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)

April 17, 2026 updated by: CSL Behring

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)

This study is a phase 2 / 3 prospective, double-blind, randomized, multicenter, placebo-controlled study for prevention of acute GVHD (aGVHD) in participants undergoing an unrelated (matched or single allele mismatched) or matched related allogeneic hematopoietic cell transplantation (HCT).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
222

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Australia
    • Queenland
      • Herston, Queenland, Australia, 4029
        • Royal Brisbane and Women's Hospital
  • Germany
      • Cologne, Germany, 50937
        • Uniklinik Koln
  • Italy
      • Calabria, Italy, 89133
        • Grande Ospedale Metropolitano Bianchi-Melacrino-Morelli
    • Catania
      • Calabria, Catania, Italy, 95123
        • University Hospital Catania
  • Japan
      • Anjo-shi, Japan, 4668602
        • Anjo Kosei Hospital
      • Bunkyō City, Japan, 1138677
        • Tokyo Metropolitan Komagome Hospital
      • Hiroshima, Japan, 7348551
        • Hiroshima University Kasumi Campus
      • Nagoya, Japan, 4538511
        • Aichi Medical Center Nagoya Daiichi Hospital
      • Nagoya, Japan, 4668550
        • Nagoya University Hospital
      • Okayama, Japan, 71008558
        • Okayama University Hospital
      • Osaka, Japan, 5418567
        • Osaka International Cancer Institute
      • Osaka, Japan, 5458586
        • Osaka Metropolitan University Hospital
      • Sapporo, Japan, 0608648
        • Hokkaido University Hospital
  • South Korea
      • Busan, South Korea, 49241
        • Pusan National University Hospital
      • Busan, South Korea, 48108
        • Inje University Haeundae Paik Hospital
      • Incheon, South Korea, 21565
        • Gachon University Gil Medical Center
      • Seoul, South Korea, 03080
        • Seoul National University Hospital
  • Spain
      • Barcelona, Spain, 08035
        • Hospital Universitario Valle de Hebron
      • Barcelona, Spain, 39008
        • Marqués de Valdecilla University Hospital
      • Salamanca, Spain, 37007
        • Salamanca University Hospital
  • Turkey (Türkiye)
      • Ankara, Turkey (Türkiye), 06200
        • Ankara Abdurrahman Yurtaslan
      • Battalgazi, Turkey (Türkiye), 44280
        • Turgut Ozal Medicine Center
  • United States
    • Arizona
      • Scottsdale, Arizona, United States, 85258
        • HonorHealth Scottsdale Shea Medical Center
    • Florida
      • St. Petersburg, Florida, United States, 33701
        • Johns Hopkins Hospital
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Emory University
    • Kansas
      • Westwood, Kansas, United States, 66205
        • University of Kansas Cancer Center
    • Massachusetts
      • Boston, Massachusetts, United States, 02215
        • Dana Farber Cancer Institute
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • University of Michigan Medical Center
    • North Carolina
      • Durham, North Carolina, United States, 27705
        • Duke University Medical Center
    • Ohio
      • Cleveland, Ohio, United States, 44106
        • University Hospital Cleveland Medical Center
    • Texas
      • San Antonio, Texas, United States, 77030
        • The University of Texas-MD Anderson Cancer Center
    • Utah
      • Salt Lake City, Utah, United States, 84113
        • University of Utah Primary Children's Hospital
    • Virginia
      • Charlottesville, Virginia, United States, 22903
        • University of Virginia Health System
    • Washington
      • Seattle, Washington, United States, 98109
        • Fred Hutchinson Cancer Research Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

8 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • • Male or female participants, >=12 years of age (>= 18 years of age for participants at German sites only), undergoing HCT for hematological malignancies, including leukemia, lymphoma, multiple myeloma, myelodysplastic syndrome, and myeloproliferative neoplasms.
  • • Planned myeloablative conditioning regimen.
  • • Participants must have a related or unrelated donor as follows:
  • - Related donor must be a 6 / 6 match for human leukocyte antigen (HLA)-A, -B, at intermediate (or higher) resolution, and -DR beta 1 (DRB1) at high resolution using deoxyribonucleic acid (DNA)-based typing.
  • - Unrelated donor must be 7 / 8 or 8 / 8 match for HLA-A, -B, and -C at intermediate (or higher) resolution, and -DRB1 at high resolution using DNA-based typing.

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 transplant.
  • • Planned use of cyclophosphamide after HCT for GVHD prophylaxis.
  • • Planned haploidentical donor.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Prevention
  • Allocation: Randomized
  • Interventional Model: Sequential Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Placebo Comparator: Placebo
Albumin solution administered intravenously
Biological: Placebo
Albumin solution administered intravenously
Experimental: AAT (low dose)
Open label. AAT is a lyophilized product for intravenous (IV) administration
Biological: AAT
AAT is a lyophilized product for IV administration.
Other Names:
  • Alpha-1 proteinase inhibitor
  • Respreeza
Experimental: AAT (medium dose)
Open label. AAT is a lyophilized product for IV administration
Biological: AAT
AAT is a lyophilized product for IV administration.
Other Names:
  • Alpha-1 proteinase inhibitor
  • Respreeza
Experimental: AAT (high dose)
Open label. AAT is a lyophilized product for IV administration
Biological: AAT
AAT is a lyophilized product for IV administration.
Other Names:
  • Alpha-1 proteinase inhibitor
  • Respreeza
Experimental: AAT (selected dose from open-label)
Double-blind. AAT is a lyophilized product for IV administration
Biological: AAT
AAT is a lyophilized product for IV administration.
Other Names:
  • Alpha-1 proteinase inhibitor
  • Respreeza

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
The time to Grade II-IV aGVHD or death
Time Frame: Through 180 days after HCT
Acute GVHD will be assessed using the Harris scoring system.
Through 180 days after HCT

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Number of deaths (relapse and nonrelapse-related)
Time Frame: Within 180, 365, and 730 days after HCT
Death by any cause
Within 180, 365, and 730 days after HCT
Maximum concentration (Cmax) of AAT
Time Frame: Before and up to 72 after infusion of AAT
Before and up to 72 after infusion of AAT
Area under the concentration curve (AUC) for AAT
Time Frame: Before and up to 72 after infusion of AAT
Before and up to 72 after infusion of AAT
Clearance (CL) of AAT
Time Frame: Before and up to 72 after infusion of AAT
Before and up to 72 after infusion of AAT
Volume of distribution (V) for AAT
Time Frame: Before and up to 72 after infusion of AAT
Before and up to 72 after infusion of AAT
Ctrough of AAT
Time Frame: Before and up to 72 after infusion of AAT
Before and up to 72 after infusion of AAT
Proportion of participants with lower gastrointestinal (GI) aGVHD or Grade III-IV aGVHD in any organ
Time Frame: Through 180 days after HCT
Through 180 days after HCT
Proportion of participants with severe infections defined by National Cancer Institute-Common Terminology Criteria for Adverse Events (NCI-CTCAE) greater than or equal to (>=) Grade 3
Time Frame: Through Day 60 after HCT
Through Day 60 after HCT
Proportion of participants with Grade II-IV aGVHD or death
Time Frame: Through 100 days and 180 days after HCT
Through 100 days and 180 days after HCT
Proportion of participants with lower GI aGVHD
Time Frame: Through Days 60, 100 and 180 after HCT
Through Days 60, 100 and 180 after HCT
Proportion of participants with severe infections defined by NCI-CTCAE >= Grade 3
Time Frame: Through 100 and 180 days after HCT
Through 100 and 180 days after HCT
Proportion of participants with Grade III-IV aGVHD or death
Time Frame: Through Days 60, 100, and 180 days after HCT
Through Days 60, 100, and 180 days after HCT
Proportion of participants with moderate to severe chronic GVHD
Time Frame: Within 180, 365, 545, and 730 days after HCT
Moderate to severe chronic GVHD graded according to National Institutes of Health (NIH) scale.
Within 180, 365, 545, and 730 days after HCT
Proportion of participants who have discontinued immune suppression therapies including standard of care GVHD prophylaxis and steroid treatment
Time Frame: Within 180 and 365 days after HCT
Within 180 and 365 days after HCT
Time to GVHD relapse-free survival
Time Frame: Within 365 and 730 days after HCT
GVHD free, relapse free, survival defined as time to any of the following events: 1) Grade III-IV acute GVHD, 2) moderate-severe chronic GVHD, 3) primary malignancy relapse or 4) death.
Within 365 and 730 days after HCT
Proportion of participants with relapse of primary malignancies
Time Frame: Through 180, 365, and 730 days after HCT
Through 180, 365, and 730 days after HCT
Proportion of participants with Grade II-IV aGVHD with an overall (complete + partial) response, complete response and partial response
Time Frame: Approximately 4 weeks after the initiation of systemic steroids during 8-week Treatment Period
Approximately 4 weeks after the initiation of systemic steroids during 8-week Treatment Period
Percent of participants with study drug related adverse events
Time Frame: Up to 365 days after HCT
Up to 365 days after HCT
Time to neutrophil engraftment
Time Frame: Through 365 days after HCT
Time to the first of 3 consecutive days of absolute neutrophil counts ? 500/µL.
Through 365 days after HCT

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
CSL Behring

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Study Physician, CSL Behring

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
March 27, 2019

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
March 19, 2026

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
March 19, 2026

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
January 14, 2019

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 14, 2019

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
January 16, 2019

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
April 22, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 17, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • CSL964_2001
  • 2018-000329-29 (EudraCT Number)
  • 2024-511164-92-00 (Ctis)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

CSL will consider on a case-by-case basis requests to share Individual Patient Data (IPD) with external bona-fide, qualified scientific and medical researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com.

IPD Sharing Time Frame

Requests for IPD will generally be considered once review by major regulatory authorities (ie FDA, EMA) is complete and the primary publication is available.

IPD Sharing Access Criteria

Proposed research should seek to answer a previously unanswered important medical or scientific question.

Applicable country specific privacy and other laws and regulations will be considered and may prevent sharing of IPD.

If the request is approved and the researcher has executed an appropriate data sharing agreement, IPD that has been appropriately anonymized will be available.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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