Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies
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ClinicalTrials.gov Identifier: NCT01652092 |
Recruitment Status :
Recruiting
First Posted : July 27, 2012
Last Update Posted : January 26, 2023
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Condition or disease | Intervention/treatment | Phase |
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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 | Drug: Alemtuzumab 0.3 mg Drug: Cyclophosphamide Drug: Busulfan Biological: Stem Cell Transplantation Drug: Fludarabine phosphate 40 mg Drug: Melphalan Drug: Alemtuzumab 0.2 mg Drug: Fludarabine phosphate 30 mg Drug: MESNA | Not Applicable |
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 (Clinical Trial) |
Estimated Enrollment : | 30 participants |
Allocation: | Non-Randomized |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies |
Actual Study Start Date : | September 4, 2012 |
Estimated Primary Completion Date : | December 2024 |
Estimated Study Completion Date : | December 2025 |

Arm | Intervention/treatment |
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Arm A: Fully Myeloablative regimen
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.
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Drug: Alemtuzumab 0.3 mg
0.3 mg/kg intravenously (IV) on days -12 through -10
Other Name: Campath-1H Drug: Cyclophosphamide cyclophosphamide 50 mg/kg IV on days -9 through -6
Other Name: Cytoxan Drug: Busulfan busulfan 0.8 or 1.1 mg/kg IV on days -5 through -2
Other Name: Myerlan Biological: Stem Cell Transplantation 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. Drug: MESNA administered as per the standard institutional protocol.
Other Names:
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Arm B: Reduced Toxicity Ablative Regimen
For use in patients with diseases including SCID, CGD, CHS and other CID. Receives Alemtuzumab 0.3 mg/kg intravenously (IV) on days -12 through -10, busulfan 0.8 or 1.1 mg/kg IV on days -9 through -6, fludarabine phosphate 40 mg/m^2 IV on days -5 through -2 and stem cell infusion on day 0.
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Drug: Alemtuzumab 0.3 mg
0.3 mg/kg intravenously (IV) on days -12 through -10
Other Name: Campath-1H Biological: Stem Cell Transplantation 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. Drug: Fludarabine phosphate 40 mg 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 Name: Fludara Drug: Busulfan busulfan 0.8 or 1.1 mg/kg IV on days -9 through -6
Other Name: Myerlan |
Arm C: Reduced Intensity Conditioning
For use in patients with diseases including HLH. Receives Alemtuzumab 0.2 mg/kg intravenously (IV) on days -14 through -10, fludarabine phosphate 30 mg/m^2 IV on days -8 through -4, melphalan 140 mg/m^2 IV on day -3 and stem cell infusion on day 0.
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Biological: Stem Cell Transplantation
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. Drug: Melphalan 140 mg/m^2 IV on day -3
Other Name: Alkeran Drug: Alemtuzumab 0.2 mg 0.2 mg/kg intravenously (IV) on days -14 through -10
Other Name: Campath 1-H Drug: Fludarabine phosphate 30 mg fludarabine 30 mg/m^2 IV on days -8 through -4
Other Name: Fludara |
Arm D: No Preparative Regimen
For use in patients with complete SCID phenotype with no evidence of maternal engraftment or residual immune function who will be receiving their stem cell transplantation from a genotypically matched donor.
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Biological: Stem Cell Transplantation
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. |
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.

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Ages Eligible for Study: | up to 50 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
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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)
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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
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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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01652092
Contact: Christen Ebens, MD | 612-626-2778 | ebens012@umn.edu |
United States, Minnesota | |
Masonic Cancer Center, University of Minnesota | Recruiting |
Minneapolis, Minnesota, United States, 55455 | |
Contact: Christen Ebens, MD 612-626-2778 ebens012@umn.edu | |
Principal Investigator: Christen Ebens, MD |
Principal Investigator: | Christen Ebens, MD | Masonic Cancer Center, University of Minnesota |
Responsible Party: | Masonic Cancer Center, University of Minnesota |
ClinicalTrials.gov Identifier: | NCT01652092 |
Other Study ID Numbers: |
2012OC055 MT2012-10C ( Other Identifier: Blood and Marrow Transplantation Program ) |
First Posted: | July 27, 2012 Key Record Dates |
Last Update Posted: | January 26, 2023 |
Last Verified: | January 2023 |
immunodeficiency disorder histiocytic disorder |
Histiocytosis, Langerhans-Cell Chediak-Higashi Syndrome Histiocytosis Granulomatous Disease, Chronic Lymphohistiocytosis, Hemophagocytic Lymphoproliferative Disorders Wiskott-Aldrich Syndrome Hyper-IgM Immunodeficiency Syndrome Hyper-IgM Immunodeficiency Syndrome, Type 1 Primary Immunodeficiency Diseases Severe Combined Immunodeficiency Immunologic Deficiency Syndromes Common Variable Immunodeficiency Syndrome Disease |
Pathologic Processes Immune System Diseases Lymphatic Diseases Phagocyte Bactericidal Dysfunction Leukocyte Disorders Hematologic Diseases Genetic Diseases, X-Linked Genetic Diseases, Inborn Chronic Disease Disease Attributes Histiocytosis, Non-Langerhans-Cell Immunoproliferative Disorders Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hemorrhagic Disorders |