This study evaluates the safety and effectiveness of haploidentical donor-based transplantation for patients with severe aplastic anemia who lack an available HLA-matched donor.
Allogeneic hematopoietic cell transplantation (HCT) is an effective therapy for several acquired and inherited bone marrow failure syndromes. Allogeneic HCT from a matched sibling donor is the first-line treatment strategy for patients under 40 years old with severe aplastic anemia. Salvage allogeneic HCT from an HLA-matched unrelated donor is recommended for patients with refractory severe aplastic anemia that is unresponsive to immunosuppressive therapy. However, many patients are unable to find a matched, unrelated donor in the registry.
The goal of this study is to develop a novel, reduced-toxicity, GVHD prophylaxis-free, highly tolerogenic haploidentical transplant regimen that is associated with few post-transplant complications or late toxicities and is available to all patients, irrespective of matched donor availability.
- To estimate the rate of engraftment at 30 days and overall survival and event-free survival at 1 year after TCR ab+ T-cell-depleted graft infusion in patients with severe aplastic anemia
Inclusion criteria for transplant recipient include:
- 21 years old or younger
- Diagnosis of severe aplastic anemia or a single lineage cytopenia
- Does not have a suitable HLA-matched sibling donor or volunteer 10/10 HLA-matched unrelated donor available in the necessary time for progenitor cell donation
- Failed at least one trial of immunosuppressive therapy by being refractory or having relapsed
- Has a suitable single haplotype matched family member donor
Exclusion Criteria for transplant recipient include:
- Diagnosis of Fanconi anemia, dyskeratosis congenita, or myelodyplastic syndrome (MDS)
- Clonal cytogenetic abnormalities consistent with pre-MDS or MDS on marrow examination
- Presence of anti-donor HLA antibodies
- Prior allogeneic hematopoietic cell transplant or prior solid organ transplant
St. Jude Children’s Research Hospital
About this study
This is a study to learn more about a type of stem cell transplant in patients with severe aplastic anemia who do not have a matched blood cell donor. A stem cell transplant is also known as a blood cell transplant or a bone marrow transplant.
When a patient needs a blood cell transplant, the first choice of a donor is a matched sibling (brother or sister) donor. When a matched sibling is not available, a matched, unrelated volunteer donor may be used. If none of these donors is available, a family member who is not a full match may be used.
If you participate in this study, you will receive blood cells that have been donated by a half-matched family member and modified in a laboratory at St. Jude. These blood cells are called TCR ab-depleted blood cells. The procedure used to process the blood cells in this study is considered experimental because it has not been approved by the U.S. Food and Drug Administration (FDA).
Once the donor cells have been collected and processed, you will receive radiation and chemotherapy (anti-cancer medicine) to prepare your bone marrow space for the new donor cells. You will be given the donor blood cells through a vein. This procedure is called infusion. These new cells find their way to your bone marrow space and begin to grow and produce new blood cells.
Purpose of this clinical trial
The main goal of this study is to learn more about the effects (good and bad) of this treatment in children and young adults with high-risk blood disorders.
- 21 years old and younger
- Diagnosis of severe aplastic anemia
- No available matched donor
HAPSAA Sponsors: St. Jude Children’s Research Hospital ClinicalTrials.gov identifier NCT04558736 Trial Start Date: January 2021 Estimated Enrollment: 21 Study Type: Interventional Study Phase: Phase 2 Conditions: Aplastic Anemia Ages: 21 years and younger Principal investigator: Amr Qudeimat, MD
Akshay Sharma, MBBS
Study Sites: St. Jude Children’s Research Hospital and collaborating sites outside the U.S. For a consultation or to discuss MNEMONIC: St. Jude Physician/Patient Referral Office
St. Jude Children’s Research Hospital
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Memphis, TN 38105 USA
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