Stem Cell Transplantation for Sickle Cell Anemia

• Status: Active, not recruiting
• Trial end date: 2022-11-01
• Target enrollment: 0
• Participant gender: All

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 results in the substitution of valine for glutamic acid at the sixth position in the B-chain of hemoglobin. This leads to sickling of the red blood cells under many conditions, such as hypoxia, dehydration, and hyperthermia. The sickling leads to vaso-occlusion, which causes irreversible damage in almost all systems in the body, including the central nervous system (CNS), lungs, heart, bones, eyes, liver, and kidneys.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Hackensack Meridian Health; Hackensack University Medical Center
• Treatments: Alemtuzumab; Fludarabine; Fludarabine phosphate; Melphalan; Vidarabine

Criteria

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)

1. Age 2-30

2. Hb SS, S-thal0, S-thal+, SC

3. Evidence of ongoing hemolysis: Hb<10, retic >5%, LDH > 500, TB>2

4. Karnofsky/Lansky score >50

5. LVSF>26% or LVEF>40%

6. DLCO >40% or O2 sat >85% for those patients that can't perform PFTs

7. GFR >70 and serum creatinine < 1.5 * ULN for age

8. ALT and AST < 5 x ULN, direct bilirubin <2 x ULN

9. 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

1. Evidence of uncontrolled bacterial, viral, or fungal infection within one month prior to initiation of the conditioning regimen

2. Pregnant or breastfeeding

3. HIV positive

4. Written informed consent not obtained