Trial Outcomes & Findings for Allogeneic SCT of CordIn™, in Patients With Hemoglobinopathies (NCT NCT02504619)
NCT ID: NCT02504619
Last Updated: 2019-06-26
Results Overview
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.
Recruitment status
TERMINATED
Study phase
PHASE1/PHASE2
Target enrollment
1 participants
Primary outcome timeframe
24 hours
Results posted on
2019-06-26
Participant Flow
Participant milestones
| Measure |
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.
|
|---|---|
|
Overall Study
STARTED
|
1
|
|
Overall Study
COMPLETED
|
1
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Allogeneic SCT of CordIn™, in Patients With Hemoglobinopathies
Baseline characteristics by cohort
| Measure |
CordIn
n=1 Participants
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.
|
|---|---|
|
Age, Categorical
<=18 years
|
1 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
|
Age, Continuous
|
2.8 Years
STANDARD_DEVIATION 0 • n=99 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
1 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
1 participants
n=99 Participants
|
PRIMARY outcome
Timeframe: 24 hoursThe 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.
Outcome measures
| Measure |
CordIn
n=1 Participants
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.
|
|---|---|
|
Number of Participants With no Acute Toxicity Associated With the Infusion of CordIn, Within 24 Hours Post-infusion.
|
1 Participants
|
PRIMARY outcome
Timeframe: 42 daysOne hundred percent of patients engrafted by day forty-two post-transplantation with donor-derived cells.
Outcome measures
| Measure |
CordIn
n=1 Participants
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.
|
|---|---|
|
The Percentage of Patients With Donor-derived Engraftment at 42 Days Following Transplantation
|
1 Participants
|
SECONDARY outcome
Timeframe: 365 daysThe percentage of patients alive at one year post-transplant was estimated using the Kaplan-Meier method.
Outcome measures
| Measure |
CordIn
n=1 Participants
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.
|
|---|---|
|
Percentage of Overall Survival at 365 Days After Transplantation
|
1 Participants
|
Adverse Events
CordIn
Serious events: 1 serious events
Other events: 1 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
CordIn
n=1 participants at risk
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.
|
|---|---|
|
Immune system disorders
GvHD
|
100.0%
1/1 • Number of events 2
|
Other adverse events
| Measure |
CordIn
n=1 participants at risk
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.
|
|---|---|
|
Gastrointestinal disorders
Hypoalbuminemia
|
100.0%
1/1 • Number of events 2
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place