Haplo T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease

• Status: Active, not recruiting
• Trial end date: 2025-12-01
• Target enrollment: 0
• Participant gender: All

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 matched adult family member donor, called haploidentical stem cell transplantation, in high-risk sickle cell disease patients.

Funding Source - FDA OOPD

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: New York Medical College
• Collaborators: Ann & Robert H Lurie Children's Hospital of Chicago; Ann and Robert H. Lurie Children's Hospital of Chicago; Children's Hospital & Research Center Oakland; Children's Hospital Medical Center, Cincinnati; Medical College of Wisconsin; Miltenyi Biomedicine GmbH; Miltenyi Biotec GmbH; Tufts Medical Center; UCSF Benioff Children's Hospital Oakland; University of California, Los Angeles; University of California, San Francisco; Washington University School of Medicine

Criteria

Inclusion Criteria:

• 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.

Exclusion Criteria:

• 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