Trial Outcomes & Findings for Reduced Intensity Conditioning Using CD3+/CD19+ Depletion for Non Malignant Transplantable Diseases (NCT NCT02277639)
NCT ID: NCT02277639
Last Updated: 2018-04-11
Results Overview
The primary objective is to determine event free survival with durable stable engraftment of donor cells at one year.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
2 participants
Primary outcome timeframe
One Year
Results posted on
2018-04-11
Participant Flow
Participant milestones
| Measure |
Bone Marrow Failure Syndrome
Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete CD3+ CD19+ peripheral stem cells. Reduced intensity conditioning will include Busulfan, Fludarbine, Cyclophosphamide followed by stem cell infusion.
CD3+/CD19+ delpletion using CliniMACs device follwing reduced intensity conditioning: Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete CD3+ CD19+ peripheral stem cells.
|
Immunodeficiency / Dysregulation
Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete CD3+ CD19+ peripheral stem cells. Reduced intensity conditioning will include Busulfan, Fludarbine, Cyclophosphamide followed by stem cell infusion.
CD3+/CD19+ delpletion using CliniMACs device follwing reduced intensity conditioning: Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete CD3+ CD19+ peripheral stem cells.
|
|---|---|---|
|
Overall Study
STARTED
|
0
|
2
|
|
Overall Study
COMPLETED
|
0
|
2
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Reduced Intensity Conditioning Using CD3+/CD19+ Depletion for Non Malignant Transplantable Diseases
Baseline characteristics by cohort
| Measure |
Bone Marrow Failure Syndrome
Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete CD3+ CD19+ peripheral stem cells. Reduced intensity conditioning will include Busulfan, Fludarbine, Cyclophosphamide followed by stem cell infusion.
CD3+/CD19+ delpletion using CliniMACs device follwing reduced intensity conditioning: Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete CD3+ CD19+ peripheral stem cells.
|
Immunodeficiency / Dysregulation
n=2 Participants
Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete CD3+ CD19+ peripheral stem cells. Reduced intensity conditioning will include Busulfan, Fludarbine, Cyclophosphamide followed by stem cell infusion.
CD3+/CD19+ delpletion using CliniMACs device follwing reduced intensity conditioning: Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete CD3+ CD19+ peripheral stem cells.
|
Total
n=2 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
—
|
9.5 years
n=7 Participants
|
9.5 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
—
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
—
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: One YearThe primary objective is to determine event free survival with durable stable engraftment of donor cells at one year.
Outcome measures
| Measure |
Bone Marrow Failure Syndrome
Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete CD3+ CD19+ peripheral stem cells. Reduced intensity conditioning will include Busulfan, Fludarbine, Cyclophosphamide followed by stem cell infusion.
CD3+/CD19+ delpletion using CliniMACs device follwing reduced intensity conditioning: Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete CD3+ CD19+ peripheral stem cells.
|
Immunodeficiency / Dysregulation
n=2 Participants
Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete CD3+ CD19+ peripheral stem cells. Reduced intensity conditioning will include Busulfan, Fludarbine, Cyclophosphamide followed by stem cell infusion.
CD3+/CD19+ delpletion using CliniMACs device follwing reduced intensity conditioning: Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete CD3+ CD19+ peripheral stem cells.
|
|---|---|---|
|
Number of Participants With Engraftment
|
—
|
2 Participants
|
Adverse Events
Bone Marrow Failure Syndrome
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Immunodeficiency / Dysregulation
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
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place