Actively Recruiting
Haplo-identical Transplantation Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for Severe Aplastic Anemia, Hypoplastic Myelodysplastic Syndrome, and PNH
Led by National Heart, Lung, and Blood Institute (NHLBI) · Updated on 2026-06-05
56
Participants Needed
1
Research Sites
104 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This research investigates the safety and effectiveness of using peripheral blood hematopoietic stem cells from a family member combined with chemotherapy to treat severe aplastic anemia (SAA), hypoplastic myelodysplastic syndrome (MDS), and paroxysmal nocturnal hemoglobinuria (PNH). These serious bone marrow disorders often require stem cell transplants, and the study aims to evaluate whether peripheral blood stem cells (PBSCs) are a good alternative to bone marrow cells, especially for patients without an HLA-matched donor. The study also assesses post-transplant cyclophosphamide as a method to prevent graft-versus-host disease (GVHD). Participants receive stem cells mobilized by G-CSF injections from a haploidentical donor, followed by chemotherapy over eight days and a single radiation treatment. The transplant procedure includes inserting a central line, administering medicines for side effects, and infusing stem cells over about four hours. Donors undergo blood and tissue tests, receive injections to boost stem cell production, and donate blood stem cells collected through a machine that separates the needed cells before returning the rest to the donor. Recipients stay in the hospital for approximately one month after transplant and are monitored weekly for up to six months near NIH with physical exams and blood tests. After day 180, participants return home but continue follow-up visits at their doctor’s office and NIH multiple times over five years. The primary study outcome is survival without moderate or severe chronic GVHD at one year, with other measures including transplant success, treatment-related mortality, disease relapse, and quality of life.
CONDITIONS
Brief Title
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosed with severe aplastic anemia with bone marrow cellularity below 30% and transfusion dependence or neutropenia
- History of severe aplastic anemia transformed to MDS with IPSS risk category INT-1 or greater and bone marrow cellularity below 30%
- PNH refractory to treatment or without access to treatment, with life-threatening thrombosis, transfusion-dependent cytopenia, or recurrent hemolytic crisis
- Intolerance or failure to respond to immunosuppressive therapy including ATG and cyclosporine
- Availability of at least one haploidentical related donor with 5/10 or greater HLA match
- Availability of a backup stem cell source (alternative haploidentical donor, matched unrelated donor, or umbilical cord blood unit)
- No detectable HLA antibodies against mismatched donor alleles
- Age between 4 and 60 years inclusive
- Ability to understand the study and provide informed consent (parents/guardians consent for ages 4-17)
You will not qualify if you...
- Availability of a fully matched (12/12) related or unrelated donor suitable for transplant
- Candidate for a 12/12 matched unrelated stem cell transplant
- ECOG performance status 2 or higher
- Major illness or organ failure incompatible with transplant survival
- Current pregnancy or unwillingness to use birth control for one year if of childbearing potential
- HIV positive status
- Diagnosis of Fanconi's anemia
- Lung diffusion capacity less than 40% (with exceptions for young children)
- Left ventricular ejection fraction below 40%
- Liver enzyme levels more than 5 times upper limit of normal
- Direct bilirubin over 3 mg/dl
- Reduced kidney function with creatinine clearance below 50 cc/min/m2 or serum creatinine over 2.5 mg/dl
- Active infection not responding to treatment
- History of cancer likely to relapse or progress within 5 years
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.
screening and enrollment visit
Duration - Duration of active treatment and initial recovery period
Participants receive a haplo-identical transplantation using peripheral blood stem cells followed by post-transplant cyclophosphamide to prevent graft-versus-host disease.
1 transplantation visit with multiple follow-up visits during initial recovery
Duration - Up to 1 year post-transplant
Participants are monitored after treatment for chronic GVHD-free survival and other transplant outcomes up to 1 year post-transplant.
Regular follow-up visits over 1 year
Trial Site Locations
Total: 1 location
1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
Research Team
J
Jennifer A Farren
R
Richard W Childs, M.D.
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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