Trial Outcomes & Findings for CD34+ Stem Cell Infusion to Augment Graft Function (NCT NCT01856582)
NCT ID: NCT01856582
Last Updated: 2019-11-22
Results Overview
Successful augmentation of graft function achieved if donor chimerism is doubled compared to the value immediately pre-infusion at 12 months.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
23 participants
Primary outcome timeframe
12 months
Results posted on
2019-11-22
Participant Flow
Participant milestones
| Measure |
CD34+ Selected Stem Cell Infusion
A single infusion of selected CD34+ stem cells will be given without any preparative regimen. Stem cells will be depleted of T-cells using a CliniMACS CD34+ cell selection device and infused into the patient with a maximum T-cell dose of 5 x 10\^4/kg. The minimum number of CD34+ cells that will be infused is 1x10\^6/kg.
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|---|---|
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Overall Study
STARTED
|
23
|
|
Overall Study
COMPLETED
|
22
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
CD34+ Selected Stem Cell Infusion
A single infusion of selected CD34+ stem cells will be given without any preparative regimen. Stem cells will be depleted of T-cells using a CliniMACS CD34+ cell selection device and infused into the patient with a maximum T-cell dose of 5 x 10\^4/kg. The minimum number of CD34+ cells that will be infused is 1x10\^6/kg.
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|---|---|
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Overall Study
Died prior to Boost infusion
|
1
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Baseline Characteristics
CD34+ Stem Cell Infusion to Augment Graft Function
Baseline characteristics by cohort
| Measure |
CD34+ Selected Stem Cell Infusion
n=23 Participants
An infusion of selected CD34+ stem cells will be given without any preparative regimen.
CD34+: CD34+ cells are selected using the CliniMACS System; without preparative regimen
|
|---|---|
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Age, Categorical
<=18 years
|
16 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
7 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
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0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
18 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
18 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
23 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 12 monthsPopulation: Selected data from this study was combined with a clinical retrospective study for analysis and published.
Successful augmentation of graft function achieved if donor chimerism is doubled compared to the value immediately pre-infusion at 12 months.
Outcome measures
| Measure |
CD34+ Selected Stem Cell Infusion
n=22 Participants
An infusion of selected CD34+ stem cells will be given without any preparative regimen.
CD34+: CD34+ cells are selected using the CliniMACS System; without preparative regimen
|
|---|---|
|
Number of Participants Who Showed Successful Augmentation of Graft Function
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3 Participants
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Adverse Events
CD34+ Selected Stem Cell Infusion
Serious events: 8 serious events
Other events: 2 other events
Deaths: 3 deaths
Serious adverse events
| Measure |
CD34+ Selected Stem Cell Infusion
n=23 participants at risk
An infusion of selected CD34+ stem cells will be given without any preparative regimen.
CD34+: CD34+ cells are selected using the CliniMACS System; without preparative regimen
|
|---|---|
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Respiratory, thoracic and mediastinal disorders
Hypoxia
|
8.7%
2/23 • Number of events 3 • All AEs were collected for 30 days post stem cell infusion. Protocol-defined AEs related to study treatment were followed for three years.
|
|
Respiratory, thoracic and mediastinal disorders
Chronic Sinusitis
|
4.3%
1/23 • Number of events 1 • All AEs were collected for 30 days post stem cell infusion. Protocol-defined AEs related to study treatment were followed for three years.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Failure
|
4.3%
1/23 • Number of events 1 • All AEs were collected for 30 days post stem cell infusion. Protocol-defined AEs related to study treatment were followed for three years.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
4.3%
1/23 • Number of events 2 • All AEs were collected for 30 days post stem cell infusion. Protocol-defined AEs related to study treatment were followed for three years.
|
|
Renal and urinary disorders
Renal Failure
|
4.3%
1/23 • Number of events 1 • All AEs were collected for 30 days post stem cell infusion. Protocol-defined AEs related to study treatment were followed for three years.
|
|
Nervous system disorders
Encephalopathy
|
4.3%
1/23 • Number of events 1 • All AEs were collected for 30 days post stem cell infusion. Protocol-defined AEs related to study treatment were followed for three years.
|
|
Gastrointestinal disorders
Ascites
|
4.3%
1/23 • Number of events 1 • All AEs were collected for 30 days post stem cell infusion. Protocol-defined AEs related to study treatment were followed for three years.
|
|
Hepatobiliary disorders
Liver Failure
|
4.3%
1/23 • Number of events 1 • All AEs were collected for 30 days post stem cell infusion. Protocol-defined AEs related to study treatment were followed for three years.
|
|
Skin and subcutaneous tissue disorders
Cellulitis
|
4.3%
1/23 • Number of events 2 • All AEs were collected for 30 days post stem cell infusion. Protocol-defined AEs related to study treatment were followed for three years.
|
|
Cardiac disorders
Hypotension
|
8.7%
2/23 • Number of events 4 • All AEs were collected for 30 days post stem cell infusion. Protocol-defined AEs related to study treatment were followed for three years.
|
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General disorders
Fever
|
4.3%
1/23 • Number of events 1 • All AEs were collected for 30 days post stem cell infusion. Protocol-defined AEs related to study treatment were followed for three years.
|
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General disorders
Multiorgan Failure
|
8.7%
2/23 • Number of events 3 • All AEs were collected for 30 days post stem cell infusion. Protocol-defined AEs related to study treatment were followed for three years.
|
|
Infections and infestations
Infection
|
17.4%
4/23 • Number of events 4 • All AEs were collected for 30 days post stem cell infusion. Protocol-defined AEs related to study treatment were followed for three years.
|
Other adverse events
| Measure |
CD34+ Selected Stem Cell Infusion
n=23 participants at risk
An infusion of selected CD34+ stem cells will be given without any preparative regimen.
CD34+: CD34+ cells are selected using the CliniMACS System; without preparative regimen
|
|---|---|
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Cardiac disorders
Hemolysis
|
4.3%
1/23 • Number of events 1 • All AEs were collected for 30 days post stem cell infusion. Protocol-defined AEs related to study treatment were followed for three years.
|
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Injury, poisoning and procedural complications
Hemorrhage
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4.3%
1/23 • Number of events 1 • All AEs were collected for 30 days post stem cell infusion. Protocol-defined AEs related to study treatment were followed for three years.
|
|
Infections and infestations
Febrile Neutropenia
|
4.3%
1/23 • Number of events 1 • All AEs were collected for 30 days post stem cell infusion. Protocol-defined AEs related to study treatment were followed for three years.
|
Additional Information
Rebecca Marsh, MD/Associate Professor of Pediatrics
Cincinnati Children's Hospital Medical Center
Phone: 513-803-9063
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place