Trial Outcomes & Findings for Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs (NCT NCT00957931)
NCT ID: NCT00957931
Last Updated: 2018-08-06
Results Overview
Stable engraftment was defined as absolute neutrophil count (ANC) \>500 cells /µL for 3 consecutive days and platelet count \>50,000 for one week without transfusion; subsequently stable engraftment was measured by percentage of donor cells.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
6 participants
Primary outcome timeframe
Up to 1 year
Results posted on
2018-08-06
Participant Flow
Participant milestones
| Measure |
Mesenchymal Stromal Cells
Patients received bone marrow transplantation using matched unrelated donors, reduced intensity conditioning regimen, and co-transplanting mesenchymal stromal cells derived from parental bone marrow.
|
|---|---|
|
Overall Study
STARTED
|
6
|
|
Overall Study
COMPLETED
|
2
|
|
Overall Study
NOT COMPLETED
|
4
|
Reasons for withdrawal
| Measure |
Mesenchymal Stromal Cells
Patients received bone marrow transplantation using matched unrelated donors, reduced intensity conditioning regimen, and co-transplanting mesenchymal stromal cells derived from parental bone marrow.
|
|---|---|
|
Overall Study
Death
|
4
|
Baseline Characteristics
Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs
Baseline characteristics by cohort
| Measure |
Mesenchymal Stromal Cells
n=6 Participants
Patients received bone marrow transplantation using matched unrelated donors, reduced intensity conditioning regimen, and co-transplanting mesenchymal stromal cells derived from parental bone marrow.
|
|---|---|
|
Age, Continuous
|
10 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=5 Participants
|
|
Number of known transfusions prior to HSCT
|
47.5 transfusions
n=5 Participants
|
|
Diagnosis
Sickle cell disease
|
4 Participants
n=5 Participants
|
|
Diagnosis
Thalassemia major
|
2 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to 1 yearStable engraftment was defined as absolute neutrophil count (ANC) \>500 cells /µL for 3 consecutive days and platelet count \>50,000 for one week without transfusion; subsequently stable engraftment was measured by percentage of donor cells.
Outcome measures
| Measure |
Mesenchymal Stromal Cells
n=6 Participants
Patients received bone marrow transplantation using matched unrelated donors, reduced intensity conditioning regimen, and co-transplanting mesenchymal stromal cells derived from parental bone marrow.
|
|---|---|
|
Count of Participants With Stable Engraftment Post Hematopoietic Cell Transplantation (HCT)
|
3 Participants
|
SECONDARY outcome
Timeframe: 6 monthsOverall survival is reported at the count of participants alive 6 months following HCT.
Outcome measures
| Measure |
Mesenchymal Stromal Cells
n=6 Participants
Patients received bone marrow transplantation using matched unrelated donors, reduced intensity conditioning regimen, and co-transplanting mesenchymal stromal cells derived from parental bone marrow.
|
|---|---|
|
Overall Survival 6 Months Following HCT
|
2 Participants
|
SECONDARY outcome
Timeframe: 1 yearOverall survival is reported at the count of participants alive 1 year following HCT.
Outcome measures
| Measure |
Mesenchymal Stromal Cells
n=6 Participants
Patients received bone marrow transplantation using matched unrelated donors, reduced intensity conditioning regimen, and co-transplanting mesenchymal stromal cells derived from parental bone marrow.
|
|---|---|
|
Overall Survival 1 Year Following HCT
|
2 Participants
|
SECONDARY outcome
Timeframe: 6 monthsDisease-free survival is defined as alive without underlying disease.
Outcome measures
| Measure |
Mesenchymal Stromal Cells
n=6 Participants
Patients received bone marrow transplantation using matched unrelated donors, reduced intensity conditioning regimen, and co-transplanting mesenchymal stromal cells derived from parental bone marrow.
|
|---|---|
|
Count of Participants With Disease-free Survival 6 Months Following HCT
|
0 Participants
|
SECONDARY outcome
Timeframe: 1 yearDisease-free survival is defined as alive without underlying disease.
Outcome measures
| Measure |
Mesenchymal Stromal Cells
n=6 Participants
Patients received bone marrow transplantation using matched unrelated donors, reduced intensity conditioning regimen, and co-transplanting mesenchymal stromal cells derived from parental bone marrow.
|
|---|---|
|
Count of Participants With Disease-free Survival 1 Year Following HCT
|
0 Participants
|
Adverse Events
Mesenchymal Stromal Cells
Serious events: 6 serious events
Other events: 0 other events
Deaths: 4 deaths
Serious adverse events
| Measure |
Mesenchymal Stromal Cells
n=6 participants at risk
Patients received bone marrow transplantation using matched unrelated donors, reduced intensity conditioning regimen, and co-transplanting mesenchymal stromal cells derived from parental bone marrow.
|
|---|---|
|
Blood and lymphatic system disorders
Graft failure with autologous recovery
|
33.3%
2/6 • 1 year
|
|
Hepatobiliary disorders
SOS
|
16.7%
1/6 • 1 year
|
|
Immune system disorders
EBV-PTLD
|
16.7%
1/6 • 1 year
|
|
Immune system disorders
Grade II GVHD
|
16.7%
1/6 • 1 year
|
|
Immune system disorders
Grade III acute GVHD
|
16.7%
1/6 • 1 year
|
|
Infections and infestations
Adenovirus reactivation
|
16.7%
1/6 • 1 year
|
|
Infections and infestations
BK virus reactivation
|
16.7%
1/6 • 1 year
|
|
Infections and infestations
CMV pneumonitis
|
16.7%
1/6 • 1 year
|
|
Infections and infestations
CMV reactivation
|
16.7%
1/6 • 1 year
|
|
Infections and infestations
CMV viremia
|
33.3%
2/6 • 1 year
|
|
Infections and infestations
Disseminated toxoplasmosis
|
16.7%
1/6 • 1 year
|
|
Infections and infestations
Gram-negative septic shock
|
16.7%
1/6 • 1 year
|
|
Infections and infestations
HHV-6 viremia
|
16.7%
1/6 • 1 year
|
|
Infections and infestations
Klebsiella and enterobacter
|
16.7%
1/6 • 1 year
|
|
Infections and infestations
Klebsiella bacteremia
|
16.7%
1/6 • 1 year
|
|
Nervous system disorders
Intracranial bleeding
|
16.7%
1/6 • 1 year
|
|
Nervous system disorders
Intracranial hemorrhage
|
16.7%
1/6 • 1 year
|
|
Nervous system disorders
PRES
|
16.7%
1/6 • 1 year
|
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place