Immune Disorder HSCT Protocol

ACTIVE NOT RECRUITING

NCT01821781

PHASE2

Sponsor: Washington University School of Medicine

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.

Official title: A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Immune Function Disorders Using a Reduced Intensity Preparatory Regime

Key Details

Gender

All

Age Range

Any - 21 Years

Study Type

INTERVENTIONAL

Enrollment

20

Start Date

2013-04-29

Completion Date

2026-04

Last Updated

2026-02-19

Healthy Volunteers

No

Conditions

Immune Deficiency Disorders Severe Combined Immunodeficiency Chronic Granulomatous Disease X-linked Agammaglobulinemia Wiskott-Aldrich Syndrome Hyper-IgM DiGeorge Syndrome Chediak-Higashi Syndrome Common Variable Immune Deficiency Immune Dysregulatory Disorders Hemophagocytic Lymphohistiocytosis IPEX Autoimmune Lymphoproliferative Syndrome X-linked Lymphoproliferative Syndrome

Interventions

DRUG

Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan

Between days -23 and -15: alemtuzumab test dose, 3mg IV or SQ Day -14: alemtuzumab, 10mg IV or SQ Day -13: alemtuzumab, 15mg IV or SQ Day -12: alemtuzumab, 20mg IV or SQ Days -8 to -4: fludarabine, 30mg/m2 IV Day -4: thiotepa 4mg/kg IV q 12 hours Day -3: melphalan, 140mg/m2 IV Day 0: stem cell infusion Day +7: G-CSF

Locations (1)

Washington University

St Louis, Missouri, United States

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