Allogeneic SCT of NiCord®, UCB-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients With Hemoglobinopathies

• Status: Unknown status
• Trial end date: 2020-01-01
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Gamida Cell ltd

Criteria

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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