Alpha-1 Antitrypsin for Graft-versus-Host Disease
(MODULAATE Trial)
Trial Summary
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
Research shows that Alpha-1 Antitrypsin (AAT) improved symptoms in patients with severe graft-versus-host disease (GVHD) who did not respond to steroids, with some achieving complete recovery. In one study, 8 out of 12 patients showed improvement, and in another, 80% of patients had a positive response, indicating AAT's potential as an effective treatment for GVHD.
12345Alpha-1 Antitrypsin (AAT) has been shown to be generally safe in humans, with studies reporting it as well tolerated and without significant toxicities. In various trials, including those for graft-versus-host disease and other conditions, no major safety concerns were observed, and treatment-related adverse events were relatively low.
13456Alpha-1 Antitrypsin (AAT) is unique because it has anti-inflammatory and immunomodulatory properties that help manage steroid-refractory graft-versus-host disease (GVHD), especially in patients who do not respond to standard steroid treatments. It works by altering immune responses and promoting tolerance, which is different from traditional treatments that primarily rely on steroids.
13457Eligibility Criteria
This trial is for males and females aged 12 or older (18+ in Germany) who are undergoing a bone marrow transplant due to blood cancers like leukemia, lymphoma, or myeloma. They must be planning an intense treatment regimen before the transplant. People with previous transplants or those getting certain T-cell treatments can't join.Inclusion Criteria
Exclusion Criteria
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive Alpha-1 Antitrypsin (AAT) or placebo intravenously to prevent acute GVHD
Follow-up
Participants are monitored for safety and effectiveness after treatment, including assessment of acute and chronic GVHD
Open-label extension (optional)
Participants may opt into continuation of treatment long-term with selected dose of AAT
Participant Groups
Alpha-1 antitrypsin (AAT) is already approved in United States, European Union for the following indications:
- Chronic obstructive pulmonary disease (COPD) associated with alpha-1 antitrypsin deficiency
- Chronic obstructive pulmonary disease (COPD) associated with alpha-1 antitrypsin deficiency