Stem Cell Transplant Prep Regimen for Immune Deficiency Disorders
Trial Summary
Do I need to stop my current medications for the trial?
The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
What data supports the effectiveness of the drug Alemtuzumab in the stem cell transplant prep regimen for immune deficiency disorders?
Alemtuzumab is effective in reducing the risk of graft-versus-host disease (GVHD) in pediatric stem cell transplants, as shown by a study where it was part of the pretransplant conditioning regimen, resulting in no chronic GVHD and a high survival rate. Additionally, Alemtuzumab has been used successfully in treating chronic lymphocytic leukemia, indicating its potential effectiveness in targeting immune cells.12345
Is there safety data for the use of thiotepa in stem cell transplant preparation?
What makes this stem cell transplant prep regimen unique for immune deficiency disorders?
This treatment regimen is unique because it uses a combination of drugs, including alemtuzumab, which helps reduce the risk of graft-versus-host disease (GVHD) by depleting T-cells (a type of immune cell), potentially leading to fewer complications compared to traditional regimens. Additionally, the use of reduced-intensity conditioning with drugs like fludarabine and melphalan aims to minimize toxicity while maintaining effectiveness.311121314
What is the purpose of this trial?
This study hypothesizes that a reduced intensity immunosuppressive preparative regimen will establish engraftment of donor hematopoietic cells with acceptable early and delayed toxicity in patients with immune function disorders. A regimen that maximizes host immune suppression is expected to reduce graft rejection and optimize donor cell engraftment.
Eligibility Criteria
This trial is for people under 28 with certain immune disorders like DiGeorge Syndrome and Wiskott-Aldrich Syndrome. They must have normal organ function, a performance status of at least 40%, and a matched donor for cell transplant. People with HIV, uncontrolled infections, or who are pregnant/breastfeeding can't join.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Preparative Regimen
Participants undergo a reduced intensity immunosuppressive preparative regimen to establish engraftment of donor hematopoietic cells
Hematopoietic Stem Cell Transplantation
Participants receive hematopoietic stem cell transplantation to optimize donor cell engraftment
Follow-up
Participants are monitored for safety and effectiveness after transplantation
Treatment Details
Interventions
- Alemtuzumab
- Fludarabine
- Melphalan
- Thiotepa
Alemtuzumab is already approved in United States, European Union for the following indications:
- Chronic lymphocytic leukemia
- Multiple sclerosis
- Multiple sclerosis
- Chronic lymphocytic leukemia
Find a Clinic Near You
Who Is Running the Clinical Trial?
Washington University School of Medicine
Lead Sponsor