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A Pilot Study of Alpha-1-Antitrypsin (AAT) in Steroid Refractory Acute Graft vs Host Disease


Phase 2
18 Years
N/A
Not Enrolling
Both
Graft vs Host Disease

Thank you

Trial Information

A Pilot Study of Alpha-1-Antitrypsin (AAT) in Steroid Refractory Acute Graft vs Host Disease


Inclusion Criteria:



- Age >18 years

- Patients must have clinical evidence* of steroid-refractory acute Graft vs Host
Disease (any organ) defined as one of the following:

- No change or progression in the stage of skin GvHD after at least 1 week of
2mg/kg/day methylprednisolone (or po equivalent)

- lack of response of visceral (liver, GI) GvHD despite treatment with 2mg/kg/day
methylprednisolone for at least 72h.

- progression of visceral GvHD despite treatment with 2mg/kg/day
methylprednisolone for at least 48h

- visceral GvHD progressing to stage 4 after 24h of 2mg/kg/d methylprednisolone

- Patients with protracted acute GvHD who have not responded to at least
0.5mg/kg/d of prednisone are considered eligible.

- Ability to understand and the willingness to sign a written informed consent
document.

- * As GvHD is a clinical diagnosis, and patients will have already been initiated on
steroid therapy at the discretion of the attending physician, tissue confirmation of
refractory GvHD by biopsy is not required for entry to this study. It is anticipated
that most, but not all, patients will have undergone tissue confirmation of the
initial diagnosis of GvHD; however lack of tissue confirmation for this clinical
syndrome is not exclusionary.

Exclusion Criteria:

- As patients with steroid refractory acute GvHD are quite ill with multiple abnormal
labs and organ dysfunction, there are no explicit laboratory values or degree of
organ dysfunction that specifically preclude enrollment on this study. Baseline lab
studies will be obtained and followed throughout this trial as the standard of care
for patients with GvHD.

- Pregnancy or Nursing Mother

- Patients may not be receiving any other investigational agents at time of study entry

- Patients with known antibodies to IgA

Type of Study:

Interventional

Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Response to Treatment

Outcome Description:

To estimate the proportion of patients with steroid refractory acute Graft vs Host Disease) GvHD who respond (PR + CR) to Alpha-1 Antitrypsin (AAT) at a dose of 60mg/kg twice weekly for 8 doses

Outcome Time Frame:

4 weeks

Safety Issue:

Yes

Principal Investigator

Pavan Reddy, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

University of Michigan Cancer Center

Authority:

United States: Food and Drug Administration

Study ID:

umcc 2012.043

NCT ID:

NCT01700036

Start Date:

July 2013

Completion Date:

June 2018

Related Keywords:

  • Graft Vs Host Disease
  • Graft vs Host Disease

Name

Location

University of Michigan Cancer CenterAnn Arbor, Michigan  48109