Status:
COMPLETED
Haploidentical Stem Cell Transplant for Patients With Sickle Cell Disease and Prior Stroke or Abnormal Transcranial Ultrasound
Lead Sponsor:
St. Jude Children's Research Hospital
Conditions:
Sickle Cell Disease
Eligibility:
All Genders
2-16 years
Phase:
PHASE1
Brief Summary
Sickle cell disease is a life-long blood condition that can cause damage to the brain and other organs of the body. Children may develop severe, debilitating clinical states, with stroke or abnormal b...
Detailed Description
Secondary objectives for this protocol include the following: * To estimate 1-year overall and event free survival after transplantation. An event is defined as toxicity (graft failure, death, grade ...
Eligibility Criteria
Inclusion
- Inclusion Criteria:
- Hemoglobin SS or S-Beta Thalassemia Sickle Cell Disease.
- Partially-matched family member with hemoglobin AA (normal) or hemoglobin AS (sickle trait) phenotype.
- Stroke (persistent neurologic deficit lasting \> 24 hours and present on MRI) or abnormal transcranial Doppler (TCD) ultrasonography requiring chronic transfusion therapy. A TCD is deemed abnormal when the velocity is greater than or equal to 200 cm/sec. Chronic transfusion therapy is defined as "packed red blood cell transfusions administered approximately every 3-5 weeks to decrease the percentage of sickle hemoglobin (Hemoglobin S) to prevent complications of sickle cell disease. This is used most commonly to treat/prevent stroke, acute chest syndrome, and/or pain crises.
- Exclusion criteria
- Karnofsky or Lansky score \< 60%
- Acute hepatitis or evidence of moderate or severe portal fibrosis on biopsy. (Biopsy will be obtained if patient on chronic transfusion therapy \> 6 months or ferritin \> 1000 ng/ml) International normalized ratio (INR) less than 2 times normal. ALT and AST less than 3 times the upper limit of normal.
- Severe renal impairment (as evidenced by GFR \< 30% predicted normal)
- Ejection fraction or shortening fraction below lower limit of normal for age.
- Pregnancy
- Lactating and pregnant females are excluded
- Positive HLA crossmatch with donor.
- No sickle cell chronic lung disease \> Stage 2
Exclusion
Key Trial Info
Start Date :
April 1 2005
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 1 2009
Estimated Enrollment :
5 Patients enrolled
Trial Details
Trial ID
NCT00152113
Start Date
April 1 2005
End Date
January 1 2009
Last Update
April 26 2017
Active Locations (1)
Enter a location and click search to find clinical trials sorted by distance.
1
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105