Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

Summary

This is a standard of care treatment guideline for allogeneic hematopoetic stem cell transplant (HSCT) in patients with primary immune deficiencies.

Conditions

• SCID
• Omenn's Syndrome
• Reticular Dysgenesis
• Wiskott-Aldrich Syndrome
• Bare Lymphocyte Syndrome
• Common Variable Immunodeficiency
• Chronic Granulomatous Disease
• CD40 Ligand Deficiency
• Hyper IgM Syndrome
• X-linked Lymphoproliferative Disease
• Hemophagocytic Lymphohistiocytosis
• Griscelli Syndrome
• Chediak-Higashi Syndrome
• Langerhan's Cell Histiocytosis

Interventions

DRUG

Alemtuzumab 0.3 mg

0.3 mg/kg intravenously (IV) on days -12 through -10

DRUG

Cyclophosphamide

cyclophosphamide 50 mg/kg IV on days -9 through -6

DRUG

Busulfan

busulfan 0.8 or 1.1 mg/kg IV on days -5 through -2

BIOLOGICAL

Stem Cell Transplantation

Unrelated donor bone marrow will be collected in the usual manner using established parameters determined by the National Marrow Donor Program. A minimum of 3 x 10\^8 nucleated cells/kg recipient weight will be collected with a goal of ≥ 5 x 10\^8 nucleated cells/kg recipient weight. Umbilical cord blood selection will be per the current University of Minnesota Cord Blood Unit Selection algorithm. One or two units may be used to obtain the minimum cell dose. One of the UCB units selected for transplantation must contain ≥ 3.5 x 10\^7 nucleated cells/kg recipient weight based on cell numbers at time of cryopreservation, and the total combined cell dose of both units must be \> 5.0 x 10\^7 nucleated cells/kg.

DRUG

Fludarabine phosphate 40 mg

40 mg/m\^2 IV on days -5 through -2 (for children \< 6 months and/or \< 10 kg weight dose at 1.33 mg/kg)

DRUG

Melphalan

140 mg/m\^2 IV on day -3

DRUG

Alemtuzumab 0.2 mg

0.2 mg/kg intravenously (IV) on days -14 through -10

DRUG

Busulfan

busulfan 0.8 or 1.1 mg/kg IV on days -9 through -6

DRUG

Fludarabine phosphate 30 mg

fludarabine 30 mg/m\^2 IV on days -8 through -4

DRUG

MESNA

administered as per the standard institutional protocol.

Sponsors & Collaborators

• Masonic Cancer Center, University of Minnesota

  lead OTHER

Principal Investigators

• Christen Ebens, MD · Masonic Cancer Center, University of Minnesota

Study Design

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Max Age

50 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2012-09-04

Primary Completion

2025-12-30

Completion

2026-12-31

Countries

• United States

Study Locations