD 2. Diarrhea 1000-1499 mL/day 3. Diarrhea \>1500 mL/day 4. Severe abdominal pain with or without ileus |
| Secondary | Percentage of Participants With Overall Survival | 6 and 12 Months Post-randomization | Overall survival is defined as survival of death from any cause. |
| Secondary | Percentage of Participants With Disease-free Survival | 6 and 12 Months Post-randomization | Disease-free survival is defined as freedom from death and relapse of the underlying malignancy. |
| Secondary | Proportion of Participants With Event-free Survival | 6 and 12 Months Post-randomization | Event-free survival is defined as freedom from acute GVHD progression, chronic GVHD, malignancy relapse, and death. |
| Secondary | Percentage of Participants With Non-relapse Mortality | 6 and 12 Months Post-randomization | Non-relapse mortality is defined as death due to any cause other than relapse of the underlying malignancy. The cumulative incidence of non-relapse mortality is described, with malignancy relapse treated as a competing risk. |
| Secondary | Percentage of Participants With Malignancy Relapse | 6 and 12 Months Post-randomization | The cumulative incidence of relapse of the primary malignancy is described, with death treated as a competing risk. |
| Secondary | Percentage of Participants With Chronic GVHD | 6 and 12 Months Post-randomization | Chronic GVHD is classified per 2005 NIH Consensus Criteria (Filipovich et al. 2005) into categories of severity: none, mild, moderate, and severe. Occurrence of chronic GVHD is defined as the occurrence of mild, moderate, or severe chronic GVHD per this classification. The cumulative incidence of chronic GVHD is described, with death and malignancy relapse treated as competing risks. |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 registered outcomesOutcomes12
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 Complete or Partial Response (CR/PR) to Acute GVHD Treatment | Days 28 and 56 Post-randomization | CR/PR was evaluated in participants remaining on study until the assessment time point. At Day 28, one prednisone and four sirolimus arm participants were excluded from the analysis due to prior study withdrawal. At Day 56, one prednisone and five sirolimus arm participants were excluded from the analysis due to prior study withdrawal. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Percentage of Participants With Complete or Partial Response (CR/PR) and Steroid Dose Less Than 0.25 mg/kg Per Day | Day 28 Post-randomization | The endpoint was evaluated only in participants that remained on study until Day 28. One participant on the prednisone arm and four on the sirolimus arm were deemed unevaluable at Day 28 due to prior study withdrawal. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Acute GVHD Response | Days 28 and 56 Post-randomization | Acute GVHD response was evaluated in participants remaining on study until the assessment day. At Day 28, one prednisone and four sirolimus arm participants were excluded from the analysis due to prior study withdrawal. At Day 56, one prednisone and five sirolimus arm participants were excluded from the analysis due to prior study withdrawal. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Percentage of Participants With Treatment Failure | Days 28 and 56 Post-randomization | Treatment failure was evaluated in participants remaining on study until the assessment day. At Day 28, one prednisone and four sirolimus arm participants were excluded from the analysis due to prior study withdrawal. At Day 56, one prednisone and five sirolimus arm participants were excluded from the analysis due to prior study withdrawal. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Percentage of Participants With Overall Survival | 6 and 12 Months Post-randomization | percentage of participants | NUMBER | |
| SECONDARY | Percentage of Participants With Disease-free Survival | 6 and 12 Months Post-randomization | percentage of participants | NUMBER | |
| SECONDARY | Proportion of Participants With Event-free Survival | 6 and 12 Months Post-randomization | percentage of participants | NUMBER | |
| SECONDARY | Percentage of Participants With Non-relapse Mortality | 6 and 12 Months Post-randomization | percentage of participants | NUMBER | |
| SECONDARY | Percentage of Participants With Malignancy Relapse | 6 and 12 Months Post-randomization | percentage of participants | NUMBER | |
| SECONDARY | Percentage of Participants With Chronic GVHD | 6 and 12 Months Post-randomization | percentage of participants | NUMBER |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 outcomesResults by group72
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 Complete or Partial Response (CR/PR) to Acute GVHD Treatment | Sirolimus | CR/PR at Day 28 / Yes | 35 | Participants | |
| Percentage of Participants With Complete or Partial Response (CR/PR) to Acute GVHD Treatment | Prednisone | CR/PR at Day 28 / Yes | 46 | Participants | |
| Percentage of Participants With Complete or Partial Response (CR/PR) to Acute GVHD Treatment | Sirolimus | CR/PR at Day 28 / No | 19 | Participants | |
| Percentage of Participants With Complete or Partial Response (CR/PR) to Acute GVHD Treatment | Prednisone | CR/PR at Day 28 / No | 17 | Participants | |
| Percentage of Participants With Complete or Partial Response (CR/PR) to Acute GVHD Treatment | Sirolimus | CR/PR at Day 56 / Yes | 34 | Participants | |
| Percentage of Participants With Complete or Partial Response (CR/PR) to Acute GVHD Treatment | Prednisone | CR/PR at Day 56 / Yes | 50 | Participants | |
| Percentage of Participants With Complete or Partial Response (CR/PR) to Acute GVHD Treatment | Sirolimus | CR/PR at Day 56 / No | 19 | Participants | |
| Percentage of Participants With Complete or Partial Response (CR/PR) to Acute GVHD Treatment | Prednisone | CR/PR at Day 56 / No | 13 | Participants | |
| Percentage of Participants With Complete or Partial Response (CR/PR) and Steroid Dose Less Than 0.25 mg/kg Per Day | Sirolimus | Yes | 36 | Participants | |
| Percentage of Participants With Complete or Partial Response (CR/PR) and Steroid Dose Less Than 0.25 mg/kg Per Day | Prednisone | Yes | 20 | Participants |
1 to 10 of 72Source ClinicalTrials.gov
See all 72 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Sirolimus | 58 | |
| Overall Study | STARTED | Prednisone | 64 | |
| Overall Study | COMPLETED | Sirolimus | 53 | |
| Overall Study | COMPLETED | Prednisone | 63 | |
| Overall Study | NOT COMPLETED | Sirolimus | 5 | |
| Overall Study | NOT COMPLETED | Prednisone | 1 |
Source ClinicalTrials.gov
Adverse events22
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 | Small intestinal obstruction | Gastrointestinal disorders | Sirolimus | 1 | 58 | 1 |
| serious | Small intestinal obstruction | Gastrointestinal disorders | Prednisone | 0 | 64 | 0 |
| serious | Hepatic cirrhosis | Hepatobiliary disorders | Sirolimus | 1 | 58 | 1 |
| serious | Hepatic cirrhosis | Hepatobiliary disorders | Prednisone | 0 | 64 | 0 |
| serious | Appendicitis | Infections and infestations | Sirolimus | 1 | 58 | 1 |
| serious | Appendicitis | Infections and infestations | Prednisone | 0 | 64 | 0 |
| serious | Diverticulitis | Infections and infestations | Sirolimus | 1 | 58 | 1 |
| serious | Diverticulitis | Infections and infestations | Prednisone | 0 | 64 | 0 |
| serious | Pneumonia | Infections and infestations | Sirolimus | 1 | 58 | 1 |
| serious | Pneumonia | Infections and infestations | Prednisone | 0 | 64 | 0 |
1 to 10 of 22Source ClinicalTrials.gov
See all 22 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Sirolimus | 12 | 58 |
| Total, all-cause mortality | Prednisone | 17 | 64 |
| Total, other adverse events | Sirolimus | 0 | 58 |
| Total, other adverse events | Prednisone | 0 | 64 |
| Total, serious adverse events | Sirolimus | 8 | 58 |
| Total, serious adverse events | Prednisone | 3 | 64 |
Source ClinicalTrials.gov
Papers3
The papers the trial lists on ClinicalTrials.gov.
Grants2
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 | Source |
|---|---|---|---|---|---|---|
| U24CA076518 | A Data Resource for Blood and Marrow Transplants and Adoptive Cellular Therapy Research | SHAW, BRONWEN; MOSKOP, AMY M | MEDICAL COLLEGE OF WISCONSIN | NATIONAL CANCER INSTITUTE | 1998 to 2025 | NIH RePORTER |
| 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 | NIH RePORTER |
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Sirolimus vs Prednisone: deaths from any cause | Trial comparisons |
| Sirolimus vs Prednisone: 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.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02806947
GET https://conductscience.org/api/trials/v1/NCT02806947/results?page=2