Sickle Cell Disease, Thalassemia
Conditions
Brief summary
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
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.
Interventions
BIOLOGICALCordIn
CordIn™ is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells.
Sponsors
Gamida Cell ltd
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Years to 25 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With no Acute Toxicity Associated With the Infusion of CordIn, Within 24 Hours Post-infusion. | 24 hours | 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. |
| The Percentage of Patients With Donor-derived Engraftment at 42 Days Following Transplantation | 42 days | One hundred percent of patients engrafted by day forty-two post-transplantation with donor-derived cells. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Overall Survival at 365 Days After Transplantation | 365 days | The percentage of patients alive at one year post-transplant was estimated using the Kaplan-Meier method. |
Countries
France, United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| CordIn Transplantation of CordIn
CordIn: CordIn™ is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells. | 1 |
| Total | 1 |
Baseline characteristics
| Characteristic | CordIn |
|---|---|
| Age, Categorical <=18 years | 1 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants |
| Age, Continuous | 2.8 Years STANDARD_DEVIATION 0 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 1 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 0 Participants |
| Region of Enrollment United States | 1 participants |
| Sex: Female, Male Female | 0 Participants |
| Sex: Female, Male Male | 1 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 1 |
| other Total, other adverse events | 1 / 1 |
| serious Total, serious adverse events | 1 / 1 |
Outcome results
Primary
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
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| CordIn | Number of Participants With no Acute Toxicity Associated With the Infusion of CordIn, Within 24 Hours Post-infusion. | 1 Participants |
Primary
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
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| CordIn | The Percentage of Patients With Donor-derived Engraftment at 42 Days Following Transplantation | 1 Participants |
Secondary
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
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| CordIn | Percentage of Overall Survival at 365 Days After Transplantation | 1 Participants |