Familial Haploidentical T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease (IND 14359)

Locations

6 United States sites

Age

2 to 20 Years

Phase

Phase 2

Study type

Interventional

Gender

All

Interventions

Drug

Compensation

Unknown

About the study

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

participation requirements

– Homozygous Hemoglobin S Disease, or Hemoglobin S Beta0/+ thalassemia

– Patients must demonstrate one or more of the following Sickle Cell Disease
Complications

1. Clinically significant neurologic event (stroke) or any neurologic deficit
lasting >24 hours that is accompanied by an infarct on cerebral MRI

2. Minimum of two episodes of acute chest syndrome.

3. Recurrent painful events (at least 3 in the 2 years prior to enrollment).

4. Abnormal TCD study requiring starting on chronic transfusion therapy.

5. 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.

participation restrictions

– 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

Locations

  • Los Angeles, California, United States, University of California Los Angeles (UCLA), 90095
  • Oakland, California, United States, Children's Hospital and Research Center Oakland, 94609
  • Chicago, Illinois, United States, Lurie Children's Hospital, 60611-2605
  • Saint Louis, Missouri, United States, Washington University/St. Louis Children's Hospital, 63110
  • Valhalla, New York, United States, New York Medical College, 10595
  • Milwaukee, Wisconsin, United States, Medical College of Wisconsin/Children's Hospital of Wisconsin, 53226