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drugset / Trial / NCT01821781

Immune Disorder HSCT Protocol

NCT01821781 ↗

Phase 2 Active not recruiting 20 enrolled Washington University School of Medicine
NaSingle-groupOpen-labelTreatment

Summary

This study hypothesizes that a reduced intensity immunosuppressive preparative regimen will establish engraftment of donor hematopoietic cells with acceptable early and delayed toxicity in patients with immune function disorders. A regimen that maximizes host immune suppression is expected to reduce graft rejection and optimize donor cell engraftment.

Timeline

Start
2013-04-29
Primary completion
2025-04-25
Completion
2026-04

Publications

Drugs

EvaluationDrugModalityDoseRoute
Subject Alemtuzumab Monoclonal antibody 20 mg Intravenous
Subject Fludarabine Small molecule 30 mg/m2 Intravenous
Subject Melphalan Small molecule 140 mg/m2 Intravenous
Subject Thiotepa Other / unclassified 4 mg/kg Intravenous

Indications

Bruton-type agammaglobulinemia Chediak-Higashi syndrome DiGeorge syndrome Wiskott-Aldrich syndrome X-linked lymphoproliferative syndrome autoimmune lymphoproliferative syndrome chronic granulomatous disease common variable immunodeficiency hyper-IgM syndrome immune dysregulation, autoimmunity, and autoinflammation immune dysregulation-polyendocrinopathy-enteropathy-X-linked syndrome severe combined immunodeficiency