Allogeneic Stem Cell Transplantation of NiCord®, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients With Hemoglobinopathies

Locations

3 United States sites

Age

2 to 45 Years

Genotypes

SC

Phase

Phase 1/Phase 2

Study type

Interventional

Gender

All

Interventions

Drug

Compensation

Unknown

About the study

Allogeneic Stem Cell Transplantation of NiCord®, Umbilical Cord Blood-Derived Ex Vivo
Expanded Stem and Progenitor Cells, in Patients with Hemoglobinopathies

participation requirements

– 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

participation restrictions

– 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

Locations

  • New York, New York, United States, Steven & Alexandra Cohen Children's Medical Center, New York, 11040
  • Durham, North Carolina, United States, Duke University Medical Center, 27705
  • Houston, Texas, United States, The University Of Texas M. D. Anderson Cancer Center, 77030-4009