Allogeneic SCT of CordIn™, in Patients With Hemoglobinopathies
Study Details
Study Description
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™.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 1/Phase 2 |
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.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: CordIn Transplantation of CordIn |
Biological: CordIn
CordIn™ is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells.
|
Outcome Measures
Primary Outcome Measures
- 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 Outcome Measures
- 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.
Eligibility Criteria
Criteria
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
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | UCSF Benioff Children's Hospital | Oakland | California | United States | 94609 |
| 2 | Children's National | Washington | District of Columbia | United States | 20010 |
| 3 | Hôpital Robert Debré | Paris | France |
Sponsors and Collaborators
- Gamida Cell ltd
Investigators
- Principal Investigator: 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
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- GC P#01.01.030
Study Results
Participant Flow
| Recruitment Details | |
|---|---|
| Pre-assignment Detail |
| Arm/Group Title | CordIn |
|---|---|
| Arm/Group Description | 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. |
| Period Title: Overall Study | |
| STARTED | 1 |
| COMPLETED | 1 |
| NOT COMPLETED | 0 |
Baseline Characteristics
| Arm/Group Title | CordIn |
|---|---|
| Arm/Group Description | 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. |
| Overall Participants | 1 |
| Age (Count of Participants) | |
| <=18 years |
1
100%
|
| Between 18 and 65 years |
0
0%
|
| >=65 years |
0
0%
|
| Age (Years) [Mean (Standard Deviation) ] | |
| Mean (Standard Deviation) [Years] |
2.8
(0)
|
| Sex: Female, Male (Count of Participants) | |
| Female |
0
0%
|
| Male |
1
100%
|
| Ethnicity (NIH/OMB) (Count of Participants) | |
| Hispanic or Latino |
0
0%
|
| Not Hispanic or Latino |
1
100%
|
| Unknown or Not Reported |
0
0%
|
| Race (NIH/OMB) (Count of Participants) | |
| American Indian or Alaska Native |
0
0%
|
| Asian |
0
0%
|
| Native Hawaiian or Other Pacific Islander |
0
0%
|
| Black or African American |
1
100%
|
| White |
0
0%
|
| More than one race |
0
0%
|
| Unknown or Not Reported |
0
0%
|
| Region of Enrollment (participants) [Number] | |
| United States |
1
100%
|
Outcome Measures
| Title | Number of Participants With no Acute Toxicity Associated With the Infusion of CordIn, Within 24 Hours Post-infusion. |
|---|---|
| Description | 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 |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | CordIn |
|---|---|
| Arm/Group Description | 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. |
| Measure Participants | 1 |
| Count of Participants [Participants] |
1
100%
|
| Title | The Percentage of Patients With Donor-derived Engraftment at 42 Days Following Transplantation |
|---|---|
| Description | One hundred percent of patients engrafted by day forty-two post-transplantation with donor-derived cells. |
| Time Frame | 42 days |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | CordIn |
|---|---|
| Arm/Group Description | 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. |
| Measure Participants | 1 |
| Count of Participants [Participants] |
1
100%
|
| Title | Percentage of Overall Survival at 365 Days After Transplantation |
|---|---|
| Description | The percentage of patients alive at one year post-transplant was estimated using the Kaplan-Meier method. |
| Time Frame | 365 days |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | CordIn |
|---|---|
| Arm/Group Description | 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. |
| Measure Participants | 1 |
| Count of Participants [Participants] |
1
100%
|
Adverse Events
| Time Frame | ||
|---|---|---|
| Adverse Event Reporting Description | ||
| Arm/Group Title | CordIn | |
| Arm/Group Description | 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. | |
All Cause Mortality |
||
| CordIn | ||
| Affected / at Risk (%) | # Events | |
| Total | 0/1 (0%) | |
Serious Adverse Events |
||
| CordIn | ||
| Affected / at Risk (%) | # Events | |
| Total | 1/1 (100%) | |
| Immune system disorders | ||
| GvHD | 1/1 (100%) | 2 |
Other (Not Including Serious) Adverse Events |
||
| CordIn | ||
| Affected / at Risk (%) | # Events | |
| Total | 1/1 (100%) | |
| Gastrointestinal disorders | ||
| Hypoalbuminemia | 1/1 (100%) | 2 |
Limitations/Caveats
More Information
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
| Name/Title | Kelly Myers |
|---|---|
| Organization | Gamida Cell |
| Phone | 026595631 |
| kelly@gamida-cell.com |
- GC P#01.01.030