NCT03459040

A Proof of Concept Pilot Trial of Alpha-1-Antitrypsin for Pre-Emption Of Steroid-Refractory Acute GVHD

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 30 (actual)
• Sponsor: John Levine · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

Bone marrow transplant (BMT) patients can develop graft-versus-host disease (GVHD), a serious and potentially fatal complication. The researchers have developed a blood test to identify patients most at risk for developing severe GVHD. Patients who consent to this study will have their blood tested up to two times after BMT to determine if they are at high risk for severe GVHD. The tests will be performed one week and two weeks after BMT. Patients who are high risk will be treated with a drug called alpha-1-antitrypsin (AAT) to see if it prevents the development of severe GVHD. Patients will receive 16 doses of AAT through a catheter placed into a blood vessel over eight weeks. AAT will be given either in the hospital or the outpatient clinic two times per week. Patients will be followed for the development of severe GVHD for up to four months from the BMT and will continue to be followed at routine clinic visits for up to one year after BMT.

Conditions

• Graft-versus-host-disease
• GVHD

Interventions

Type	Name	Description
DRUG	Alpha 1-Antitrypsin	AAT will be given either in the hospital or the outpatient clinic two times per week.

Timeline

• Start date: 2018-08-17
• Primary completion: 2020-08-21
• Completion: 2020-08-21
• First posted: 2018-03-08
• Last updated: 2021-07-12
• Results posted: 2021-05-24

Locations

5 sites across 1 country: United States

Regulatory

• FDA-regulated drug study