Reduced Intensity Stem Cell Transplantation for Sickle Cell Anemia in Patients 2-30 Years Old

Locations

1 United States site

Age

2 to 30 Years

Genotypes

SS, SC

Phase

Phase 3

Study type

Interventional

Gender

All

Interventions

Drug

Procedure

Compensation

Unknown

About the study

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.

participation requirements

– 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

participation restrictions

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

Locations

  • Hackensack, New Jersey, United States, Hackensack University Medical Center, 07601