TCD HCT for Fanconi Anemia
Trial Summary
What is the purpose of this trial?
This is a phase II trial of T cell receptor alpha/beta depletion (α/β TCD) peripheral blood stem cell (PBSC) transplantation in patients with inherited bone marrow failure (BMF) disorders to eliminate the need for routine graft-versus-host disease (GVHD) immune suppression leading to earlier immune recovery and potentially a reduction in the risk of severe infections after transplantation.
Will I have to stop taking my current medications?
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.
What data supports the effectiveness of the treatment T Cell Receptor α/β TCD HCT for Fanconi Anemia?
A study involving 24 patients with Fanconi Anemia showed that using TCRαβ+/CD19+-depleted stem cell transplantation led to successful engraftment in 91.6% of patients, with high survival rates and low incidence of severe complications. This suggests that the treatment is effective in achieving stable blood cell production and reducing complications in these patients.12345
Is T Cell Receptor α/β TCD HCT generally safe for humans?
What makes the T Cell Receptor α/β TCD HCT treatment unique for Fanconi Anemia?
The T Cell Receptor α/β TCD HCT treatment is unique because it involves the selective depletion of α/β T cells, which can reduce the risk of graft-versus-host disease (a condition where the donor cells attack the recipient's body) while preserving other immune cells that can help fight infections and support recovery.1011121314
Research Team
Margaret MacMillan, MD
Principal Investigator
Masonic Cancer Center, University of Minnesota
Eligibility Criteria
This trial is for patients with Fanconi anemia who have a suitable donor for blood cell transplant, are under 65 years old, and have good heart and lung function. They must not be pregnant or breastfeeding, have had solid tumor cancer in the last 2 years, or active infections. Participants need to agree to use contraception during treatment.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
T cell receptor alpha/beta depletion (α/β TCD) peripheral blood stem cell transplantation
Follow-up
Participants are monitored for safety and effectiveness after treatment
Long-term follow-up
Monitoring for chronic graft versus host disease and overall survival
Treatment Details
Interventions
- T Cell Receptor α/β TCD HCT
T Cell Receptor α/β TCD HCT is already approved in European Union, United States for the following indications:
- Inherited Bone Marrow Failure Disorders
- Fanconi Anemia
- Primary Immunodeficiency Diseases
- Inherited Bone Marrow Failure Disorders
- Fanconi Anemia
- Primary Immunodeficiency Diseases
- Nonmalignant Diseases
Find a Clinic Near You
Who Is Running the Clinical Trial?
Masonic Cancer Center, University of Minnesota
Lead Sponsor