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

Phase 1

Recruiting

• Conditions: Acute Graft versus host disease; MedDRA version: 20.1Level: PTClassification code: 10018651Term: Graft versus host disease Class: 100000004870; Therapeutic area: Phenomena and Processes [G] - Immune system processes [G12]

• Registration Number: CTIS2024-511164-92-00
• Lead Sponsor: CSL Behring LLC

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-04-29
• Last Update Posted: 22 July 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 310

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 transplant.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: Primary objective is to evaluate the efficacy of AAT at the selected dose for the prevention of acute GVHD following HCT.;Secondary Objective: Key Secondary Objective is To evaluate the efficacy of AAT for the prevention of severe aGVHD and infections following HCT. Also, 1. To further evaluate the efficacy of AAT for the prevention of complications after HCT. 2. To evaluate the safety of AAT based on investigational product- (IP)- related AEs. 3. To evaluate the steady state PK of AAT in HCT recipients.;Primary end point(s): The time to Grade II-IV aGVHD or death through 180 days after hematopoietic cell transplantation (HCT).