Allogeneic SCT of CordIn™, in Patients With Hemoglobinopathies

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

CordIn™ is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells. The overall study objectives are to evaluate the safety and efficacy of CordIn™.

CordIn™ is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells.

The trial ends when the last patient completes their last visit. The overall study objectives are to evaluate the safety and efficacy of CordIn™: single ex-vivo expanded cord blood unit transplantation in patients with hemoglobinopathies (sickle cell disease or thalassemia major) following a preparative therapy.

Inclusion Criteria:

• Patient is a candidate for allogeneic SCT for treatment of SCD or thalassemia
• Patients must have one partially HLA-matched CBUs
• Adequate Karnofsky Performance score or Lansky Play-Performance scale
• Sufficient physiological reserves
• Signed written informed consent

Exclusion Criteria:

• Prior allogeneic HSCT
• Evidence of HIV infection or HIV positive serology
• Evidence of active Hepatitis B, Hepatitis C or EBV as determined by serology or PCR
• Active or uncontrolled infection
• Pregnancy or lactation