Alpha 1 Anti-Trypsin (AAT) in Treating Patients With Acute Graft-Versus-Host Disease GVHD)

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

20 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-10-11

Study Completion Date

2017-01-15

Brief Summary

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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.

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Detailed Description

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PRIMARY OBJECTIVES:

I. Determine the safety and tolerability of AAT in patients with steroid non-responsive acute GVHD.

II. Characterize pharmacodynamic effects of AAT on pro-inflammatory cytokines, heparan sulfate, and the spectrum of peripheral blood T cells.

III. Determine clinical responses of GVHD to AAT in patients with steroid non-responsive acute GVHD.

OUTLINE: This is a phase I/II dose-escalation study of AAT.

Patients will receive AAT intravenously (IV) on study days 1, 3, 5, and 7. Patients who experience no toxicity and in whom GVHD is stable or improved after the day 7 dose can continue therapy with AAT on days 9, 11, 13 and 15 for a total of 8 doses.

Conditions

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Graft-Versus-Host Disease (GVHD) Acute on Chronic

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

The study will have three escalating alpha 1 anti-trypsin (AAT) dose cohorts with six patients in each cohort (Phase I). Based on response and toxicity, an additional six patients will be enrolled in the optimal dose (Phase II).

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cohort 1 (30 mg/kg)

Alpha 1 anti-trypsin (AAT) will be administered intravenously at a dose of 90 mg/kg (loading dose) on day 1 followed by 30mg/kg (maintenance dose) every other day (QOD) on days 3, 5, 7, 9, 11, 13 \& 15.

Group Type OTHER

Alpha 1-Proteinase Inhibitor, Human 1 MG [Glassia]

Intervention Type DRUG

Alpha 1-Proteinase Inhibitor, Human 1 MG \[Glassia\] at various levels over different days

Cohort 2 (60 mg/kg)

AAT will be administered intravenously at a dose of 90 mg/kg (loading dose) on day 1 followed by 60 mg/kg (maintenance dose) QOD on days 3, 5, 7, 9, 11, 13 \& 15.

Group Type OTHER

Alpha 1-Proteinase Inhibitor, Human 1 MG [Glassia]

Intervention Type DRUG

Alpha 1-Proteinase Inhibitor, Human 1 MG \[Glassia\] at various levels over different days

Cohort 3 (90 mg/kg)

AAT will be administered intravenously at a dose of 90 mg/kg on days 1, 3, 5, 7, 9, 11, 13 \& 15.

Group Type OTHER

Alpha 1-Proteinase Inhibitor, Human 1 MG [Glassia]

Intervention Type DRUG

Alpha 1-Proteinase Inhibitor, Human 1 MG \[Glassia\] at various levels over different days

Interventions

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Alpha 1-Proteinase Inhibitor, Human 1 MG [Glassia]

Alpha 1-Proteinase Inhibitor, Human 1 MG \[Glassia\] at various levels over different days

Intervention Type DRUG

Eligibility Criteria

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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

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No