Not recruitingRare disease

Study on the Safety and Effectiveness of Human Alpha1-Proteinase Inhibitor for Preventing Graft-Versus-Host Disease in Patients Undergoing Hematopoietic Cell Transplant.

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition known as Graft-versus-Host Disease (GVHD), which can occur after a patient receives a hematopoietic cell transplant (HCT). This condition happens when the donated cells attack the recipient's body. The study is testing a treatment called Alpha-1 Antitrypsin (AAT), which is being evaluated for its ability to prevent GVHD in patients who have undergone HCT. The treatment involves using a medication called Respreeza, which is a powder and solvent that is mixed to form a solution for infusion into the bloodstream.

The purpose of the study is to assess the safety and effectiveness of AAT in preventing GVHD. Participants in the study will receive either the AAT treatment or a placebo. The study will follow participants over a period of time to monitor for any signs of GVHD, as well as any side effects or other health changes. The study aims to determine if AAT can reduce the occurrence of GVHD and improve the overall health outcomes for patients receiving HCT.

Throughout the study, participants will receive regular medical check-ups and monitoring to ensure their safety and to track the progress of the treatment. The study will provide valuable information on whether AAT can be a beneficial treatment option for preventing GVHD in patients undergoing HCT. This research is important for improving the care and outcomes for patients who require this type of transplant.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Upon joining the study, you will be assessed to ensure you meet the eligibility criteria. This includes being at least 12 years old and undergoing a hematopoietic cell transplant (HCT) for certain blood cancers.
A planned myeloablative conditioning regimen is required before the transplant.
Step 2
Treatment initiation
The treatment involves the administration of Alpha-1 Antitrypsin (AAT) to prevent acute graft-versus-host disease (GVHD).
AAT is given as a solution for infusion, which means it is administered directly into your bloodstream through a vein.
Step 3
Monitoring and follow-up
You will be monitored for the development of Grade II-IV acute GVHD or any severe infections for up to 180 days after the transplant.
Regular assessments will be conducted to check for any adverse effects related to the study drug and to evaluate the effectiveness of the treatment.
Step 4
Long-term evaluation
The study will continue to monitor your health for up to 730 days after the transplant to assess long-term outcomes such as survival without GVHD relapse and the discontinuation of immune suppression therapies.
Your response to the treatment, including any relapses of the primary malignancy, will be evaluated during this period.

Who can join the trial?

5 criteria

Participants can be either male or female.
Participants must be at least 12 years old. If the study is conducted in Germany, participants must be at least 18 years old.
Participants should be undergoing a procedure called **HCT**. This stands for **Hematopoietic Cell Transplantation**, which is a treatment for certain blood-related cancers.
The types of blood-related cancers include leukemia, lymphoma, multiple myeloma, myelodysplastic syndrome, and myeloproliferative neoplasms.
Participants must have a planned **myeloablative conditioning regimen**. This is a strong treatment given before the transplant to prepare the body to receive new cells.

Who cannot join the trial?

5 criteria

Patients who have a different medical condition than the one being studied, which is Acute Graft versus Host Disease. This is a condition that can occur after a transplant, where the donated cells attack the recipient's body.
Patients who are not within the specified age range for the study. The study includes certain age groups, so if a patient is too young or too old, they may not be eligible.
Patients who are not part of the specific clinical trial group being studied. This means the study is looking for people with certain characteristics or conditions.
Patients who are not male or female, as the study includes both genders.
Patients who are considered part of a vulnerable population. This means people who might need special protection or care, such as children or those with certain disabilities, may not be eligible.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Azienda Ospedaliero-Universitaria Policlinico G. Rodolico-San Marco Di Catania
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Catania, Italy
Hospital General Universitario Gregorio Maranon
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Madrid, Spain
Hospital Universitario De Salamanca
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Salamanca, Spain

Trial locations

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Investigated drugs

Alpha-1 Antitrypsin (AAT) is being studied for its potential to prevent graft versus host disease (GVHD) in patients who have received a hematopoietic cell transplant. GVHD is a condition where the donated cells attack the recipient's body. AAT is a protein that may help reduce inflammation and protect tissues, which could be beneficial in preventing this complication after a transplant.

Human Alpha1-Proteinase Inhibitor

What is already known about the treatment

Alpha-1 Antitrypsin – This medication is administered intravenously and is currently being studied in clinical trials for its potential to prevent acute graft versus host disease in patients undergoing hematopoietic cell transplantation. It is in the investigational stage, with ongoing research to determine its safety and effectiveness. The main therapeutic indication is the prevention of acute graft versus host disease. At the molecular level, Alpha-1 Antitrypsin works by inhibiting enzymes that can cause tissue damage, thereby reducing inflammation and immune response. It is classified pharmacologically as a protease inhibitor.

Investigated diseases

Acute Graft versus Host Disease – This condition occurs when donor immune cells attack the recipient's body after a hematopoietic cell transplantation. It typically affects the skin, liver, and gastrointestinal tract. Symptoms can include skin rashes, jaundice, and diarrhea. The disease is classified into grades based on severity, with Grade II-IV indicating more severe forms. It usually develops within the first 100 days post-transplantation. The progression can vary, with some cases resolving spontaneously while others may persist or worsen.