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
- Conditions
- Graft versus host diseaseMedDRA version: 20.1 Level: PT Classification code 10018651 Term: Graft versus host disease System Organ Class: 10021428 - Immune system disordersTherapeutic area: Body processes [G] - Immune system processes [G12]
- Registration Number
- EUCTR2018-000329-29-GB
- Lead Sponsor
- CSL Behring LLC
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- Not specified
- Target Recruitment
- 310
Inclusion Criteria
1. Male or female subjects, =12 years of age (= 18 years of age for subjects at German sites only who are enrolled into Part 2), undergoing HCT for hematological malignancies, including leukemia, lymphoma multiple myeloma, MDS and MPN
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
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
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To evaluate the efficacy of AAT at the selected dose for the prevention of acute GVHD following HCT.;<br> Primary end point(s): Grade II-IV acute graft versus host disease-free survival (aGFS)<br> ;<br> Timepoint(s) of evaluation of this end point: Through 180 days post-hematopoietic cell transplantation (HCT)<br> ;<br> Secondary Objective: 1. To evaluate the efficacy of AAT, including the prevention of post-hematopoietic cell transplant complications. <br> 2. To evaluate the safety of AAT, based on incidence of systemic infections and related adverse events. <br> 3. To evaluate the pharmacokinetics of AAT in HCT recipients.<br>
- Secondary Outcome Measures
Name Time Method