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This study hypothesizes that a reduced intensity immunosuppressive preparative regimen will establish engraftment of donor hematopoietic cells with acceptable early and delayed toxicity in patients with immune function disorders. A regimen that maximizes host immune suppression is expected to reduce graft rejection and optimize donor cell engraftment.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Preparative | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan | Drug | Between days -23 and -15: alemtuzumab test dose, 3mg IV or SQ Day -14: alemtuzumab, 10mg IV or SQ Day -13: alemtuzumab, 15mg IV or SQ Day -12: alemtuzumab, 20mg IV or SQ Days -8 to -4: fludarabine, 30mg/m2 IV Day -4: thiotepa 4mg/kg IV q 12 hours Day -3: melphalan, 140mg/m2 IV Day 0: stem cell infusion Day +7: G-CSF |
| Measure | Description | Time Frame |
|---|---|---|
| Number of participants with donor engraftment | 1 year post transplant |
| Measure | Description | Time Frame |
|---|---|---|
| Major Transplant Related Toxicities | 1 years post transplant | |
| Time to neutrophil recovery | within 100 days post transplant | |
| Number of patient with acute GVHD |
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Inclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Washington University | St Louis | Missouri | 63110 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 31599480 | Derived | Bhatt ST, Schulz G, Hente M, Slater A, Murray L, Shenoy S, Bednarski JJ. A single-center experience using alemtuzumab, fludarabine, melphalan, and thiotepa as conditioning for transplantation in pediatric patients with chronic granulomatous disease. Pediatr Blood Cancer. 2020 Jan;67(1):e28030. doi: 10.1002/pbc.28030. Epub 2019 Oct 10. |
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| Type | Date | Date Unknown |
|---|---|---|
| Release | May 15, 2026 | |
| Reset | Jun 11, 2026 | |
| Release | Jun 12, 2026 |
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|
| 180 days post transplant |
| Number of participants with infectious complications | 2 years post transplant |
| Time to immune reconstitution | 2 years post transplant |
| Overall survival | 2 years post transplant |
| Time to platelet recovery | within 100 days post transplant |
| Number of patients with chronic GVHD | 2 years post transplant |
| Disease free survival | 2 years post transplant |
| Reset | Jul 9, 2026 |
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| Release Date | Unrelease Date | Unrelease Date Unknown | Reset Date | MCP Release Number |
|---|---|---|---|---|
| May 15, 2026 | Jun 11, 2026 | |||
| Jun 12, 2026 | Jul 9, 2026 |
| ID | Term |
|---|---|
| D016511 | Severe Combined Immunodeficiency |
| D006105 | Granulomatous Disease, Chronic |
| C537409 | Bruton type agammaglobulinemia |
| D014923 | Wiskott-Aldrich Syndrome |
| D053306 | Hyper-IgM Immunodeficiency Syndrome |
| D004062 | DiGeorge Syndrome |
| D002609 | Chediak-Higashi Syndrome |
| D017074 | Common Variable Immunodeficiency |
| D051359 | Lymphohistiocytosis, Hemophagocytic |
| D056735 | Autoimmune Lymphoproliferative Syndrome |
| D008232 | Lymphoproliferative Disorders |
| D007153 | Immunologic Deficiency Syndromes |
| D007154 | Immune System Diseases |
| 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 |
| D010585 | Phagocyte Bactericidal Dysfunction |
| D007960 | Leukocyte Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D002908 | Chronic Disease |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D008231 | Lymphopenia |
| D007970 | Leukopenia |
| D000095542 | Cytopenia |
| D006474 | Hemorrhagic Disorders |
| D004406 | Dysgammaglobulinemia |
| D001796 | Blood Protein Disorders |
| D058165 | 22q11 Deletion Syndrome |
| D019465 | Craniofacial Abnormalities |
| D009139 | Musculoskeletal Abnormalities |
| D009140 | Musculoskeletal Diseases |
| D006330 | Heart Defects, Congenital |
| D018376 | Cardiovascular Abnormalities |
| D002318 | Cardiovascular Diseases |
| D006331 | Heart Diseases |
| D044148 | Lymphatic Abnormalities |
| D008206 | Lymphatic Diseases |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D025063 | Chromosome Disorders |
| D007011 | Hypoparathyroidism |
| D010279 | Parathyroid Diseases |
| D004700 | Endocrine System Diseases |
| D000417 | Albinism |
| D015785 | Eye Diseases, Hereditary |
| D005128 | Eye Diseases |
| D015616 | Histiocytosis, Non-Langerhans-Cell |
| D015614 | Histiocytosis |
| D001327 | Autoimmune Diseases |
| D007160 | Immunoproliferative Disorders |
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| ID | Term |
|---|---|
| C024352 | fludarabine |
| D013852 | Thiotepa |
| D008558 | Melphalan |
| ID | Term |
|---|---|
| D063088 | Phosphoramides |
| D009943 | Organophosphorus Compounds |
| D009930 | Organic Chemicals |
| D013721 | Triethylenephosphoramide |
| D001388 | Aziridines |
| D001389 | Azirines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D009588 | Nitrogen Mustard Compounds |
| D009150 | Mustard Compounds |
| D006846 | Hydrocarbons, Halogenated |
| D006838 | Hydrocarbons |
| D010649 | Phenylalanine |
| D024322 | Amino Acids, Aromatic |
| D000598 | Amino Acids, Cyclic |
| D000596 | Amino Acids |
| D000602 | Amino Acids, Peptides, and Proteins |
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