Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

Status: Recruiting

Location: See location...

Intervention Type: Drug, Biological

Study Type: Interventional

Study Phase: Not Applicable

SUMMARY

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

Eligibility

Participation Requirements

Sex: All

Maximum Age: 50

Healthy Volunteers: f

View:

• Diagnosis of immunodeficiency or histiocytic disorder including the following:

‣ Severe combined immunodeficiency (SCID - all variants)

⁃ Second bone marrow transplant (BMT) for SCID (after graft rejection)

⁃ Omenn's Syndrome

⁃ Reticular dysgenesis

⁃ Wiskott-Aldrich syndrome

⁃ Major histocompatibility complex (MHC) Class II deficiency (bare lymphocyte syndrome)

⁃ Hyper IgM Syndrome (CD40 Ligand Deficiency)

⁃ Common variable immunodeficiency (CVID) with severe phenotype

⁃ Chronic Granulomatous Disease (CGD)

⁃ Other severe Combined Immune Deficiencies (CID)

⁃ Hemophagocytic Lymphohistiocytosis (HLH)

⁃ X-linked Lymphoproliferative Disease (XLP)

⁃ Chediak-Higashi Syndrome (CHS)

⁃ Griscelli Syndrome

⁃ Langerhans Cell Histiocytosis (LCH)

• Acceptable stem cell sources include:

‣ HLA identical or 1 antigen matched sibling donor eligible to donate bone marrow

⁃ HLA identical or up to a 1 antigen mismatched unrelated BM donor

⁃ Sibling donor cord blood with acceptable HLA match and cell dose as per current institutional standards

⁃ Single unrelated umbilical cord blood unit with 0-2 antigen mismatch and minimum cell dose of \>5 x 10\^7 nucleated cells/kg as per current institutional guidelines

⁃ Double unrelated umbilical cord blood units that are:

• up to 2 antigen mismatched to the patient

∙ up to 2 antigen mismatched to each other

∙ minimum cell dose of at least one single unit must be ≥ 3.5 x 10\^7 nucleated cells/kg

∙ combined dose of both units must provide a total cell dose of ≥ 5 x 10\^7 nucleated cells/kg

• Age: 0 to 50 years

• Adequate organ function and performance status.

Locations

United States

Minnesota

Masonic Cancer Center, University of Minnesota

RECRUITING

Minneapolis

Contact Information

Primary

Christen Ebens, MD

ebens012@umn.edu

612-626-2778

Time Frame

Start Date: 2012-09-04

Estimated Completion Date: 2026-12

Participants

Target number of participants: 30

Treatments

Other: Arm A: Fully Myeloablative regimen

For use in patients with diseases including Wiskott-Aldrich syndrome, MHC Class II deficiency, hypomorphic SCID, etc. Receives Alemtuzumab 0.3 mg/kg intravenously (IV) on days -12 through -10, cyclophosphamide 50 mg/kg IV plus MESNA on days -9 through -6, busulfan 0.8 or 1.1 mg/kg IV on days -5 through -2 and stem cell infusion on day 0.

Other: Arm B: Reduced Toxicity Ablative Regimen

For use in patients with diseases including SCID, CGD, CHS and other CID. Receives Alemtuzumab 0.3 mg/kg intravenously (IV) on days -12 through -10, busulfan 0.8 or 1.1 mg/kg IV on days -9 through -6, fludarabine phosphate 40 mg/m\^2 IV on days -5 through -2 and stem cell infusion on day 0.

Other: Arm C: Reduced Intensity Conditioning

For use in patients with diseases including HLH. Receives Alemtuzumab 0.2 mg/kg intravenously (IV) on days -14 through -10, fludarabine phosphate 30 mg/m\^2 IV on days -8 through -4, melphalan 140 mg/m\^2 IV on day -3 and stem cell infusion on day 0.

Other: Arm D: No Preparative Regimen

For use in patients with complete SCID phenotype with no evidence of maternal engraftment or residual immune function who will be receiving their stem cell transplantation from a genotypically matched donor.

Authors

Angela R. Smith

Related Therapeutic Areas

Primary Immunodeficiency (PID)

Hypomelanotic Disorder

Reticulohistiocytoma

Hemophagocytic Lymphohistiocytosis

Non-Langerhans-Cell Histiocytosis

Langerhans Cell Histiocytosis

Wiskott-Aldrich Syndrome

Common Variable Immune Deficiency

Chronic Granulomatous Disease