Status:
COMPLETED
Stem Cell Transplantation for Sickle Cell Anemia
Lead Sponsor:
Hackensack Meridian Health
Conditions:
Sickle Cell Disease
Eligibility:
All Genders
2-30 years
Phase:
PHASE3
Brief Summary
This protocol will be investigating the use of stem cell transplantation, in related donors, to cure sickle cell disease. Sickle cell disease is a recessive disorder caused by a point mutation that re...
Detailed Description
Primary objective: 1\) To determine disease free survival (DFS) at two years after matched sibling transplant using bone marrow (BM) after a conditioning regimen consisting of distal timed Alemtuzuma...
Eligibility Criteria
Inclusion
- Inclusion Criteria:
- Patient Eligibility
- 1\) Matched sibling donors (9-10/10 marrow/PBSC or 5-6/6 UCB (single or double) with a total TNC dose of greater than 5 x 107/kg recipient weight)
- Age 2-30
- Hb SS, S-thal0, S-thal+, SC
- Evidence of ongoing hemolysis: Hb\<10, retic \>5%, LDH \> 500, TB\>2
- Karnofsky/Lansky score \>50
- LVSF\>26% or LVEF\>40%
- DLCO \>40% or O2 sat \>85% for those patients that can't perform PFTs
- GFR \>70 and serum creatinine \< 1.5 \* ULN for age
- ALT and AST \< 5 x ULN, direct bilirubin \<2 x ULN
- If the patient has been on chronic transfusion or has a ferritin \>1000, liver biopsy should be done and show no evidence of bridging fibrosis or cirrhosis
- Exclusion criteria
- Evidence of uncontrolled bacterial, viral, or fungal infection within one month prior to initiation of the conditioning regimen
- Pregnant or breastfeeding
- HIV positive
- Written informed consent not obtained
Exclusion
Key Trial Info
Start Date :
June 1 2011
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 1 2021
Estimated Enrollment :
30 Patients enrolled
Trial Details
Trial ID
NCT01877837
Start Date
June 1 2011
End Date
September 1 2021
Last Update
November 7 2022
Active Locations (1)
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1
Hackensack University Medical Center
Hackensack, New Jersey, United States, 07601