Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

Brief Title

Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

Official Title

Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

Brief 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

Study Type


Primary Outcome

Neutrophil Engraftment

Secondary Outcome

 Incidence of Graft Failure




Alemtuzumab 0.3 mg

Study Arms / Comparison Groups

 Arm A: Fully Myeloablative regimen
Description:  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.


* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status


Estimated Enrollment


Start Date

September 4, 2012

Completion Date

December 2022

Primary Completion Date

December 2022

Eligibility Criteria

        Inclusion Criteria:

          -  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

               -  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

                    -  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.

        Exclusion Criteria

          -  pregnant or breastfeeding

          -  active, uncontrolled infection and/or HIV positive

          -  acute hepatitis or evidence of moderate or severe portal fibrosis or cirrhosis on




N/A - 50 Years

Accepts Healthy Volunteers



Angela R. Smith, M.D., 612-626-2778, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations



Organization ID


Secondary IDs


Responsible Party


Study Sponsor

Masonic Cancer Center, University of Minnesota

Study Sponsor

Angela R. Smith, M.D., Principal Investigator, Masonic Cancer Center, University of Minnesota

Verification Date

April 2022