Back to resultsAllogeneic SCT of CordIn™, in Patients With Hemoglobinopathies
Primary Purpose
Sickle Cell Disease, Thalassemia
Status
Terminated
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
CordIn
Sponsored by
About this trial
This is an interventional treatment trial for Sickle Cell Disease
Eligibility 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
Sites / Locations
- UCSF Benioff Children's Hospital
- Children's National
- Hôpital Robert Debré
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
CordIn
Arm Description
Transplantation of CordIn
Outcomes
Primary Outcome Measures
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.
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.
Secondary Outcome Measures
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.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02504619
Brief Title
Allogeneic SCT of CordIn™, in Patients With Hemoglobinopathies
Official Title
Allogeneic Stem Cell Transplantation of CordIn™, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients With Hemoglobinopathies
Study Type
Interventional
2. Study Status
Record Verification Date
June 2019
Overall Recruitment Status
Terminated
Why Stopped
lack of recruitment
Study Start Date
April 4, 2016 (Actual)
Primary Completion Date
August 2016 (Actual)
Study Completion Date
December 2017 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Gamida Cell ltd
4. Oversight
Data Monitoring Committee
Yes
5. 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™.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sickle Cell Disease, Thalassemia
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
1 (Actual)
8. Arms, Groups, and Interventions
Arm Title
CordIn
Arm Type
Experimental
Arm Description
Transplantation of CordIn
Intervention Type
Biological
Intervention Name(s)
CordIn
Intervention Description
CordIn™ is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells.
Primary Outcome Measure Information:
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
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
Secondary Outcome Measure Information:
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
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
25 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jean-Hugues Dalle, MD
Organizational Affiliation
Robert Debre, Paris, France
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Franco Locatelli, MD
Organizational Affiliation
IRCCS Ospedale Pediatrico Bambino Gesu, Rome, Italy
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Allistair Abraham, MD
Organizational Affiliation
Children's National, Washington DC
Official's Role
Principal Investigator
Facility Information:
Facility Name
UCSF Benioff Children's Hospital
City
Oakland
State/Province
California
ZIP/Postal Code
94609
Country
United States
Facility Name
Children's National
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20010
Country
United States
Facility Name
Hôpital Robert Debré
City
Paris
Country
France
12. IPD Sharing Statement
Learn more about this trial
Allogeneic SCT of CordIn™, in Patients With Hemoglobinopathies
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