Actively Recruiting
Allogeneic Hematopoietic Stem Cell Transplantation Using G-CSF Mobilized CD34+ Selected Cells Co-Infused With Reduced Dose Non-Mobilized Donor T-Cells for Severe Aplastic Anemia and Bone Marrow Failure Syndromes
Led by National Heart, Lung, and Blood Institute (NHLBI) · Updated on 2026-05-29
120
Participants Needed
2
Research Sites
26 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating a new approach to allogeneic hematopoietic stem cell transplantation (aHSCT) for treating severe aplastic anemia, myelodysplastic syndrome, and other bone marrow failure syndromes. This study aims to reduce the risk of graft rejection and severe complications such as chronic graft-versus-host disease (cGVHD) by modifying the transplant procedure. The trial focuses on patients aged 4 to 80 years who have blood diseases that may benefit from stem cell transplants. The treatment involves collecting stem cells and white blood cells from donors who are related or unrelated but matched through a donor program. Donors undergo procedures including initial white blood cell donation, injections of filgrastim to mobilize bone marrow cells into the blood, followed by stem cell collection. Recipients receive conditioning chemotherapy before transplant, followed by an infusion of donor stem cells and a controlled dose of donor T-cells to reduce complications. After transplant, recipients take medications to prevent rejection and remain hospitalized for up to one month, followed by regular follow-up visits. Participants will have multiple assessments including physical exams, medical history reviews, and laboratory tests. Researchers will monitor transplant success, immune system recovery, incidence of cGVHD, and overall health outcomes over several years. Quality of life will be evaluated before and after transplant at multiple timepoints up to five years. The study includes careful long-term follow-up to understand transplant outcomes and side effects.
CONDITIONS
Brief Title
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients aged 4 to 80 years with severe aplastic anemia, paroxysmal nocturnal hemoglobinuria, myelodysplastic syndrome, pure red cell aplasia, or related bone marrow failure syndromes
- Weight greater than 15 kg
- Availability of HLA matched or single locus mismatched related donor or 10/10 matched unrelated donor
- Germline gene testing if suspected familial bone marrow failure syndrome
- Related donors aged 4 to 80 years eligible and willing to donate blood samples for research
- Unrelated donors must meet National Marrow Donor Program eligibility criteria
You will not qualify if you...
- Major illness or organ failure incompatible with transplant survival
- Lung diffusion capacity (DLCO) less than 40% predicted (with exceptions for young children)
- Left ventricular ejection fraction less than 40%
- Severe kidney or liver dysfunction (creatinine >2.5 mg/dl, bilirubin >4 mg/dl, or high liver enzymes)
- Pregnant or lactating individuals
- Diagnosis of Fanconi anemia
- Performance status of 3 or higher
- Other active malignancies likely to relapse within 5 years (except some hematologic malignancies)
- Active infections not responding to treatment
- Inability to understand study and provide informed consent (with special consent rules for children aged 8-17)
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Day 0 and following immediate recovery period
Participants undergo allogeneic hematopoietic stem cell transplantation using G-CSF mobilized CD34+ selected hematopoietic precursor cells co-infused with a reduced dose of non-mobilized donor T-cells after conditioning with cyclophosphamide, fludarabine, and anti-thymocyte globulin (ATG).
1 transplant visit (in-person) followed by frequent hospital visits during initial recovery
Duration - Up to 5 years post-transplant
Participants are monitored for transplant-related outcomes including chronic graft versus host disease, engraftment success, transplant related mortality, and overall survival with assessments of health-related quality of life.
Visits at 30 and 100 days post transplant, then every 6 months up to 5 years
Trial Site Locations
Total: 2 locations
1
University of Maryland, Baltimore (UMB)
Baltimore, Maryland, United States, 21201
Completed
2
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
Research Team
M
Melissa M Spencer, R.N.
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
2
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