Alpha 1 Anti-Trypsin (AAT) in Treating Patients With Acute Graft-Versus-Host Disease GVHD)
CompletedPhase 1, PHASE2Results postedLast updated 30 October 2018
What this trial tests
Phase 1, PHASE2 trial testing Alpha 1-Proteinase Inhibitor, Human 1 MG [Glassia] in Graft-Versus-Host Disease (GVHD) Acute on Chronic in 20 participants. Completed in 15 January 2017.
18 and older, any sex, with Graft-Versus-Host Disease (GVHD) Acute on Chronic. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Number (Percentage) of Patients at Each Dosing Cohort Who Experience no Toxicity and in Whom Graft Versus Host Disease (GVHD) is Stable or ImprovedPrimary· Adverse events were reported through 15 days after the last dose of AAT. GVHD response assessed at study day 28.
Toxicity and adverse events were assessed using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v4.0. All adverse events were reported regardless of attribution to alpha 1 anti-trypsin (AAT). GVHD response was defined per standard criteria for improvement, no change or progression of signs/symptoms in skin rash (% body surface area), GI (nausea, vomiting, anorexia, diarrhea, GI bleeding, abdominal cramping) and hepatic function (serum bilirubin levels). For this outcome measure, the requirement for additional GVHD treatment beyond AAT was not included
Group
Value
95% CI
Cohort 1 (30 mg/kg)
6
Cohort 2 (60 mg/kg)
2
Cohort 3 (90 mg/kg)
2
Number (Percentage) of Patients at Each Dosing Cohort Experiencing an Unexpected Serious Adverse Event (SAE)Secondary· SAEs were reported through 30 days after the last dose of alpha 1 anti-trypsin (AAT).
Serious adverse events included death, a life-threatening adverse drug experience, inpatient hospitalization or prolongation of existing hospitalization, a persistent or significant disability/ incapacity, or congenital anomaly/birth defect. Significant events that do not meet these criteria may be considered serious if they jeopardize the patient and require a medical intervention to prevent one of the outcomes above. An "unexpected" adverse event is defined as an event that is not identified in nature, severity or frequency in the current investigator brochure/package insert/product informat
Group
Value
95% CI
Cohort 1 (30 mg/kg)
2
Cohort 2 (60 mg/kg)
0
Cohort 3 (90 mg/kg)
0
Number (Percentage) of Patients at Each Dosing Cohort Who Experience One or More Suspected Serious Adverse Reactions (Infusion Related Reactions)Secondary· Within 48 hours after each infusion
Serious adverse reactions were assessed by the NCI CTCAE v4.0.
Group
Value
95% CI
Cohort 1 (30 mg/kg)
0
Cohort 2 (60 mg/kg)
0
Cohort 3 (90 mg/kg)
0
Number (Percentage) of Patients at Each Dosing Cohort Who Experience One or More Thrombotic or Thrombo-embolic EventsSecondary· Events were reported through 15 days after the last dose of AAT.
Events were assessed using the NCI CTCAE v4.0.
Group
Value
95% CI
Cohort 1 (30 mg/kg)
0
Cohort 2 (60 mg/kg)
0
Cohort 3 (90 mg/kg)
0
Number (Percentage) of Patients at Each Dosing Cohort With Occurrence of InfectionsSecondary· Infections were reported through 15 days after the last dose of AAT.
Infections were assessed using NCI CTCAE v4.0.
Group
Value
95% CI
Cohort 1 (30 mg/kg)
7
Cohort 2 (60 mg/kg)
5
Cohort 3 (90 mg/kg)
5
Number (Percentage) of Patients at Each Dosing Cohort With Progression of GVHDSecondary· GVHD responses were assessed on day 28 after starting AAT therapy or at time of death if patient died before study day 28.
GVHD responses were assessed using criteria established by the Center for International Blood and Marrow Transplant Research and criteria from the Acute GVHD Activity Index. Patients who required additional systemic GVHD treatment beyond AAT before study day 28 were defined as having progressive GVHD.
Group
Value
95% CI
Cohort 1 (30 mg/kg)
4
Cohort 2 (60 mg/kg)
5
Cohort 3 (90 mg/kg)
4
Adverse events — posted to ClinicalTrials.gov
Time frame: Adverse events were reported through 15 days after the last dose of AAT. Serious adverse events were reported if they occurred within 30 days after the last dose of AAT..
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
This phase I/II trial evaluates the efficacy and adverse effects of alpha 1 anti-trypsin (AAT) for the treatment of acute graft-versus-host disease (GVHD) after hematopoietic stem cell transplantation.
Publications & conference data
5 peer-reviewed publications reference this trial (live from Europe PMC):
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Fred Hutchinson Cancer Center
Last refreshed: 30 October 2018
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT01523821.