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Hematopoietic Stem Cell Transplantation for Patients With Sickle Cell Disease and Prior Stroke or Abnormal Transcranial Doppler Ultrasound Using Reduced-Intensity Conditioning and T-Cell Depleted HSC From Partially Matched Family Donors


Phase 1
2 Years
16 Years
Not Enrolling
Both
Sickle Cell Disease

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Trial Information

Hematopoietic Stem Cell Transplantation for Patients With Sickle Cell Disease and Prior Stroke or Abnormal Transcranial Doppler Ultrasound Using Reduced-Intensity Conditioning and T-Cell Depleted HSC From Partially Matched Family Donors


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 III/IV acute GHVD), or a
sickle-related event (stroke, acute chest syndrome, pain crisis).

- To obtain preliminary information regarding donor engraftment among different cell
subsets, including unsorted mononuclear cell, and lymphoid fractions during the first
year after transplant.

- To observe the rate of acute and chronic GVHD during the first year after transplant.

- To assess the proportion of research participants who experience poor graft integrity
and therefore require additional donor stem cells or lymphocytes.

- To document the effect of stem cell transplant on the central nervous system as defined
by radiological imaging and neuropsychological testing.

- To investigate immune reconstitution after transplantation


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

Type of Study:

Interventional

Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

To assess the safety of haploidentical stem cell transplantation for children and adolescents with severe sickle cell disease and stroke or abnormal transcranial Doppler ultrasound requiring chronic transfusion therapy.

Outcome Time Frame:

September 2007

Safety Issue:

Yes

Principal Investigator

Wing Leung, M.D.

Investigator Role:

Principal Investigator

Investigator Affiliation:

St. Jude Children's Research Hospital

Authority:

United States: Food and Drug Administration

Study ID:

SCDHAP

NCT ID:

NCT00152113

Start Date:

April 2005

Completion Date:

January 2009

Related Keywords:

  • Sickle Cell Disease
  • Sickle Cell Anemia
  • Stem Cell Transplantation
  • Haploidentical stem cell transplant
  • T cell depletion
  • CliniMACS device
  • Anemia, Sickle Cell

Name

Location
St. Jude Children's Research Hospital Memphis, Tennessee  38105-2794