Trial Outcomes & Findings for Red Cell Half Life Determination in Patients With and Without Sickle Cell Disease (NCT NCT04476277)
NCT ID: NCT04476277
Last Updated: 2024-02-13
Results Overview
Mean Days of Red Blood Cells (RBC) survival in participants with Sickle Cell Disease (SCD), participants with SCD who have undergone stem cell transplant, participants with Sickle Cell Trait, and healthy volunteers. Peripheral blood samples were analyzed by flow cytometry until biotin was not detectable on RBC.
Recruitment status
COMPLETED
Study phase
EARLY_PHASE1
Target enrollment
22 participants
Primary outcome timeframe
Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22.
Results posted on
2024-02-13
Participant Flow
Participant milestones
| Measure |
Sickle Cell Disease Pre-Transplantation
Autologous cells will be collected in participants with Sickle Cell Disease Pre-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Disease Post-Transplantation
Autologous cells will be collected in participants with Sickle Cell Disease Post-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Trait (HbAS)
Autologous cells will be collected in participants with Sickle Cell Trait (HbAS) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
HbAA (Healthy Volunteers)
Autologous cells will be collected in participants with HbAA (Healthy volunteers) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
6
|
6
|
7
|
3
|
|
Overall Study
COMPLETED
|
6
|
6
|
6
|
3
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
1
|
0
|
Reasons for withdrawal
| Measure |
Sickle Cell Disease Pre-Transplantation
Autologous cells will be collected in participants with Sickle Cell Disease Pre-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Disease Post-Transplantation
Autologous cells will be collected in participants with Sickle Cell Disease Post-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Trait (HbAS)
Autologous cells will be collected in participants with Sickle Cell Trait (HbAS) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
HbAA (Healthy Volunteers)
Autologous cells will be collected in participants with HbAA (Healthy volunteers) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
|---|---|---|---|---|
|
Overall Study
Withdrawal by Subject
|
0
|
0
|
1
|
0
|
Baseline Characteristics
Red Cell Half Life Determination in Patients With and Without Sickle Cell Disease
Baseline characteristics by cohort
| Measure |
Sickle Cell Disease Pre-Transplantation
n=6 Participants
Autologous cells will be collected in participants with Sickle Cell Disease Pre-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Disease Post-Transplantation
n=6 Participants
Autologous cells will be collected in participants with Sickle Cell Disease Post-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Trait (HbAS)
n=7 Participants
Autologous cells will be collected in participants with Sickle Cell Trait (HbAS) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
HbAA (Healthy Volunteers)
n=3 Participants
Autologous cells will be collected in participants with HbAA (Healthy volunteers) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Total
n=22 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
6 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
3 Participants
n=4 Participants
|
22 Participants
n=21 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
14 Participants
n=21 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
8 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
6 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
3 Participants
n=4 Participants
|
22 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Black or African American
|
6 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
3 Participants
n=4 Participants
|
22 Participants
n=21 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Region of Enrollment
United States
|
6 participants
n=5 Participants
|
6 participants
n=7 Participants
|
7 participants
n=5 Participants
|
3 participants
n=4 Participants
|
22 participants
n=21 Participants
|
PRIMARY outcome
Timeframe: Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22.Population: Analysis includes participants that have completed study and had no exchange or simple red blood cell transfusions.
Mean Days of Red Blood Cells (RBC) survival in participants with Sickle Cell Disease (SCD), participants with SCD who have undergone stem cell transplant, participants with Sickle Cell Trait, and healthy volunteers. Peripheral blood samples were analyzed by flow cytometry until biotin was not detectable on RBC.
Outcome measures
| Measure |
Sickle Cell Disease Pre-Transplantation
n=6 Participants
Autologous cells will be collected in participants with Sickle Cell Disease Pre-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Disease Post-Transplantation
n=5 Participants
Autologous cells will be collected in participants with Sickle Cell Disease Post-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Trait (HbAS)
n=6 Participants
Autologous cells will be collected in participants with Sickle Cell Trait (HbAS) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
HbAA (Healthy Volunteers)
n=3 Participants
Autologous cells will be collected in participants with HbAA (Healthy volunteers) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
|---|---|---|---|---|
|
Mean Red Blood Cells Lifespan in Participants
|
64.1 day
Interval 35.0 to 91.0
|
113.4 day
Interval 105.0 to 119.0
|
126.0 day
Interval 119.0 to 147.0
|
123.7 day
Interval 91.0 to 147.0
|
SECONDARY outcome
Timeframe: Baseline, 3 months, 6 monthsPopulation: Analysis includes participants that have completed study and had no exchange or simple red blood cell transfusions.
Number of participants with Antibody detection to biotin
Outcome measures
| Measure |
Sickle Cell Disease Pre-Transplantation
n=6 Participants
Autologous cells will be collected in participants with Sickle Cell Disease Pre-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Disease Post-Transplantation
n=5 Participants
Autologous cells will be collected in participants with Sickle Cell Disease Post-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Trait (HbAS)
n=6 Participants
Autologous cells will be collected in participants with Sickle Cell Trait (HbAS) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
HbAA (Healthy Volunteers)
n=3 Participants
Autologous cells will be collected in participants with HbAA (Healthy volunteers) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
|---|---|---|---|---|
|
Number of Participants With Antibody Detection
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22.Population: Pre-specified in the protocol to only assess this Outcome Measure in the Sickle Cell Disease Arms/Groups.
Mean White Blood Cell (WBC) count between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation
Outcome measures
| Measure |
Sickle Cell Disease Pre-Transplantation
n=6 Participants
Autologous cells will be collected in participants with Sickle Cell Disease Pre-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Disease Post-Transplantation
n=5 Participants
Autologous cells will be collected in participants with Sickle Cell Disease Post-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Trait (HbAS)
Autologous cells will be collected in participants with Sickle Cell Trait (HbAS) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
HbAA (Healthy Volunteers)
Autologous cells will be collected in participants with HbAA (Healthy volunteers) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
|---|---|---|---|---|
|
Mean White Blood Cell Count
|
9.0 K/mcL
Standard Deviation 4.5
|
6.5 K/mcL
Standard Deviation 2.4
|
—
|
—
|
SECONDARY outcome
Timeframe: Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22.Population: Pre-specified in the protocol to only assess this Outcome Measure in the Sickle Cell Disease Arms/Groups.
Mean Red Blood Cell (RBC) Count between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation
Outcome measures
| Measure |
Sickle Cell Disease Pre-Transplantation
n=6 Participants
Autologous cells will be collected in participants with Sickle Cell Disease Pre-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Disease Post-Transplantation
n=5 Participants
Autologous cells will be collected in participants with Sickle Cell Disease Post-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Trait (HbAS)
Autologous cells will be collected in participants with Sickle Cell Trait (HbAS) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
HbAA (Healthy Volunteers)
Autologous cells will be collected in participants with HbAA (Healthy volunteers) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
|---|---|---|---|---|
|
Mean Red Blood Cell Count
|
3.0 M/mcL
Standard Deviation 1.0
|
4.3 M/mcL
Standard Deviation 0.8
|
—
|
—
|
SECONDARY outcome
Timeframe: Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22.Population: Pre-specified in the protocol to only assess this Outcome Measure in the Sickle Cell Disease Arms/Groups.
Mean Hemoglobin (Hb) Value between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation
Outcome measures
| Measure |
Sickle Cell Disease Pre-Transplantation
n=6 Participants
Autologous cells will be collected in participants with Sickle Cell Disease Pre-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Disease Post-Transplantation
n=5 Participants
Autologous cells will be collected in participants with Sickle Cell Disease Post-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Trait (HbAS)
Autologous cells will be collected in participants with Sickle Cell Trait (HbAS) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
HbAA (Healthy Volunteers)
Autologous cells will be collected in participants with HbAA (Healthy volunteers) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
|---|---|---|---|---|
|
Mean Hemoglobin Value
|
8.8 g/dL
Standard Deviation 2.1
|
13.0 g/dL
Standard Deviation 2.2
|
—
|
—
|
SECONDARY outcome
Timeframe: Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22.Population: Pre-specified in the protocol to only assess this Outcome Measure in the Sickle Cell Disease Arms/Groups.
Mean Hematocrit (Hct) value between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation
Outcome measures
| Measure |
Sickle Cell Disease Pre-Transplantation
n=6 Participants
Autologous cells will be collected in participants with Sickle Cell Disease Pre-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Disease Post-Transplantation
n=5 Participants
Autologous cells will be collected in participants with Sickle Cell Disease Post-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Trait (HbAS)
Autologous cells will be collected in participants with Sickle Cell Trait (HbAS) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
HbAA (Healthy Volunteers)
Autologous cells will be collected in participants with HbAA (Healthy volunteers) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
|---|---|---|---|---|
|
Mean Hematocrit Value
|
25.0 % of Hematocrit
Standard Deviation 6.3
|
38.3 % of Hematocrit
Standard Deviation 5.8
|
—
|
—
|
SECONDARY outcome
Timeframe: Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22.Population: Pre-specified in the protocol to only assess this Outcome Measure in the Sickle Cell Disease Arms/Groups.
Mean Value of Mean Corpuscular Volume (MCV) between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation
Outcome measures
| Measure |
Sickle Cell Disease Pre-Transplantation
n=6 Participants
Autologous cells will be collected in participants with Sickle Cell Disease Pre-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Disease Post-Transplantation
n=5 Participants
Autologous cells will be collected in participants with Sickle Cell Disease Post-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Trait (HbAS)
Autologous cells will be collected in participants with Sickle Cell Trait (HbAS) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
HbAA (Healthy Volunteers)
Autologous cells will be collected in participants with HbAA (Healthy volunteers) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
|---|---|---|---|---|
|
Mean Value of Mean Corpuscular Volume
|
86.7 fL
Standard Deviation 12.8
|
89.4 fL
Standard Deviation 2.3
|
—
|
—
|
SECONDARY outcome
Timeframe: Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22.Population: Pre-specified in the protocol to only assess this Outcome Measure in the Sickle Cell Disease Arms/Groups.
Mean Absolute Reticulocyte Count (ARC) between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation
Outcome measures
| Measure |
Sickle Cell Disease Pre-Transplantation
n=6 Participants
Autologous cells will be collected in participants with Sickle Cell Disease Pre-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Disease Post-Transplantation
n=5 Participants
Autologous cells will be collected in participants with Sickle Cell Disease Post-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Trait (HbAS)
Autologous cells will be collected in participants with Sickle Cell Trait (HbAS) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
HbAA (Healthy Volunteers)
Autologous cells will be collected in participants with HbAA (Healthy volunteers) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
|---|---|---|---|---|
|
Mean Absolute Reticulocyte Count
|
192.7 K/mcL
Standard Deviation 84.8
|
152.9 K/mcL
Standard Deviation 96.4
|
—
|
—
|
SECONDARY outcome
Timeframe: Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22.Population: Pre-specified in the protocol to only assess this Outcome Measure in the Sickle Cell Disease Arms/Groups.
Mean Aspartate Aminotransferase (AST) value between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation
Outcome measures
| Measure |
Sickle Cell Disease Pre-Transplantation
n=6 Participants
Autologous cells will be collected in participants with Sickle Cell Disease Pre-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Disease Post-Transplantation
n=5 Participants
Autologous cells will be collected in participants with Sickle Cell Disease Post-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Trait (HbAS)
Autologous cells will be collected in participants with Sickle Cell Trait (HbAS) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
HbAA (Healthy Volunteers)
Autologous cells will be collected in participants with HbAA (Healthy volunteers) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
|---|---|---|---|---|
|
Mean Aspartate Aminotransferase Value
|
26.1 IU/L
Standard Deviation 12.7
|
26.1 IU/L
Standard Deviation 5.5
|
—
|
—
|
SECONDARY outcome
Timeframe: Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22.Population: Pre-specified in the protocol to only assess this Outcome Measure in the Sickle Cell Disease Arms/Groups.
Mean Total Bilirubin value between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation
Outcome measures
| Measure |
Sickle Cell Disease Pre-Transplantation
n=6 Participants
Autologous cells will be collected in participants with Sickle Cell Disease Pre-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Disease Post-Transplantation
n=5 Participants
Autologous cells will be collected in participants with Sickle Cell Disease Post-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Trait (HbAS)
Autologous cells will be collected in participants with Sickle Cell Trait (HbAS) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
HbAA (Healthy Volunteers)
Autologous cells will be collected in participants with HbAA (Healthy volunteers) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
|---|---|---|---|---|
|
Mean Total Bilirubin Value
|
7.1 mg/dL
Standard Deviation 1.0
|
1.1 mg/dL
Standard Deviation 1.7
|
—
|
—
|
SECONDARY outcome
Timeframe: Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22.Population: Pre-specified in the protocol to only assess this Outcome Measure in the Sickle Cell Disease Arms/Groups.
Mean Lactate Dehydrogenase (LDH) value between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation
Outcome measures
| Measure |
Sickle Cell Disease Pre-Transplantation
n=6 Participants
Autologous cells will be collected in participants with Sickle Cell Disease Pre-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Disease Post-Transplantation
n=5 Participants
Autologous cells will be collected in participants with Sickle Cell Disease Post-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Trait (HbAS)
Autologous cells will be collected in participants with Sickle Cell Trait (HbAS) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
HbAA (Healthy Volunteers)
Autologous cells will be collected in participants with HbAA (Healthy volunteers) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
|---|---|---|---|---|
|
Mean Lactate Dehydrogenase Value
|
323.9 unit/L
Standard Deviation 254.1
|
253.7 unit/L
Standard Deviation 323.9
|
—
|
—
|
SECONDARY outcome
Timeframe: Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22.Population: Pre-specified in the protocol to only assess this Outcome Measure in the Sickle Cell Disease Arms/Groups.
Mean Adult Hemoglobin (HbA) percentage between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation
Outcome measures
| Measure |
Sickle Cell Disease Pre-Transplantation
n=6 Participants
Autologous cells will be collected in participants with Sickle Cell Disease Pre-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Disease Post-Transplantation
n=5 Participants
Autologous cells will be collected in participants with Sickle Cell Disease Post-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Trait (HbAS)
Autologous cells will be collected in participants with Sickle Cell Trait (HbAS) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
HbAA (Healthy Volunteers)
Autologous cells will be collected in participants with HbAA (Healthy volunteers) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
|---|---|---|---|---|
|
Mean Adult Hemoglobin Percentage
|
2.8 % of Adult Hemoglobin (HbA)
Standard Deviation 6.9
|
59.6 % of Adult Hemoglobin (HbA)
Standard Deviation 8.2
|
—
|
—
|
SECONDARY outcome
Timeframe: Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22.Population: Pre-specified in the protocol to only assess this Outcome Measure in the Sickle Cell Disease Arms/Groups.
Mean Sickle Hemoglobin (HbS) Percentage between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation
Outcome measures
| Measure |
Sickle Cell Disease Pre-Transplantation
n=6 Participants
Autologous cells will be collected in participants with Sickle Cell Disease Pre-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Disease Post-Transplantation
n=5 Participants
Autologous cells will be collected in participants with Sickle Cell Disease Post-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Trait (HbAS)
Autologous cells will be collected in participants with Sickle Cell Trait (HbAS) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
HbAA (Healthy Volunteers)
Autologous cells will be collected in participants with HbAA (Healthy volunteers) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
|---|---|---|---|---|
|
Mean Sickle Hemoglobin Percentage
|
68.7 % of Sickle Hemoglobin (HbS)
Standard Deviation 17.1
|
36.7 % of Sickle Hemoglobin (HbS)
Standard Deviation 7.5
|
—
|
—
|
SECONDARY outcome
Timeframe: Sickle Cell Disease Pre-Transplantation cohort time frame is as follows: baseline, twice weekly up to week 3 then weekly up to week 22. All other cohorts, time frame is as follows: baseline, weekly up to week 4, then every other week up to week 22.Population: Pre-specified in the protocol to only assess this Outcome Measure in the Sickle Cell Disease Arms/Groups.
Mean Fetal Hemoglobin (HbF) Percentage between Sickle Cell Disease Pre-transplantation and Sickle Cell Disease Post-Transplantation
Outcome measures
| Measure |
Sickle Cell Disease Pre-Transplantation
n=6 Participants
Autologous cells will be collected in participants with Sickle Cell Disease Pre-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Disease Post-Transplantation
n=5 Participants
Autologous cells will be collected in participants with Sickle Cell Disease Post-Transplantation and biotin-labeled ex vivo and reinfused to measure red cell survival
|
Sickle Cell Trait (HbAS)
Autologous cells will be collected in participants with Sickle Cell Trait (HbAS) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
HbAA (Healthy Volunteers)
Autologous cells will be collected in participants with HbAA (Healthy volunteers) and biotin-labeled ex vivo and reinfused to measure red cell survival
|
|---|---|---|---|---|
|
Mean Fetal Hemoglobin Percentage
|
10.4 % of Fetal Hemoglobin
Standard Deviation 9.1
|
.5 % of Fetal Hemoglobin
Standard Deviation .8
|
—
|
—
|
Adverse Events
Sickle Cell Disease Pre-Transplantation
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Sickle Cell Disease Post-Transplantation
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Sickle Cell Trait (HbAS)
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
HbAA (Healthy Volunteers)
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Mathew Hsieh, M.D.
National Heart, Lung, and Blood Institute (NHLBI)
Phone: 301.402.7687
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place