3 United States sites
2 to 45 Years
SC
Phase 1/Phase 2
Interventional
All
Drug
Unknown
Allogeneic Stem Cell Transplantation of NiCord®, Umbilical Cord Blood-Derived Ex Vivo
Expanded Stem and Progenitor Cells, in Patients with Hemoglobinopathies
– Must be 2 – 45 years of age and at least 10 kg
– Must have clinically severe SCD (SS, SC or SBeta0 Thal) or thalassemia major and be
eligible for myeloablative SCT
– Must have two partially HLA-matched CBUs for part 1; and one partially HLA-matched CBU
for part 2
– Back-up autologous stem cells harvested from bone marrow
– Adequate Karnofsky Performance score or Lansky Play-Performance scale
– Sufficient physiological reserves
– Signed written informed consent
– HLA-matched related donor able to donate
– Severe alloimmunization with inability to guarantee a supply of adequate PRBC donors
– Prior allogeneic hematopoietic SCT within the last 12 months or reduced-intensity
transplant within the past 6 months
– Human immunodeficiency virus (HIV) infection
– Active or uncontrolled infection
– Pregnancy or lactation