Trial Outcomes & Findings for SCD-Haplo: Phase II Study of HLA-Haploidentical SCT for Aggressive SCD (NCT NCT02013375)
NCT ID: NCT02013375
Last Updated: 2019-08-28
Results Overview
To determine the engraftment at Day +60 following HLA-haploidentical hematopoietic stem cell transplant protocol using immunosuppressive agents and low-dose total body irradiation (TBI) for conditioning and post-transplant cyclophosphamide in patients with sickle cell disease.
TERMINATED
PHASE2
2 participants
Up to Day 60 post-transplant.
2019-08-28
Participant Flow
Subjects are referred by their primary care physicians to the UIC Sickle Cell clinic for specialty care. Subjects are evaluated to see if they are eligible for studies and if they are, the subject is presented with the option of enrolling into a research study or participating in alternative therapies that have also been presented to the subject.
Participant milestones
| Measure |
Haploidentical Transplant
All subjects will undergo pre-conditioning treatment with alemtuzumab (0.3 mg/kg) and total body irradiation (300cGy), followed by stem cell transplant, and post-transplant with cyclophosphamide (50mg/kg/day) and sirolimus (target trough level of 10-15ng/mL).
|
|---|---|
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Overall Study
STARTED
|
2
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
2
|
Reasons for withdrawal
| Measure |
Haploidentical Transplant
All subjects will undergo pre-conditioning treatment with alemtuzumab (0.3 mg/kg) and total body irradiation (300cGy), followed by stem cell transplant, and post-transplant with cyclophosphamide (50mg/kg/day) and sirolimus (target trough level of 10-15ng/mL).
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|---|---|
|
Overall Study
Did not engraft
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1
|
Baseline Characteristics
SCD-Haplo: Phase II Study of HLA-Haploidentical SCT for Aggressive SCD
Baseline characteristics by cohort
| Measure |
Haploidentical Transplant
n=2 Participants
All subjects will undergo pre-conditioning treatment with alemtuzumab (0.3 mg/kg Day -5, Day -4, Day -3) and total body irradiation (300cGy), followed by stem cell transplant, and post-transplant treatment with cyclophosphamide (50mg/kg/day) and sirolimus (target trough level of 10-15ng/mL).
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|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
2 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
38 Years
STANDARD_DEVIATION 1 • n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
|
2 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
2 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to Day 60 post-transplant.To determine the engraftment at Day +60 following HLA-haploidentical hematopoietic stem cell transplant protocol using immunosuppressive agents and low-dose total body irradiation (TBI) for conditioning and post-transplant cyclophosphamide in patients with sickle cell disease.
Outcome measures
| Measure |
Haploidentical Transplant
n=2 Participants
All subjects will undergo pre-conditioning treatment with alemtuzumab (0.3 mg/kg Day -5, Day -4, Day -3) and total body irradiation (300cGy), followed by stem cell transplant, and post-transplant treatment with cyclophosphamide (50mg/kg/day) and sirolimus (target trough level of 10-15ng/mL).
|
|---|---|
|
Engraftment Rate
|
0 Participants
|
SECONDARY outcome
Timeframe: Up to one year post-transplantPopulation: No subjects engrafted
To assess the frequency of acute and chronic complications of sickle cell disease during and after HLA-haploidentical hematopoietic stem cell transplantation with this protocol. The acute complications include vaso-occlusive pain episodes, acute chest syndrome, stroke, and priapism. The chronic complications include nephropathy, retinopathy, osteonecrosis, pulmonary artery pressures, cardiomyopathy, and chronic lung disease.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to one year post-transplant.Population: No subjects engrafted
To determine the overall and disease-free survival of patients with sickle cell disease receiving HLA-haploidentical hematopoietic stem cell transplantation with this protocol.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to one year post-transplant.Population: 0 subjects engrafted
To determine the incidence of acute and chronic graft-versus-host disease, the incidence of infectious complications, and the transplant related mortality in sickle cell disease patients after HLA-haploidentical hematopoietic stem cell transplantation with this protocol.
Outcome measures
Outcome data not reported
Adverse Events
Haploidentical Transplant
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