MODULAATE Study. Alpha-1 AntiTrypsin for prevention of GVHD post HSCT
Research type
Research Study
Full title
A Phase 2/3, Multicenter, randOmised, 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 haematopoietic stem cell transplantation (MODULAATE Study)
IRAS ID
242329
Contact name
Amit Patel
Contact email
Sponsor organisation
CSL Behring LLC
Eudract number
2018-000329-29
Duration of Study in the UK
4 years, 0 months, 0 days
Research summary
The participants will have a blood cancer that can be treated with a donated blood or bone marrow transplant, also called a haematopoietic stem cell transplant (HSCT). Before the transplant participants will begin to receive treatments to prevent graft-versus-host disease (GVHD). GVHD is a complication where the donor cells attack and damage some of the transplant recipient's tissues. The purpose of the study is to see if a treatment called Alpha 1-antitrypsin (AAT) along with the current standard of care can prevent GVHD better than the standard of care alone. The study will have 2 parts, the first part of the study is being carried out to see how well AAT is tolerated and to measure AAT levels in blood. Three different dose levels of AAT will be studied in Part 1, (low, medium or high) along with standard GVHD treatment. The dose that is well tolerated and maintains high levels in the blood will be used in Part 2. The second part of the study is being carried out to see if AAT along with standard treatment is effective in preventing GVHD and if so, how it compares with the standard treatment plus placebo. Approximately 310 participants will take part. All participants will receive conditioning, HCT and standard of care treatment to prevent GVHD. This is a randomised double blind study and after the first 50 participants there is an equal chance of receiving study drug or placebo. Participation in this study is expected to last approximately 1 year. This includes a 28 day screening period, 2 month treatment period and a 10 month follow-up period. Although the protocol allows for participants from 12 years old as the sites in the UK don’t treat paediatric patients the study will only be open to adults in the UK.
REC name
West Midlands - Edgbaston Research Ethics Committee
REC reference
19/WM/0046
Date of REC Opinion
31 May 2019
REC opinion
Further Information Favourable Opinion