Trial · NCT04167514
Treatment of GVHD in Hematopoietic Stem Cell Transplant (HSCT) Recipients Using AAT Plus Corticosteroids (CS) Compared With Corticosteroids Alone
NCT04167514, Phase 3, Completed, CSL Behring; results first posted 2025-08-29
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Treatment of GVHD in Hematopoietic Stem Cell Transplant (HSCT) Recipients Using AAT Plus Corticosteroids (CS) Compared With Corticosteroids Alone. Trials, record NCT04167514. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04167514
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Treatment of GVHD in Hematopoietic Stem Cell Transplant (HSCT) Recipients Using AAT Plus Corticosteroids (CS) Compared With Corticosteroids Alone
- NCT number
- NCT04167514
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 136 participants, actual
- Lead sponsor
- CSL Behring
- Conditions
- Graft Versus Host Disease (GVHD)
- Interventions
- Alpha-1 antitrypsin (AAT), Placebo
- First posted
- 2019-11-19
- Last updated
- 2026-07-28
- Results first posted
- 2025-08-29
Source ClinicalTrials.gov
Registered outcomes12
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Overall Response Rate (ORR) to Acute Graft-versus-Host Disease (GVHD) Treatment | At Day 28 | The overall response is defined as having a CR or PR at Day 28, along with: being alive, free of any next-line GVHD therapy, and free of escalation of prednisone-equivalent steroid dose to 2.5 milligrams per kilogram (mg/kg)/day or more. The percentage of participants with CR or PR is reported here. Wilson score confidence intervals (CIs) are reported here as a measure of dispersion. The CR was defined as a score of 0 for the GVHD staging in all evaluable organs. The PR was defined as an improvement in one or more organs involved with GVHD symptoms without progression in others. Acute GVHD was graded and assessed for response based on Harris (Mount Sinai Acute GVHD International Consortium \[MAGIC\]) criteria (stage 0, 1, 2, 3, 4) for skin, liver, upper gastrointestinal (GI) tract, and lower GI tract. Participants who had an escalation of prednisone-equivalent steroid dose to 2.5 mg/kg/day or higher were classified as non-responders (NR). |
| Secondary | Duration of Response (DOR) | Up to 12 months | The DOR was defined as time from the Day 28 response (CR or PR) until the first of the following events occurs: progression of acute GVHD, death, any next-line GVHD therapy, or escalation of prednisone-equivalent steroids to greater than or equal to (\>=) 2.5 mg/kg/day. Wald CIs are reported here as a measure of dispersion. |
| Secondary | Number of Participants With Non-relapse Mortality (NRM) Event | At 6 and 12 months | An event of NRM was death without prior evidence of relapse/progression of the primary disease, where relapse/progression was treated as a competing risk. Cumulative incidence of NRM at 6 and 12 months post-randomization is reported here for this outcome measure. |
| Secondary | Number of Overall and Progression-free Survival Events | At 6 and 12 months | An event for overall survival (OS) was death from any cause, while an event for progression-free survival (PFS) was death from any cause or relapse/progression of the primary disease. |
| Secondary | Number of Participants With GVHD-free Survival | At Day 56 | GVHD-free survival was defined as participants being alive, free of acute or chronic GVHD, and free of any next-line GVHD therapy or escalation of steroids to \>=2.5 mg/kg/day prednisone or equivalent for treatment of GVHD. |
| Secondary | Percentage of Participants With Response | At Day 7, 14, 21, 28, 56, and 86 | The percentage of participants with CR, PR (including subset with very good partial response \[VGPR\]), and treatment failure (TF). The designation of TF consisted of participants with NR, mixed response (MR) or progression. The initiation of additional systemic (next-line) GVHD therapies, escalation of prednisone equivalent steroid dose to \>= 2.5 mg/kg/day, or death from any cause prior to the assessment timepoint was also considered a TF. Simultaneous Goodman CIs are provided for category proportions for each arm at each assessment time. |
| Secondary | Percentage of Participants With Response Allowing for Approved Next-line Therapy | At Day 7, 14, 21, 28, 56, and 86 | The percentage of participants with CR, PR (including subset with VGPR), and TF allowing for treatment with next-line therapy. The designation of TF consisted of participants with NR, MR, or progression. The escalation of prednisone-equivalent steroid dose to \>= 2.5 mg/kg/day or death from any cause prior to the assessment timepoint was also considered a TF. Simultaneous Goodman CIs are provided for category proportions for each arm at each assessment time. |
| Secondary | Incidence of Grade 2 to 3 Systemic Infections | Up to 90 days (including 30 days after last dose of study drug) | The cumulative incidence of Grade 2 to 3 systemic infections. Grade 2 to 3 systemic infections were defined according to the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) Manual of Procedures. |
| Secondary | Percentage of Participants With Grade 3 to 5 Treatment-emergent Adverse Events (TEAEs) | Up to 30 days after the last dose of study drug | The incidence of Grade 3 to 5 TEAEs (per Common Terminology Criteria for Adverse Events \[CTCAE\] Version 5.0). Wilson score CIs are reported here as a measure of dispersion. |
| Secondary | Cumulative Incidence of Chronic GVHD | At 6 and 12 months | Chronic GVHD was defined per National Institutes of Health (NIH) Consensus Criteria. Diagnosis of chronic GVHD of any severity (mild, moderate, or severe) was considered an event for this outcome measure, where death before cGVHD was treated as a competing risk. |
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 | Overall Response Rate (ORR) to Acute Graft-versus-Host Disease (GVHD) Treatment | At Day 28 | Analysis was performed on the ITT population. The ITT population consisted of all randomized participants, classified according to their randomized treatment assignments. All randomized participants were included, regardless of whether the assigned study treatment was truly administered. | percentage of participants | NUMBER |
| SECONDARY | Duration of Response (DOR) | Up to 12 months | Analysis was performed on the ITT population participants with a response as defined in the measure description of the primary outcome measure. The ITT population consisted of all randomized participants, classified according to their randomized treatment assignments. All randomized participants were included, regardless of whether the assigned study treatment was truly administered. | days | MEDIAN |
| SECONDARY | Number of Participants With Non-relapse Mortality (NRM) Event | At 6 and 12 months | Analysis was performed on the ITT population. The ITT population consisted of all randomized participants, classified according to their randomized treatment assignments. All randomized participants were included, regardless of whether the assigned study treatment was truly administered. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Overall and Progression-free Survival Events | At 6 and 12 months | Analysis was performed on the ITT population. The ITT population consisted of all randomized participants, classified according to their randomized treatment assignments. All randomized participants were included, regardless of whether the assigned study treatment was truly administered. | events | NUMBER |
| SECONDARY | Number of Participants With GVHD-free Survival | At Day 56 | Analysis was performed on the ITT population. The ITT population consisted of all randomized participants, classified according to their randomized treatment assignments. All randomized participants were included, regardless of whether the assigned study treatment was truly administered. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Percentage of Participants With Response | At Day 7, 14, 21, 28, 56, and 86 | Analysis was performed on the ITT population. The ITT population consisted of all randomized participants, classified according to their randomized treatment assignments. All randomized participants were included, regardless of whether the assigned study treatment was truly administered. Here, the 'number analyzed' for Day 86 includes responses for only those participants who remained on maintenance treatment after Day 28. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Response Allowing for Approved Next-line Therapy | At Day 7, 14, 21, 28, 56, and 86 | Analysis was performed on the ITT population. The ITT population consisted of all randomized participants, classified according to their randomized treatment assignments. All randomized participants were included, regardless of whether the assigned study treatment was truly administered. Here, the 'number analyzed' for Day 86 includes responses for only those participants who remained on maintenance treatment after Day 28. | percentage of participants | NUMBER |
| SECONDARY | Incidence of Grade 2 to 3 Systemic Infections | Up to 90 days (including 30 days after last dose of study drug) | Analysis was performed on the safety population. The safety population included all randomized participants who received at least one dose of study treatment. Participants were classified according to the treatment received. | events | NUMBER |
| SECONDARY | Percentage of Participants With Grade 3 to 5 Treatment-emergent Adverse Events (TEAEs) | Up to 30 days after the last dose of study drug | Analysis was performed on the safety population. The safety population included all randomized participants who received at least one dose of study treatment. Participants were classified according to the treatment received. | percentage of participants | NUMBER |
| SECONDARY | Cumulative Incidence of Chronic GVHD | At 6 and 12 months | Analysis was performed on the ITT population. The ITT population consisted of all randomized participants, classified according to their randomized treatment assignments. All randomized participants were included, regardless of whether the assigned study treatment was truly administered. | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 outcomesResults by group104
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) to Acute Graft-versus-Host Disease (GVHD) Treatment | Alpha-1 Antitrypsin (AAT) | 60.0 | 95% Confidence Interval 47.86 to 71.03 | percentage of participants | |
| Overall Response Rate (ORR) to Acute Graft-versus-Host Disease (GVHD) Treatment | Placebo | 45.1 | 95% Confidence Interval 34.05 to 56.60 | percentage of participants | |
| Duration of Response (DOR) | Alpha-1 Antitrypsin (AAT) | 82 | 95% Confidence Interval 22 to 176 | days | |
| Duration of Response (DOR) | Placebo | 154 | 95% Confidence Interval 57 to NA | days | |
| Number of Participants With Non-relapse Mortality (NRM) Event | Alpha-1 Antitrypsin (AAT) | At 6 months | 12 | Participants | |
| Number of Participants With Non-relapse Mortality (NRM) Event | Placebo | At 6 months | 14 | Participants | |
| Number of Participants With Non-relapse Mortality (NRM) Event | Alpha-1 Antitrypsin (AAT) | At 12 months | 13 | Participants | |
| Number of Participants With Non-relapse Mortality (NRM) Event | Placebo | At 12 months | 14 | Participants | |
| Number of Overall and Progression-free Survival Events | Alpha-1 Antitrypsin (AAT) | OS: At 6 months | 16 | events | |
| Number of Overall and Progression-free Survival Events | Placebo | OS: At 6 months | 18 | events |
1 to 10 of 104Source ClinicalTrials.gov
See all 104 results by groupParticipant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Alpha-1 Antitrypsin (AAT) | 65 | |
| Overall Study | STARTED | Placebo | 71 | |
| Overall Study | Treated | Alpha-1 Antitrypsin (AAT) | 63 | |
| Overall Study | Treated | Placebo | 69 | |
| Overall Study | COMPLETED | Alpha-1 Antitrypsin (AAT) | 38 | |
| Overall Study | COMPLETED | Placebo | 41 | |
| Overall Study | NOT COMPLETED | Alpha-1 Antitrypsin (AAT) | 27 | |
| Overall Study | NOT COMPLETED | Placebo | 30 |
Source ClinicalTrials.gov
Adverse events164
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 | Hyperglycaemia | Metabolism and nutrition disorders | Alpha-1 Antitrypsin (AAT) | 13 | 63 | 15 |
| other | Hyperglycaemia | Metabolism and nutrition disorders | Placebo | 11 | 69 | 15 |
| other | Hypomagnesaemia | Metabolism and nutrition disorders | Alpha-1 Antitrypsin (AAT) | 10 | 63 | 15 |
| other | Hypomagnesaemia | Metabolism and nutrition disorders | Placebo | 10 | 69 | 23 |
| other | Hypocalcaemia | Metabolism and nutrition disorders | Alpha-1 Antitrypsin (AAT) | 7 | 63 | 16 |
| other | Hypocalcaemia | Metabolism and nutrition disorders | Placebo | 12 | 69 | 26 |
| other | Hyperkalaemia | Metabolism and nutrition disorders | Alpha-1 Antitrypsin (AAT) | 6 | 63 | 11 |
| other | Hyperkalaemia | Metabolism and nutrition disorders | Placebo | 9 | 69 | 13 |
| other | Hypoalbuminaemia | Metabolism and nutrition disorders | Alpha-1 Antitrypsin (AAT) | 9 | 63 | 27 |
| other | Hypoalbuminaemia | Metabolism and nutrition disorders | Placebo | 6 | 69 | 12 |
1 to 10 of 164Source ClinicalTrials.gov
See all 164 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Alpha-1 Antitrypsin (AAT) | 21 | 65 |
| Total, all-cause mortality | Placebo | 23 | 71 |
| Total, other adverse events | Alpha-1 Antitrypsin (AAT) | 37 | 63 |
| Total, other adverse events | Placebo | 42 | 69 |
| Total, serious adverse events | Alpha-1 Antitrypsin (AAT) | 11 | 63 |
| Total, serious adverse events | Placebo | 23 | 69 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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 comparisons3
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Alpha-1 Antitrypsin (AAT) vs Placebo: Number of Participants With GVHD-free Survival | Trial comparisons |
| Alpha-1 Antitrypsin (AAT) vs Placebo: deaths from any cause | Trial comparisons |
| Alpha-1 Antitrypsin (AAT) vs Placebo: 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/NCT04167514
GET https://conductscience.org/api/trials/v1/NCT04167514/results?page=2