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Using Alpha-1 Antitrypsin to Prevent Graft-versus-host Disease in Transplant Patients

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)

Phase2; Phase3 Interventional CSL Behring · NCT03805789

This study is testing if a medication called Alpha-1 Antitrypsin can help prevent a serious condition called graft-versus-host disease in patients receiving stem cell transplants for blood cancers.

Quick facts

PhasePhase2; Phase3
Study typeInterventional
Enrollment310 (estimated)
Ages12 Years and up
SexAll
SponsorCSL Behring Industry-sponsored
Drugs / interventionsalemtuzumab, cyclophosphamide
Locations34 sites (Scottsdale, Arizona and 33 other locations)
Trial IDNCT03805789 on ClinicalTrials.gov

What this trial studies

This study evaluates the safety and efficacy of Alpha-1 Antitrypsin (AAT) in preventing acute graft-versus-host disease (aGVHD) in patients undergoing hematopoietic cell transplantation (HCT). It is a phase 2/3, double-blind, randomized, multicenter, placebo-controlled trial involving participants with hematological malignancies. Eligible patients will receive either AAT or a placebo following a planned myeloablative conditioning regimen. The study aims to determine if AAT can effectively reduce the incidence of aGVHD in this patient population.

Who should consider this trial

Good fit: Ideal candidates are males and females aged 12 and older undergoing HCT for hematological malignancies with a suitable related or unrelated donor.

Not a fit: Patients who have previously undergone autologous or allogeneic HCT or those receiving T cell depletion therapies may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly reduce the risk of aGVHD, improving outcomes for patients undergoing HCT.

How similar studies have performed: While similar approaches have been explored, this specific use of AAT for aGVHD prevention is novel and has not been extensively tested in prior studies.

Eligibility criteria

Show full inclusion / exclusion criteria 
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.

Where this trial is running

Scottsdale, Arizona and 33 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Acute-graft-versus-host Disease
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.