Status:
ACTIVE_NOT_RECRUITING
Haplo T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease
Lead Sponsor:
New York Medical College
Collaborating Sponsors:
UCSF Benioff Children's Hospital Oakland
Medical College of Wisconsin
Conditions:
Sickle Cell Disease
Eligibility:
All Genders
2-20 years
Phase:
PHASE2
Brief Summary
This study is being done to determine the safety and outcome (long-term control) of a high-dose chemotherapy regimen followed by an infusion of CD34 selected (immune cells) stem cells from a partially...
Detailed Description
The purpose of this study is to investigate host myeloimmunosuppressive conditioning followed by familial haploidentical T cell depleted allogeneic stem cell transplantation in patients with high risk...
Eligibility Criteria
Inclusion
- Homozygous Hemoglobin S Disease, or Hemoglobin S Beta0/+ thalassemia
- Patients must demonstrate one or more of the following Sickle Cell Disease Complications
- Clinically significant neurologic event (stroke) or any neurologic deficit lasting \>24 hours that is accompanied by an infarct on cerebral MRI
- Minimum of two episodes of acute chest syndrome.
- Recurrent painful events (at least 3 in the 2 years prior to enrollment).
- Abnormal TCD study requiring starting on chronic transfusion therapy.
- At least one silent infarct lesion on a MRI scan of the head.
- A familial haploidentical donor without homozygous sickle cell disease
- Adequate organ function (renal, liver, cardiac and pulmonary function)
- Karnofsky or Lansky (age appropriate) Performance Score ≥50%
- Liver biopsy is optional to assess for iron overload in chronically transfused patients.
Exclusion
- Females who are pregnant or breast-feeding
- SCD Patients with documented uncontrolled infection
- SCD patients who have an unaffected HLA matched family donor willing to proceed to donation
- Karnofsky/Lansky (age appropriate) Performance Score \<50% (hemiplegia alone secondary to a previous stroke is not an exclusion)
- Demonstrated lack of compliance with medical care.
- Clinically significant fibrosis or cirrhosis of the liver
- Previously received a HSCT
Key Trial Info
Start Date :
January 1 2012
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 1 2026
Estimated Enrollment :
21 Patients enrolled
Trial Details
Trial ID
NCT01461837
Start Date
January 1 2012
End Date
December 1 2026
Last Update
August 8 2025
Active Locations (6)
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1
University of California Los Angeles (UCLA)
Los Angeles, California, United States, 90095
2
Children's Hospital and Research Center Oakland
Oakland, California, United States, 94609
3
Lurie Children's Hospital
Chicago, Illinois, United States, 60611-2605
4
Washington University/St. Louis Children's Hospital
St Louis, Missouri, United States, 63110