Allogeneic SCT of CordIn™, in Patients With Hemoglobinopathies

Study Overview
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Brief Summary
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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™.

Detailed Description
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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.

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

Conditions
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Sickle Cell Disease, Thalassemia

Intervention / Treatment
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* Biological: CordIn


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 25 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: CordIn<br>Transplantation of CordIn | Biological: CordIn<br>* CordIn™ is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 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. | 42 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 365 days |