Clinical trial NCT01652092: Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies
Official title: Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
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
Keywords: immunodeficiency disorder
histiocytic disorder
Lead sponsor: Masonic Cancer Center, University of Minnesota
Sponsor class: OTHER
First posted: 2012-07-27
Enrollment: 30
Sex: ALL
Maximum age (years): 50
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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

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

Primary outcomes:
measure: Neutrophil Engraftment
timeFrame: Day 42
Secondary outcomes:
measure: Incidence of Graft Failure
timeFrame: Day 100
measure: Incidence of Chimerism
timeFrame: Day 100, 6 Months, 1 Year
measure: Incidence of Acute Graft-Versus-Host Disease
timeFrame: Day 100
measure: Incidence of Chronic Graft-Versus-Host Disease
timeFrame: 6 Months and 1 Year
measure: Incidence of Transplant-Related Mortality
timeFrame: 6 Months
measure: Disease-Free Survival
timeFrame: 6 Months
measure: Overall Survival
timeFrame: 6 Months
Other outcomes:
Locations:
city: Minneapolis
country: United States
facility: Masonic Cancer Center, University of Minnesota
state: Minnesota
Study officials:
affiliation: Masonic Cancer Center, University of Minnesota
name: Christen Ebens, MD
role: PRINCIPAL_INVESTIGATOR
References: