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NCT Number: NCT01652092

Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

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

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This study is active but is not currently recruiting participants.

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Key information

Conditions

SCID Albinism Bare Lymphocyte Syndrome Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Blood Protein Disorders CD40 Ligand Deficiency Chediak-Higashi Syndrome Chronic Disease Chronic Granulomatous Disease Common Variable Immunodeficiency Congenital, Hereditary, and Neonatal Diseases and Abnormalities Cytopenia DNA Repair-Deficiency Disorders Disease Attributes Dysgammaglobulinemia Eye Diseases Eye Diseases, Hereditary Genetic Diseases, Inborn Genetic Diseases, X-Linked Granulomatous Disease, Chronic Griscelli Syndrome Hematologic Diseases Hemic and Lymphatic Diseases Hemophagocytic Lymphohistiocytosis Hemorrhagic Disorders Histiocytosis Histiocytosis, Langerhans-Cell Histiocytosis, Non-Langerhans-Cell Hyper IgM Syndrome Hyper-IgM Immunodeficiency Syndrome Immune System Diseases Immunologic Deficiency Syndromes Immunoproliferative Disorders Infant, Newborn, Diseases Langerhan's Cell Histiocytosis Leukocyte Disorders Leukopenia Lung Diseases Lung Diseases, Interstitial Lymphatic Diseases Lymphohistiocytosis, Hemophagocytic Lymphopenia Lymphoproliferative Disorders Metabolic Diseases Nutritional and Metabolic Diseases Omenn's Syndrome Pathologic Processes Pathological Conditions, Signs and Symptoms Phagocyte Bactericidal Dysfunction Primary Immunodeficiency Diseases Respiratory Tract Diseases Reticular Dysgenesis Severe Combined Immunodeficiency Wiskott-Aldrich Syndrome X-linked Lymphoproliferative Disease

Age range

Up to 50 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Masonic Cancer Center, University of Minnesota

Minneapolis, Minnesota, 55455, United States

About this study

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

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Alemtuzumab 0.3 mg

Drug

0.3 mg/kg intravenously (IV) on days -12 through -10

Other names: Campath-1H

Cyclophosphamide

Drug

cyclophosphamide 50 mg/kg IV on days -9 through -6

Other names: Cytoxan

busulfan

Drug

busulfan 0.8 or 1.1 mg/kg IV on days -5 through -2

Other names: Myerlan

Stem Cell Transplantation

Biological

Unrelated donor bone marrow will be collected in the usual manner using established parameters determined by the National Marrow Donor Program. A minimum of 3 x 10^8 nucleated cells/kg recipient weight will be collected with a goal of ≥ 5 x 10^8 nucleated cells/kg recipient weight.

Umbilical cord blood selection will be per the current University of Minnesota Cord Blood Unit Selection algorithm. One or two units may be used to obtain the minimum cell dose. One of the UCB units selected for transplantation must contain ≥ 3.5 x 10^7 nucleated cells/kg recipient weight based on cell numbers at time of cryopreservation, and the total combined cell dose of both units must be > 5.0 x 10^7 nucleated cells/kg.

Fludarabine phosphate 40 mg

Drug

40 mg/m^2 IV on days -5 through -2 (for children < 6 months and/or < 10 kg weight dose at 1.33 mg/kg)

Other names: Fludara

melphalan

Drug

140 mg/m^2 IV on day -3

Other names: Alkeran

Alemtuzumab 0.2 mg

Drug

0.2 mg/kg intravenously (IV) on days -14 through -10

Other names: Campath 1-H

Fludarabine phosphate 30 mg

Drug

fludarabine 30 mg/m^2 IV on days -8 through -4

Other names: Fludara

Mesna

Drug

administered as per the standard institutional protocol.

Other names: mercaptoethane sulfonate Na (Na being the symbol for sodium), Mesnex

Primary outcomes

  1. Neutrophil Engraftment

    Time frame: Day 42

    Neutrophil engraftment is defined as the first day of three consecutive days where the neutrophil count (absolute neutrophil count) is 500 cells/mm3 (0.5 x 109/L) or greater.

Secondary outcomes

  1. Incidence of Graft Failure

    Time frame: Day 100

    Graft failure is defined as not accepting donated cells. The donated cells do not make the new white blood cells, red blood cells and platelets.

  2. Incidence of Chimerism

    Time frame: Day 100, 6 Months, 1 Year

    a state in bone marrow transplantation in which bone marrow and host cells exist compatibly without signs of graft-versus-host rejection disease.

  3. Incidence of Acute Graft-Versus-Host Disease

    Time frame: Day 100

    Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host.

  4. Incidence of Chronic Graft-Versus-Host Disease

    Time frame: 6 Months and 1 Year

    Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host.

  5. Incidence of Transplant-Related Mortality

    Time frame: 6 Months

    In the field of transplantation, toxicity is high and all deaths without previous relapse or progression are usually considered as related to transplantation.

  6. Disease-Free Survival

    Time frame: 6 Months

    the length of time after treatment ends that a patient survives without any signs or symptoms of that cancer or any other type of cancer. In a clinical trial, measuring the disease-free survival is one way to see how well a new treatment works.

  7. Overall Survival

    Time frame: 6 Months

    Overall survival will be defined as time from enrollment to date of death or censored at the date of last documented contact for patients still alive.

Sponsors and collaborators

Lead sponsor

Masonic Cancer Center, University of Minnesota

Other

Registry information

Important dates

Study start
2012
Primary completion
2025
Study completion
2026
First posted
Jul 27, 2012
Registry last updated
Jun 17, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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