Stem Cell Transplant for Aplastic Anemia
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial explores a new treatment approach for individuals with dyskeratosis congenita or severe aplastic anemia. Patients first receive a special mix of medications, including Alemtuzumab (also known as Campath, Lemtrada, or MabCampath), Anti-thymocyte globulin, Cyclophosphamide (also known as Cytoxan, Neosar, or Endoxan), and Fludarabine (also known as Fludara, Oforta, or Beneflur), followed by a stem cell transplant. The goal is to determine if this combination can improve bone marrow function, reducing the need for red blood cell and platelet transfusions. Individuals experiencing frequent transfusions or diagnosed with these conditions and bone marrow failure might be suitable candidates. As an unphased trial, this study offers participants the chance to contribute to groundbreaking research that could lead to new treatment options.
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.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
In a previous study, alemtuzumab demonstrated that 67% of patients with severe aplastic anemia survived for six years, although it can weaken the immune system for up to two years. Anti-thymocyte globulin (ATG) achieved a response rate of about 70% to 77% within the first year. However, patients using rabbit ATG experienced lower survival rates.
Research has shown that cyclophosphamide can lead to high survival rates, with up to 75% of patients surviving eight years after treatment, especially when combined with total body irradiation. Fludarabine-based treatments have demonstrated an 83.7% survival rate, with some studies reporting 100% survival in certain situations.
Hematopoietic stem cell transplants (HSCT) have demonstrated impressive long-term survival rates, often above 75%, making them a promising option for bone marrow failure. Combining total body irradiation with HSCT has also resulted in excellent outcomes, with survival rates over 75%.
These treatments are generally well-tolerated, but side effects like weakened immunity and other complications can occur. Each treatment has its own risks and benefits, so discussing these with a healthcare provider is important before deciding to participate in a clinical trial.12345Why are researchers excited about this trial's treatments?
Researchers are excited about these treatments for aplastic anemia because they employ a unique combination of medications and techniques that could improve outcomes for patients. Unlike standard treatments, which typically involve immunosuppressive therapy or bone marrow transplants from matched donors, these investigational arms incorporate a fludarabine-based preparative regimen. This regimen includes novel combinations of drugs like alemtuzumab and anti-thymocyte globulin, as well as total body irradiation, to optimize the body's readiness for a stem cell transplant. By exploring these diverse approaches, researchers hope to enhance the efficacy and safety of stem cell transplants, potentially offering new solutions for patients with severe aplastic anemia and related conditions.
What evidence suggests that this trial's treatments could be effective for aplastic anemia?
Research has shown that a treatment plan combining fludarabine, cyclophosphamide, antithymocyte globulin (ATG), and total body irradiation (TBI) is promising for severe aplastic anemia. In this trial, participants in arms B, C, and E will receive this regimen followed by a stem cell transplant. Studies have found that this approach leads to high survival rates, with some reports indicating that over 75% of patients survive for at least eight years. This method is particularly effective for patients receiving transplants from unrelated donors, offering hope for those without a matching family donor.
For dyskeratosis congenita, a condition where the body struggles to produce enough blood cells, participants in arms A and D will receive a stem cell transplant using alemtuzumab and fludarabine. The survival rate for patients using similar treatments is about 71%, comparable to other less intense treatment options. These treatments aim to replace the faulty blood system with healthy donor cells, providing a lifeline to those affected.26789Who Is on the Research Team?
Jakub Tolar, MD
Principal Investigator
University of Minnesota
Are You a Good Fit for This Trial?
This trial is for people aged 0-70 with dyskeratosis congenita or severe aplastic anemia needing blood transfusions. Participants must have specific blood counts, organ function levels, and genetic mutations related to the conditions. Pregnant individuals, those with uncontrolled infections, certain liver issues, or a diagnosis of Fanconi anemia are excluded.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Preparative Regimen
Participants receive a fludarabine-based preparative regimen including alemtuzumab, cyclophosphamide, and fludarabine, followed by a stem cell transplant
Engraftment Monitoring
Participants are monitored for neutrophil and platelet engraftment
Follow-up
Participants are monitored for regimen-related mortality, acute and chronic graft-versus-host disease, and secondary malignancies
What Are the Treatments Tested in This Trial?
Interventions
- Alemtuzumab
- Anti-thymocyte globulin
- Cyclophosphamide
- Fludarabine
- Hematopoietic Stem Cell Transplant
- Total Body Irradiation
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?
Masonic Cancer Center, University of Minnesota
Lead Sponsor