Treatment of GVHD in Hematopoietic Stem Cell Transplant (HSCT) Recipients Using AAT Plus Corticosteroids (CS) Compared With Corticosteroids Alone

CSL Behring

Status and phase: Completed; Phase 3

Conditions: Graft Versus Host Disease (GVHD)

Treatments: Drug: Placebo; Biological: Alpha-1 antitrypsin (AAT)

Study type: Interventional

Funder types: NETWORK; Industry; NIH

Identifiers

NCT04167514

BMT CTN Protocol 1705 (Other Identifier)

CSL964_5001 / BMT CTN 1705

Details and patient eligibility

About

Study CSL964_5001 will investigate the efficacy of AAT with corticosteroids compared with corticosteroids alone as first line therapy for patients with high-risk acute GVHD

Enrollment: 136 patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Patients 12 years of age or older
• Initial presentation of acute GVHD after allogeneic hematopoietic cell transplantation for any indication
• Any graft or donor source or conditioning intensity
• Clinical diagnosis of acute GVHD requiring systemic therapy with corticosteroids

Exclusion criteria

• Prior exogenous AAT exposure for GVHD prophylaxis
• Relapsed, progressing, or persistent malignancy
• de novo chronic GVHD or overlap syndrome developing before or present at the time of enrollment
• Receiving other drugs for the treatment of GVHD
• Receiving systemic CS for any indication within 7 days before the onset of acute GVHD

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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136 participants in 2 patient groups, including a placebo group

AAT

Experimental group

Description:

Alpha-1 antitrypsin (AAT) is a lyophilized powder for intravenous administration

Treatment:

Biological: Alpha-1 antitrypsin (AAT)

Placebo

Placebo Comparator group

Description:

Albumin solution administered intravenously

Treatment:

Drug: Placebo