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

Interventional


Primary Outcome

Neutrophil Engraftment

Secondary Outcome

 Incidence of Graft Failure

Condition

SCID

Intervention

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.

Publications

* 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

Drug

Estimated Enrollment

30

Start Date

September 4, 2012

Completion Date

December 2022

Primary Completion Date

December 2021

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
                  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.

        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
             biopsy
      

Gender

All

Ages

N/A - 50 Years

Accepts Healthy Volunteers

No

Contacts

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

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT01652092

Organization ID

2012OC055

Secondary IDs

MT2012-10C

Responsible Party

Sponsor

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

January 2021