Phase 2
N=20
Alpha 1 Anti-Trypsin (AAT) in Treating Patients With Acute Graft-Versus-Host Disease GVHD)
Graft-Versus-Host Disease (GVHD) Acute on Chronic
Bottom Line
View on ClinicalTrials.gov: NCT01523821 ↗Enrolled (actual)
20
Serious AEs
85.0%
Results posted
Oct 2018
Primary outcome: Primary: Number (Percentage) of Patients at Each Dosing Cohort Who Experience no Toxicity and in Whom Graft Versus Host Disease (GVHD) is Stable or Improved — 6; 2; 2 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Alpha 1-Proteinase Inhibitor, Human 1 MG [Glassia] (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Fred Hutchinson Cancer Center
- Primary completion
- Jan 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number (Percentage) of Patients at Each Dosing Cohort Who Experience no Toxicity and in Whom Graft Versus Host Disease (GVHD) is Stable or Improved |
6; 2; 2 | — |
| SECONDARY Number (Percentage) of Patients at Each Dosing Cohort Experiencing an Unexpected Serious Adverse Event (SAE) |
2; 0; 0 | — |
| SECONDARY Number (Percentage) of Patients at Each Dosing Cohort Who Experience One or More Suspected Serious Adverse Reactions (Infusion Related Reactions) |
0; 0; 0 | — |
| SECONDARY Number (Percentage) of Patients at Each Dosing Cohort Who Experience One or More Thrombotic or Thrombo-embolic Events |
0; 0; 0 | — |
| SECONDARY Number (Percentage) of Patients at Each Dosing Cohort With Occurrence of Infections |
7; 5; 5 | — |
| SECONDARY Number (Percentage) of Patients at Each Dosing Cohort With Progression of GVHD |
4; 5; 4 | — |
Summary
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.
Eligibility Criteria
Inclusion Criteria
- Patients transplanted from related or unrelated, human leukocyte antigen (HLA) matched or mismatched donors
- Patients transplanted with hematopoietic stem cells from any source
- Patients receiving calcineurin inhibitors as part of graft versus host disease (GVHD) prophylaxis
- Patients with acute GVHD grades II-IV developing despite GVHD prophylaxis
- Patients who have not shown a satisfactory response to methylprednisolone-equivalent doses at 2 mg/kg/day, based on adjusted body weight
- Signed and dated informed consent
Exclusion Criteria
- Patients who have received any systemic agents in addition to steroids for treatment of GVHD
- Patients unable to give informed consent
- Patients with manifestations of classic chronic GVHD
- Patients with evidence of recurrent malignancy
- Patients with acute/chronic GVHD overlap syndrome
- Patients whose GVHD developed after donor lymphocyte infusion (DLI)
- Patients with severe organ dysfunction, defined as
- On dialysis
- Requiring oxygen (O2) at more than 2 l/min
- Uncontrolled arrhythmia or heart failure
- Veno-occlusive disease (sinusoidal obstruction syndrome)
- Patients with uncontrolled infections
Data sourced from ClinicalTrials.gov (NCT01523821). 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.