Actively Recruiting
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
Led by National Heart, Lung, and Blood Institute (NHLBI) · Updated on 2026-05-13
56
Participants Needed
1
Research Sites
484 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Background: Severe aplastic anemia (SAA), and myelodysplastic syndrome (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) cause serious blood problems. Stem cell transplants using bone marrow or blood plus chemotherapy can help. Researchers want to see if using peripheral blood stem cells (PBSCs) rather than bone marrow cells works too. PBSCs are easier to collect and have more cells that help transplants. Objectives: To see how safely and effectively SAA, MDS and PNH are treated using peripheral blood hematopoietic stem cells from a family member plus chemotherapy. Eligibility: Recipients ages 4-60 with SAA, MDS or PNH and their relative donors ages 4-75 Design: Recipients will have: * Blood, urine, heart, and lung tests * Scans * Bone marrow sample Recipients will need a caregiver for several months. They may make fertility plans and a power of attorney. Donors will have blood and tissue tests, then injections to boost stem cells for 5-7 days. Donors will have blood collected from a tube in an arm or leg vein. A machine will separate stem cells and maybe white blood cells. The rest of the blood will be returned into the other arm or leg. In the hospital for about 1 month, recipients will have: * Central line inserted in the neck or chest * Medicines for side effects * Chemotherapy over 8 days and radiation 1 time * Stem cell transplant over 4 hours Up to 6 months after transplant, recipients will stay near NIH for weekly physical exams and blood tests. At day 180, recipients will go home. They will have tests at their doctor s office and NIH several times over 5 years.
CONDITIONS
Official 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 less than 30% and transfusion dependence or neutropenia
- History of severe aplastic anemia transformed to myelodysplastic syndrome with specific risk criteria
- Paroxysmal nocturnal hemoglobinuria refractory to treatment or with serious symptoms
- Patients with intolerance or failure to respond to immunosuppressive therapy
- Availability of at least one haploidentical related donor matching at least 5 out of 10 HLA markers
- Availability of a backup stem cell source in case of graft rejection
- No detectable HLA antibodies against mismatched donor alleles
- Ages 4 to 60 years inclusive
- Ability to understand the study and provide informed consent (parent or guardian consent for minors)
You will not qualify if you...
- Availability of a fully matched (12/12) related or unrelated donor suitable for transplant
- Candidate for a fully matched unrelated stem cell transplant
- ECOG performance status of 2 or more
- Major illness or organ failure incompatible with transplant survival
- Current pregnancy or unwillingness to use birth control 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 less than 40%
- Transaminases more than 5 times the upper limit of normal
- Direct bilirubin over 3 mg/dl
- Creatinine clearance less than 50 cc/min/BSAm2
- Serum creatinine over 2.5 mg/dl
- Active infection not responding to treatment
- History of malignant disease likely to relapse within 5 years
AI-Screening
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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
CONTACT
R
Richard W Childs, M.D.
CONTACT
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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