Haplo T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease
Running, not enrolling · Phase 2
Conditions studied: Sickle Cell Disease
In brief
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 matched adult family member donor, called haploidentical stem cell transplantation, in high-risk sickle cell disease patients. Funding Source - FDA OOPD
Key facts
- Study ID
- NCT01461837
- Run by
- New York Medical College
- People needed
- 21
- Starts
- 2012-01-01
- Expected to finish
- 2026-12-01
- Last updated by the study team
- 2025-08-08
Who can join
Age: 2 and older, up to 20. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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.
You may not qualify if…
- 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
Where it is running
- University of California Los Angeles (UCLA) — Los Angeles, California, United States
- Children's Hospital and Research Center Oakland — Oakland, California, United States
- Lurie Children's Hospital — Chicago, Illinois, United States
- Washington University/St. Louis Children's Hospital — St Louis, Missouri, United States
- New York Medical College — Valhalla, New York, United States
- Medical College of Wisconsin/Children's Hospital of Wisconsin — Milwaukee, Wisconsin, United States
Full record on ClinicalTrials.gov
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