Phase 3
Completed N=136
Treatment of GVHD in Hematopoietic Stem Cell Transplant (HSCT) Recipients Using AAT Plus Corticosteroids (CS) Compared With Corticosteroids Alone
Graft Versus Host Disease (GVHD)
Source: ClinicalTrials.gov NCT04167514 ↗
Enrolled (actual)
136
Serious AEs
25.8%
Results posted
Aug 2025
Primary outcomePrimary: Overall Response Rate (ORR) to Acute Graft-versus-Host Disease (GVHD) Treatment — 60.0; 45.1 percentage of participants — p=0.034
◆ Published Evidence
No publication linked
No peer-reviewed publication reporting this trial's results has been linked yet. This can indicate results are unpublished — a known publication-bias signal. We re-check periodically.
Summary
Study CSL964\_5001 will investigate the efficacy of AAT with corticosteroids compared with corticosteroids alone as first line therapy for patients with high-risk acute GVHD
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Overall Response Rate (ORR) to Acute Graft-versus-Host Disease (GVHD) Treatment |
60.0; 45.1 | 0.034 sig |
| SECONDARY Duration of Response (DOR) |
82; 154 | — |
| SECONDARY Number of Participants With Non-relapse Mortality (NRM) Event |
12; 14; 13; 14 | — |
| SECONDARY Number of Overall and Progression-free Survival Events |
16; 18; 20; 23; 25; 28 | — |
| SECONDARY Number of Participants With GVHD-free Survival |
29; 25 | — |
| SECONDARY Percentage of Participants With Response |
29.2; 33.8; 33.8; 25.4; 36.9; 40.8 | — |
| SECONDARY Percentage of Participants With Response Allowing for Approved Next-line Therapy |
29.2; 33.8; 36.9; 31.0; 33.8; 35.2 | — |
| SECONDARY Incidence of Grade 2 to 3 Systemic Infections |
20; 22 | — |
| SECONDARY Percentage of Participants With Grade 3 to 5 Treatment-emergent Adverse Events (TEAEs) |
46.0; 65.2 | — |
| SECONDARY Cumulative Incidence of Chronic GVHD |
13; 9; 20; 14 | — |
| SECONDARY Cumulative Incidence of Disease Relapse/ Progression of Primary Disease |
14; 14; 15; 15 | — |
Eligibility Criteria
Inclusion Criteria
- Patients 12 years of age or older
- Initial presentation of acute GVHD after allogeneic hematopoietic cell transplantation for any indication
- Any graft or donor source or conditioning intensity
- Clinical diagnosis of acute GVHD requiring systemic therapy with corticosteroids
Exclusion Criteria
- Prior exogenous AAT exposure for GVHD prophylaxis
- Relapsed, progressing, or persistent malignancy
- de novo chronic GVHD or overlap syndrome developing before or present at the time of enrollment
- Receiving other drugs for the treatment of GVHD
- Receiving systemic CS for any indication within 7 days before the onset of acute GVHD
Data sourced from ClinicalTrials.gov (NCT04167514). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.