NCT01652092

Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

• Status: Active Not Recruiting
• Phase: N/A
• Study type: Interventional
• Enrollment: 57 (actual)
• Sponsor: Masonic Cancer Center, University of Minnesota · Academic / Other
• Sex: All
• Age: 50 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed description

Based on diagnosis and clinical history, a determination of the most appropriate regimen will be made based on the following prep plans: Arm A: Fully Myeloablative Preparative Regimen, Arm B: Reduced Toxicity Ablative Preparative Regimen, Arm C: Reduced Intensity Conditioning, Arm D: No Preparative Regimen

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

Type	Name	Description
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.

Timeline

• Start date: 2012-09-04
• Primary completion: 2025-12-30
• Completion: 2026-12-01
• First posted: 2012-07-27
• Last updated: 2026-01-15

Locations

1 site across 1 country: United States