drugset
drugset / Trial / NCT01461837

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

NCT01461837 ↗

Phase 2 Active not recruiting 21 enrolled New York Medical College Ann & Robert H Lurie Children's Hospital of Chicago · collabMedical College of Wisconsin · collabMiltenyi Biomedicine GmbH · collabTufts Medical Center · collabUCSF Benioff Children's Hospital Oakland · collabUniversity of California, Los Angeles · collabUniversity of California, San Francisco · collabWashington University School of Medicine · collab
NaSingle-groupOpen-labelTreatment

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

Timeline

Start
2012-01
Primary completion
2025-12
Completion
2026-12

Publications

Drugs

EvaluationDrugModalityDoseRoute
Subject CD34 selected T-cell depleted allogeneic SCT Cell therapy — —
Background Azathioprine Small molecule 3 mg/kg —
Background Busulfan Small molecule 3.2 mg/kg —
Background Cyclophosphamide Other / unclassified 50 mg/kg —
Background Fludarabine Small molecule 30 mg/m2 —
Background Hydroxyurea Other / unclassified 60 mg/kg —
Background Thiotepa Other / unclassified 10 mg/kg Intravenous
Background Thymoglobulin Protein / enzyme biologic 2 mg/kg —

Indications

sickle cell disease