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| ID | Type | Description | Link |
|---|---|---|---|
| MT2012-10C | Other Identifier | Blood and Marrow Transplantation Program |
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This is a standard of care treatment guideline for allogeneic hematopoetic stem cell transplant (HSCT) in patients with primary immune deficiencies.
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
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Arm A: Fully Myeloablative regimen | Other | 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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| Arm B: Reduced Toxicity Ablative Regimen | Other | 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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| Arm C: Reduced Intensity Conditioning | Other | 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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| Arm D: No Preparative Regimen | Other |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Alemtuzumab 0.3 mg | Drug | 0.3 mg/kg intravenously (IV) on days -12 through -10 |
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| Measure | Description | Time Frame |
|---|---|---|
| Neutrophil Engraftment | 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. | Day 42 |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of Graft Failure | 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. | Day 100 |
| Incidence of Chimerism |
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Inclusion Criteria:
Diagnosis of immunodeficiency or histiocytic disorder including the following:
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:
Age: 0 to 50 years
Adequate organ function and performance status.
Exclusion Criteria
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| Name | Affiliation | Role |
|---|---|---|
| Meera Srikanthan, MD | Masonic Cancer Center, University of Minnesota | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Masonic Cancer Center, University of Minnesota | Minneapolis | Minnesota | 55455 | United States |
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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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| Cyclophosphamide | Drug | cyclophosphamide 50 mg/kg IV on days -9 through -6 |
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| Busulfan | Drug | busulfan 0.8 or 1.1 mg/kg IV on days -5 through -2 |
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| 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. |
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| 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) |
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| Melphalan | Drug | 140 mg/m^2 IV on day -3 |
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| Alemtuzumab 0.2 mg | Drug | 0.2 mg/kg intravenously (IV) on days -14 through -10 |
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| Busulfan | Drug | busulfan 0.8 or 1.1 mg/kg IV on days -9 through -6 |
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| Fludarabine phosphate 30 mg | Drug | fludarabine 30 mg/m^2 IV on days -8 through -4 |
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| MESNA | Drug | administered as per the standard institutional protocol. |
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a state in bone marrow transplantation in which bone marrow and host cells exist compatibly without signs of graft-versus-host rejection disease.
| Day 100, 6 Months, 1 Year |
| Incidence of Acute Graft-Versus-Host Disease | Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host. | Day 100 |
| Incidence of Chronic Graft-Versus-Host Disease | Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host. | 6 Months and 1 Year |
| Incidence of Transplant-Related Mortality | In the field of transplantation, toxicity is high and all deaths without previous relapse or progression are usually considered as related to transplantation. | 6 Months |
| Disease-Free Survival | 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. | 6 Months |
| Overall Survival | 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. | 6 Months |
| ID | Term |
|---|---|
| D016511 | Severe Combined Immunodeficiency |
| C538361 | Reticular dysgenesis |
| D014923 | Wiskott-Aldrich Syndrome |
| D017074 | Common Variable Immunodeficiency |
| D006105 | Granulomatous Disease, Chronic |
| D053306 | Hyper-IgM Immunodeficiency Syndrome |
| D008232 | Lymphoproliferative Disorders |
| D051359 | Lymphohistiocytosis, Hemophagocytic |
| D002609 | Chediak-Higashi Syndrome |
| D006646 | Histiocytosis, Langerhans-Cell |
| D007153 | Immunologic Deficiency Syndromes |
| ID | Term |
|---|---|
| D000081207 | Primary Immunodeficiency Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |
| D049914 | DNA Repair-Deficiency Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D007154 | Immune System Diseases |
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D008231 | Lymphopenia |
| D007970 | Leukopenia |
| D000095542 | Cytopenia |
| D006474 | Hemorrhagic Disorders |
| D007960 | Leukocyte Disorders |
| D040181 | Genetic Diseases, X-Linked |
| D010585 | Phagocyte Bactericidal Dysfunction |
| D002908 | Chronic Disease |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D004406 | Dysgammaglobulinemia |
| D001796 | Blood Protein Disorders |
| D008206 | Lymphatic Diseases |
| D007160 | Immunoproliferative Disorders |
| D015616 | Histiocytosis, Non-Langerhans-Cell |
| D015614 | Histiocytosis |
| D000417 | Albinism |
| D015785 | Eye Diseases, Hereditary |
| D005128 | Eye Diseases |
| D017563 | Lung Diseases, Interstitial |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
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| ID | Term |
|---|---|
| D000074323 | Alemtuzumab |
| D003520 | Cyclophosphamide |
| D002066 | Busulfan |
| D033581 | Stem Cell Transplantation |
| C042382 | fludarabine phosphate |
| D008558 | Melphalan |
| D015080 | Mesna |
| D012964 | Sodium |
| ID | Term |
|---|---|
| D061067 | Antibodies, Monoclonal, Humanized |
| D000911 | Antibodies, Monoclonal |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
| D007162 | Immunoproteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D012712 | Serum Globulins |
| D005916 | Globulins |
| D010752 | Phosphoramide Mustards |
| D009588 | Nitrogen Mustard Compounds |
| D009150 | Mustard Compounds |
| D006846 | Hydrocarbons, Halogenated |
| D006838 | Hydrocarbons |
| D009930 | Organic Chemicals |
| D063088 | Phosphoramides |
| D009943 | Organophosphorus Compounds |
| D002072 | Butylene Glycols |
| D006018 | Glycols |
| D000438 | Alcohols |
| D008698 | Mesylates |
| D000476 | Alkanesulfonates |
| D017738 | Alkanesulfonic Acids |
| D000473 | Alkanes |
| D006839 | Hydrocarbons, Acyclic |
| D013451 | Sulfonic Acids |
| D013456 | Sulfur Acids |
| D013457 | Sulfur Compounds |
| D017690 | Cell Transplantation |
| D064987 | Cell- and Tissue-Based Therapy |
| D001691 | Biological Therapy |
| D013812 | Therapeutics |
| D014180 | Transplantation |
| D013514 | Surgical Procedures, Operative |
| D010649 | Phenylalanine |
| D024322 | Amino Acids, Aromatic |
| D000598 | Amino Acids, Cyclic |
| D000596 | Amino Acids |
| D013438 | Sulfhydryl Compounds |
| D008672 | Metals, Alkali |
| D004602 | Elements |
| D007287 | Inorganic Chemicals |
| D019565 | Metals, Light |
| D008670 | Metals |
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