Treatment of GVHD in Hematopoietic Stem Cell Transplant (HSCT) Recipients Using AAT Plus Corticos… (NCT04167514) | Clinical Trial Compass
CompletedPhase 3
Treatment of GVHD in Hematopoietic Stem Cell Transplant (HSCT) Recipients Using AAT Plus Corticosteroids (CS) Compared With Corticosteroids Alone
United States136 participantsStarted 2020-01-09
Plain-language 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
Who can participate
Age range
12 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1This trial compared alpha-1 antitrypsin (AAT) plus corticosteroids against corticosteroids alone for acute GVHD — since the trial is now completed, has the data been published yet, and what did it show about whether adding AAT actually improved response rates?
2Since this was a Phase 3 trial, it was designed to confirm effectiveness in a larger group — does that mean there's more safety and dosing information available here than there would be from an earlier-phase study, and how does that affect your confidence in AAT as an option for my situation?
3The trial measured 'Overall Response Rate' as its main goal — can you explain what that means in practical terms for someone with acute GVHD, and how that outcome compares to what we'd expect from standard corticosteroid treatment alone?
4Given that this trial has already completed enrollment, are there any follow-on studies or expanded access programs involving AAT for GVHD that might still be worth exploring, or is standard corticosteroid therapy the most realistic path for me right now?
5How does the severity or type of my acute GVHD compare to what this trial was studying, and based on the results, would my care team consider AAT alongside corticosteroids to be a relevant option worth discussing for my treatment plan?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Overall Response Rate (ORR) to Acute Graft-versus-Host Disease (GVHD) Treatment