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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
1
CordIn™ is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells.
UCSF Benioff Children's Hospital
Oakland, California, United States
Children's National
Washington D.C., District of Columbia, United States
Hôpital Robert Debré
Paris, France
Number of Participants With no Acute Toxicity Associated With the Infusion of CordIn, Within 24 Hours Post-infusion.
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.
Time frame: 24 hours
The Percentage of Patients With Donor-derived Engraftment at 42 Days Following Transplantation
One hundred percent of patients engrafted by day forty-two post-transplantation with donor-derived cells.
Time frame: 42 days
Percentage of Overall Survival at 365 Days After Transplantation
The percentage of patients alive at one year post-transplant was estimated using the Kaplan-Meier method.
Time frame: 365 days
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