Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

Launched by MASONIC CANCER CENTER, UNIVERSITY OF MINNESOTA · Jul 26, 2012

Keywords

ClinConnect Summary

This clinical trial is looking at a treatment called allogeneic hematopoietic stem cell transplant (HSCT) for patients with certain primary immune deficiencies. These are conditions where the immune system doesn't work properly, making it hard for the body to fight infections. Some of the conditions included in the study are Severe Combined Immunodeficiency (SCID), Wiskott-Aldrich Syndrome, and Chronic Granulomatous Disease, among others. The trial is currently recruiting participants, and it is open to individuals aged 0 to 50 who have been diagnosed with these immunodeficiencies and have a suitable stem cell donor.

If you or a family member are eligible, you can expect to undergo a stem cell transplant, which involves receiving healthy stem cells from a donor to help rebuild a stronger immune system. Potential participants should be in good health overall and have proper organ function. However, individuals who are pregnant, have active infections, or certain liver conditions cannot join the trial. This treatment could offer hope for better health and improved immune function for those with these serious conditions.

Gender

ALL

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