The safety and efficacy of Alpha-1 Antitrypsin (AAT) for the prevention of graft-versus-host disease (GVHD) in patients receiving hematopoietic cell transplant

A Phase 2/3, Multicenter, randOmized, Double-blind, placebo-controlled, stUdy to evaLuate the safety and efficacy of Alpha-1 AntiTrypsin for the prEvention of graft-versus-host disease in patients receiving hematopoietic cell transplant (MODULAATE Study) - MODULAATE

Status

Not yet recruiting

Phases

Phase 2Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2018-000329-29-ES

Enrollment

310

Registered

2018-10-09

Start date

2019-01-28

Completion date

Unknown

Last updated

2019-02-28

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Graft versus host disease MedDRA version: 20.1 Level: PT Classification code 10018651 Term: Graft versus host disease System Organ Class: 10021428 - Immune system disorders

Interventions

Trade Name: Respreeza Product Name: Alpha-1 antitrypsin Product Code: Alpha-1 proteinase inhibitor Pharmaceutical Form: Powder and solvent for solution

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Male or female subjects, =12 years of age, undergoing HCT for hematological malignancies, including leukemia, lymphoma and multiple myeloma 2. Planned myeloablative conditioning regimen Are the trial subjects under 18? yes Number of subjects for this age range: 10 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 290 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 10

Exclusion criteria

Exclusion criteria: 1. Prior autologous or allogeneic HCT 2. T-cell depleted transplant or planned use of anti-T cell antibody therapy either ex vivo or in vivo 3. Planned umbilical cord blood (UCB) transplant

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate the efficacy of AAT at the selected dose for the prevention of acute GVHD following HCT.;Primary end point(s): Percent of subjects with acute graft-versus-host-disease-free survival;Timepoint(s) of evaluation of this end point: 180 days post-hematopoietic cell transplantation; Secondary Objective: 1. To evaluate the efficacy of AAT, including the prevention of post-hematopoietic cell transplant complications. 2. To evaluate the safety of AAT, based on incidence of systemic infections and related adverse events. 3. To evaluate the pharmacokinetics of AAT in HCT recipients.	—

Measure

Time frame

Main Objective: To evaluate the efficacy of AAT at the selected dose for the prevention of acute GVHD following HCT.;Primary end point(s): Percent of subjects with acute graft-versus-host-disease-free survival;Timepoint(s) of evaluation of this end point: 180 days post-hematopoietic cell transplantation; Secondary Objective: 1. To evaluate the efficacy of AAT, including the prevention of post-hematopoietic cell transplant complications. 2. To evaluate the safety of AAT, based on incidence of systemic infections and related adverse events. 3. To evaluate the pharmacokinetics of AAT in HCT recipients.

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Secondary

Measure	Time frame
Secondary end point(s): -Percent of subjects with Grade II-IV acute GVHD -Percent of subjects with Grade III-IV acute GVHD -Number of subjects with all-cause mortality -Percent of subjects with Grade II aGVHD -Percent of subjects with Grade III aGVHD -Percent of subjects with Grade IV aGVHD -Time to all-cause mortality -Time to non-relapse mortality -Percent of subjects with moderate-to-severe chronic GVHD -Percent of subjects with discontinuation of immune suppression -Time to neutrophil engraftmen -Time to GVHD relapse-free survival -Percent of subjects with relapse of primary malignancies -Percent of subjects with systemic infections -Percent of subjects with study drug related adverse events -Maximum concentration (Cmax) of AAT -Area under the concentration curve (AUC) for AAT -Clearance (CL) of AAT -Volume of distribution (V) for AAT ; Timepoint(s) of evaluation of this end point: -Within 100 and 180 days post-HCT -Within 100 and 180 days post-hematopoietic cell transplant (HCT) -Within 180 and 365 days post-HCT -Within 100 and 180 days post-HCT -Within 100 and 180 days post-HCT -Within 100 and 180 days post-HCT -Up to 365 days post-HCT -Up to 365 days post-HCT -Within 180 and 365 days post-HCT -Within 180 and 365 days post-HCT -Up to 365 days post-HCT -Up to 365 days post-HCT -Within 180 and 365 days post-HCT -At Days 60 and 180 post-HCT -Up to 365 days post-HCT -Before and up to 72 after infusion of AAT	—

Measure

Time frame

Secondary end point(s): -Percent of subjects with Grade II-IV acute GVHD -Percent of subjects with Grade III-IV acute GVHD -Number of subjects with all-cause mortality -Percent of subjects with Grade II aGVHD -Percent of subjects with Grade III aGVHD -Percent of subjects with Grade IV aGVHD -Time to all-cause mortality -Time to non-relapse mortality -Percent of subjects with moderate-to-severe chronic GVHD -Percent of subjects with discontinuation of immune suppression -Time to neutrophil engraftmen -Time to GVHD relapse-free survival -Percent of subjects with relapse of primary malignancies -Percent of subjects with systemic infections -Percent of subjects with study drug related adverse events -Maximum concentration (Cmax) of AAT -Area under the concentration curve (AUC) for AAT -Clearance (CL) of AAT -Volume of distribution (V) for AAT ; Timepoint(s) of evaluation of this end point: -Within 100 and 180 days post-HCT -Within 100 and 180 days post-hematopoietic cell transplant (HCT) -Within 180 and 365 days post-HCT -Within 100 and 180 days post-HCT -Within 100 and 180 days post-HCT -Within 100 and 180 days post-HCT -Up to 365 days post-HCT -Up to 365 days post-HCT -Within 180 and 365 days post-HCT -Within 180 and 365 days post-HCT -Up to 365 days post-HCT -Up to 365 days post-HCT -Within 180 and 365 days post-HCT -At Days 60 and 180 post-HCT -Up to 365 days post-HCT -Before and up to 72 after infusion of AAT

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Countries

Australia, Belgium, Canada, France, Germany, Hungary, Italy, Poland, Spain, Sweden, United Kingdom, United States

Contacts

Public ContactTrial Registration Coordinator

CSL Behring LLC

clinicaltrials@cslbehring.com6108784000

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026