Title
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
Phase
Phase 1/Phase 2Lead Sponsor
Gamida CellStudy Type
InterventionalStatus
Terminated Results PostedIndication/Condition
Sickle Cell Disease ThalassemiaIntervention/Treatment
codeine ...Study Participants
1CordIn™ 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.
CordIn™ is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells.
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
| Event Type | Organ System | Event Term | CordIn |
|---|
The number of patients with grade 4 or 5 toxicity were estimated together with 95% confidence limits based on the binomial distribution. The proportion with toxicity grades 1, 2, and 3 was also estimated.
One hundred percent of patients engrafted by day forty-two post-transplantation with donor-derived cells.
The percentage of patients alive at one year post-transplant was estimated using the Kaplan-Meier method.
