The Safety and Efficacy of Alpha-1 Antitrypsin (AAT) for the Prevention of Graft-Versus-host Disease (GVHD) in Patients Receiving Hematopoietic Cell Transplant (MODULAATE)

CSL Behring

Status and phase: Enrolling; Phase 3; Phase 2

Conditions: Acute-graft-versus-host Disease

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

Study type: Interventional

Funder types: Industry

Identifiers

NCT03805789

2018-000329-29 (EudraCT Number)

CSL964_2001

Details and patient eligibility

About

This study is a Phase 2/3 prospective, double-blind, randomized, multi-center, placebo-controlled study for prevention of acute GVHD (aGVHD) in subjects undergoing an allogeneic hematopoietic cell transplant (HCT).

Enrollment: 310 estimated patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Male or female subjects, ≥12 years of age (≥ 18 years of age for subjects at German sites only), undergoing HCT for hematological malignancies, including leukemia, lymphoma, multiple myeloma, myelodysplastic syndrome and myeloproliferative neoplasms
• Planned myeloablative conditioning regimen

Exclusion criteria

• Prior autologous or allogeneic HCT
• T-cell depleted transplant or planned use of anti-T cell antibody therapy either ex vivo or in vivo (ie, anti-thymocyte globulin [ATG], alemtuzumab) for GVHD prophylaxis
• Planned umbilical cord blood (UCB) transplant

Trial design

Primary purpose: Prevention

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: Double Blind

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310 participants in 5 patient groups, including a placebo group

AAT (low dose)

Experimental group

Description:

Open label. Alpha-1 antitrypsin (AAT) is a lyophilized product for intravenous administration

Treatment:

Biological: Alpha-1 antitrypsin (AAT)

AAT (medium dose)

Experimental group

Description:

Open label. AAT is a lyophilized product for intravenous administration

Treatment:

Biological: Alpha-1 antitrypsin (AAT)

AAT (high dose)

Experimental group

Description:

Open label. AAT is a lyophilized product for intravenous administration

Treatment:

Biological: Alpha-1 antitrypsin (AAT)

AAT (selected dose from open-label)

Experimental group

Description:

Double-blind. AAT is a lyophilized product for intravenous administration

Treatment:

Biological: Alpha-1 antitrypsin (AAT)

Placebo

Placebo Comparator group

Description:

Albumin solution administered intravenously

Treatment:

Biological: Placebo