Anemia Clinical Trial
Official title:
Phase I/II Trial Of Hematopoietic Stem Cell Transplant (HSCT) For Children With A Genetic Disease Of Blood Cells Without An HLA-Matched Sibling Donor
This is a clinical trial of bone marrow transplantation for patients with the diagnosis of a
genetic disease of blood cells that do not have an HLA-matched sibling donor. Genetic
diseases of blood cell include: Red blood cell defects e.g. hemoglobinopathies (sickle cell
disease and thalassemia), Blackfan-Diamond anemia and congenital or chronic hemolytic
anemias; White blood cells defects/immune deficiencies e.g. chronic granulomatous disease,
Wiskott-Aldrich syndrome,Osteopetrosis, Kostmann's syndrome (congenital neutropenia),
Hereditary Lymphohistiocytosis (HLH); Platelets defects e.g.Congenital amegakaryocytic
thrombocytopenia; Metabolic/storage disorders e.g. leukodystrophies,mucopolysaccharidoses as
Hurler disease;Stem cell defects e.g.reticular agenesis, among many other rare similar
conditions.
The study treatment plan uses a new transplant treatment regimen that aims to try to
decrease the acute toxicities and complications associated with the standard treatment plans
and to improve outcome
The blood stem cells will be derived from either unrelated donor or unrelated umbilical cord
blood.
This is a pilot clinical trial of hematopoietic stem cell transplantation for patients with
the diagnosis of a genetic disease of blood cells that do not have an HLA-matched sibling
donor. The stem cells will be derived from a 1) matched unrelated donor (MUD) or 2)
unrelated umbilical cord blood (UCB). Patients will receive a novel conditioning regimen
with Busulfan, Cytoxan and Fludarabine (Bu/Cy/Flu) and either Alemtuzumab (Campath 1H) for
recipients of a MUD or rabbit Antithymocyte Globulin (rATG) for recipients of unrelated UCB
prior to hematopoietic stem cell transplant (HSCT).
It is hypothesized that reduced dosages of Cytoxan will decrease the acute toxicities
associated with the standard chemotherapies of Busulfan and Cytoxan (i.e. sinusoidal
obstructive syndrome (SOS), hemorrhagic cystitis and mucositis). And the addition of
fludarabine to a conditioning regimen with myeloablative doses of Busulfan and reduced
dosages of Cytoxan prior to HSCT will overcome the engraftment barrier posed by an intact
immune system, which is seen in patients with a genetic disease.
;
Allocation: Non-Randomized, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
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