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lack of recruitment
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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™.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| CordIn | Experimental | Transplantation of CordIn |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| CordIn | Biological | CordIn™ is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells. |
|
| 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 |
| 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 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Jean-Hugues Dalle, MD | Robert Debre, Paris, France | Principal Investigator |
| Franco Locatelli, MD | IRCCS Ospedale Pediatrico Bambino Gesu, Rome, Italy | Principal Investigator |
| Allistair Abraham, MD | Children's National, Washington DC | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCSF Benioff Children's Hospital | Oakland | California | 94609 | United States | ||
| Children's National |
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| ID | Title | Description |
|---|---|---|
| FG000 | 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. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | 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. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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. | Posted | Count of Participants | Participants | No | 24 hours |
|
|
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| GvHD | Immune system disorders | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Hypoalbuminemia | Gastrointestinal disorders | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Kelly Myers | Gamida Cell | 026595631 | kelly@gamida-cell.com |
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| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| D013789 | Thalassemia |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
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| Washington D.C. |
| District of Columbia |
| 20010 |
| United States |
| Hôpital Robert Debré | Paris | France |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | Years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
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| 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. | Posted | Count of Participants | Participants | 42 days |
|
|
|
| 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. | Posted | Count of Participants | Participants | 365 days |
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| 0 |
| 1 |
| 1 |
| 1 |
| 1 |
| 1 |
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| D006425 |
| Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |