Ignite Creation Date:
2025-12-24 @ 11:32 PM
Ignite Modification Date:
2025-12-25 @ 9:20 PM
Study NCT ID:
NCT00006056
Status:
UNKNOWN
Last Update Posted:
2005-06-24
First Post:
2000-07-05
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Pilot Study of Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients With Life Threatening Hemophagocytic Disorders
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D002609', 'term': 'Chediak-Higashi Syndrome'}, {'id': 'D006086', 'term': 'Graft vs Host Disease'}, {'id': 'D008232', 'term': 'Lymphoproliferative Disorders'}, {'id': 'D051359', 'term': 'Lymphohistiocytosis, Hemophagocytic'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D015614', 'term': 'Histiocytosis'}, {'id': 'D007154', 'term': 'Immune System Diseases'}, {'id': 'D003141', 'term': 'Communicable Diseases'}, {'id': 'D000081207', 'term': 'Primary Immunodeficiency Diseases'}, {'id': 'D035583', 'term': 'Rare Diseases'}], 'ancestors': [{'id': 'D000417', 'term': 'Albinism'}, {'id': 'D015785', 'term': 'Eye Diseases, Hereditary'}, {'id': 'D005128', 'term': 'Eye Diseases'}, {'id': 'D010585', 'term': 'Phagocyte Bactericidal Dysfunction'}, {'id': 'D007960', 'term': 'Leukocyte Disorders'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D007153', 'term': 'Immunologic Deficiency Syndromes'}, {'id': 'D008206', 'term': 'Lymphatic Diseases'}, {'id': 'D007160', 'term': 'Immunoproliferative Disorders'}, {'id': 'D015616', 'term': 'Histiocytosis, Non-Langerhans-Cell'}, {'id': 'D007239', 'term': 'Infections'}, {'id': 'D020969', 'term': 'Disease Attributes'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D000961', 'term': 'Antilymphocyte Serum'}, {'id': 'D002066', 'term': 'Busulfan'}, {'id': 'D003520', 'term': 'Cyclophosphamide'}, {'id': 'D016572', 'term': 'Cyclosporine'}, {'id': 'D005047', 'term': 'Etoposide'}, {'id': 'D000069585', 'term': 'Filgrastim'}, {'id': 'D008727', 'term': 'Methotrexate'}], 'ancestors': [{'id': 'D007106', 'term': 'Immune Sera'}, {'id': 'D000906', 'term': 'Antibodies'}, {'id': 'D007136', 'term': 'Immunoglobulins'}, {'id': 'D007162', 'term': 'Immunoproteins'}, {'id': 'D001798', 'term': 'Blood Proteins'}, {'id': 'D011506', 'term': 'Proteins'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}, {'id': 'D012712', 'term': 'Serum Globulins'}, {'id': 'D005916', 'term': 'Globulins'}, {'id': 'D001688', 'term': 'Biological Products'}, {'id': 'D045424', 'term': 'Complex Mixtures'}, {'id': 'D002072', 'term': 'Butylene Glycols'}, {'id': 'D006018', 'term': 'Glycols'}, {'id': 'D000438', 'term': 'Alcohols'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D008698', 'term': 'Mesylates'}, {'id': 'D000476', 'term': 'Alkanesulfonates'}, {'id': 'D017738', 'term': 'Alkanesulfonic Acids'}, {'id': 'D000473', 'term': 'Alkanes'}, {'id': 'D006839', 'term': 'Hydrocarbons, Acyclic'}, {'id': 'D006838', 'term': 'Hydrocarbons'}, {'id': 'D013451', 'term': 'Sulfonic Acids'}, {'id': 'D013456', 'term': 'Sulfur Acids'}, {'id': 'D013457', 'term': 'Sulfur Compounds'}, {'id': 'D010752', 'term': 'Phosphoramide Mustards'}, {'id': 'D009588', 'term': 'Nitrogen Mustard Compounds'}, {'id': 'D009150', 'term': 'Mustard Compounds'}, {'id': 'D006846', 'term': 'Hydrocarbons, Halogenated'}, {'id': 'D063088', 'term': 'Phosphoramides'}, {'id': 'D009943', 'term': 'Organophosphorus Compounds'}, {'id': 'D003524', 'term': 'Cyclosporins'}, {'id': 'D010456', 'term': 'Peptides, Cyclic'}, {'id': 'D047028', 'term': 'Macrocyclic Compounds'}, {'id': 'D011083', 'term': 'Polycyclic Compounds'}, {'id': 'D010455', 'term': 'Peptides'}, {'id': 'D011034', 'term': 'Podophyllotoxin'}, {'id': 'D013764', 'term': 'Tetrahydronaphthalenes'}, {'id': 'D009281', 'term': 'Naphthalenes'}, {'id': 'D011084', 'term': 'Polycyclic Aromatic Hydrocarbons'}, {'id': 'D006841', 'term': 'Hydrocarbons, Aromatic'}, {'id': 'D006844', 'term': 'Hydrocarbons, Cyclic'}, {'id': 'D005960', 'term': 'Glucosides'}, {'id': 'D006027', 'term': 'Glycosides'}, {'id': 'D002241', 'term': 'Carbohydrates'}, {'id': 'D016179', 'term': 'Granulocyte Colony-Stimulating Factor'}, {'id': 'D003115', 'term': 'Colony-Stimulating Factors'}, {'id': 'D006023', 'term': 'Glycoproteins'}, {'id': 'D006001', 'term': 'Glycoconjugates'}, {'id': 'D016298', 'term': 'Hematopoietic Cell Growth Factors'}, {'id': 'D016207', 'term': 'Cytokines'}, {'id': 'D036341', 'term': 'Intercellular Signaling Peptides and Proteins'}, {'id': 'D001685', 'term': 'Biological Factors'}, {'id': 'D000630', 'term': 'Aminopterin'}, {'id': 'D011622', 'term': 'Pterins'}, {'id': 'D011621', 'term': 'Pteridines'}, {'id': 'D006574', 'term': 'Heterocyclic Compounds, 2-Ring'}, {'id': 'D000072471', 'term': 'Heterocyclic Compounds, Fused-Ring'}, {'id': 'D006571', 'term': 'Heterocyclic Compounds'}]}}, 'protocolSection': {'designModule': {'phases': ['NA'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'primaryPurpose': 'TREATMENT'}, 'enrollmentInfo': {'count': 40}}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2000-03'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2003-10', 'lastUpdateSubmitDate': '2005-06-23', 'studyFirstSubmitDate': '2000-07-05', 'studyFirstSubmitQcDate': '2000-07-05', 'lastUpdatePostDateStruct': {'date': '2005-06-24', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2000-07-06', 'type': 'ESTIMATED'}}, 'conditionsModule': {'keywords': ['Chediak-Higashi syndrome', 'X-linked lymphoproliferative syndrome', 'disease-related problem/condition', 'familial erythrophagocytic lymphohistiocytosis', 'genetic diseases and dysmorphic syndromes', 'graft versus host disease', 'hematologic disorders', 'hemophagocytic lymphohistiocytosis', 'histiocytosis', 'immunologic disorders and infectious disorders', 'primary immunodeficiency disease', 'rare disease', 'virus-associated hemophagocytic syndrome'], 'conditions': ['Chediak-Higashi Syndrome', 'Graft Versus Host Disease', 'X-Linked Lymphoproliferative Syndrome', 'Familial Erythrophagocytic Lymphohistiocytosis', 'Hemophagocytic Lymphohistiocytosis', 'Virus-Associated Hemophagocytic Syndrome']}, 'descriptionModule': {'briefSummary': 'OBJECTIVES: I. Determine the efficacy of unrelated donor hematopoietic stem cell transplantation in the treatment of patients with life threatening hemophagocytic disorders.\n\nII. Determine the rate of disease free survival, incidence of graft failure, and incidence of graft versus host disease in these patients after undergoing this treatment regimen.', 'detailedDescription': 'PROTOCOL OUTLINE: Patients receive oral busulfan twice a day on days -9 to -6; cyclophosphamide IV over 1 hour on days -5 to -2; etoposide IV over 4 hours on days -5 to -3; and anti-thymocyte globulin IV twice a day on days -2 and -1 and days 1 and 2. Patients undergo allogeneic hematopoietic stem cell transplantation on day 0. Filgrastim (G-CSF) is administered subcutaneously beginning on day 1 and continuing until blood counts recover. Patients receive graft versus host disease prophylaxis with methotrexate IV on days 1, 3, 6, and 11 and cyclosporine IV over 1-4 hours (orally once the patients resumes eating) every 12 hours (every 8 hours for pediatric patients) starting on or prior to day -3 and continuing up to 1 year.\n\nPatients are followed at days 28 and 100, at 6 months and 1 year, and then annually for 5 years.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '55 Years', 'minimumAge': '0 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'PROTOCOL ENTRY CRITERIA:\n\n--Disease Characteristics--\n\nPatients diagnosed with any of the following active but stable, or nonactive/quiescent, hemophagocytic disorders:\n\n* Hemophagocytic lymphohistiocytosis (HLH)\n* Fever greater than 38.5 degrees Celsius\n* Splenomegaly (greater than 3 cm below costal margin)\n* Hemophagocytosis in bone marrow or spleen or lymph nodes\n* Disease may be confirmed by positive family history\n* No evidence of malignancy\n* Hypertriglyceridemia and/or hypofibrinogenemia\n* Fasting triglycerides at least 2.0 mmol/L or at least 3 standard deviations above normal for age\n* Fibrinogen no greater than 1.5 g/L or no greater than 3 standard deviations above normal\n* Cytopenia (affecting at least 2 of 3 lineages in the peripheral blood)\n* Hemoglobin less than 9.0 g/L\n* Platelet count less than 100,000/mm3\n\nX-linked lymphoproliferative disorder (XLP)\n\nTwo or more maternally related males manifesting at least one of the following XLP phenotypes:\n\n* Fulminant infectious mononucleosis\n* Dysgammaglobulinemia\n* Malignant lymphoma/lymphoproliferative disorder\n* Aplastic anemia\n* Lymphoid granulomatosis/vasculitis OR\n* A maternally related male in an established XLP kindred who has strong genetic (RFLP) linkage to the XLP locus\n\nChediak-Higashi syndrome\n\nPartial oculocutaneous albinism (hair, skin, eyes)\n\nFrequent bacterial infections\n\nLarge peroxidase positive granules in leukocytes of peripheral blood or bone marrow\n\nPositive family history or parental consanguinity is supportive of the diagnosis\n\nMay not have entered accelerated phase as defined by any of the following:\n\n* Lymphadenopathy\n* Pancytopenia\n* Histiocytes with hemophagocytosis in bone marrow, lymph nodes, liver, or spleen\n\nViral associated hemophagocytic syndrome (VAHS)\n\nRelapsed after prior therapy or supportive care\n\nDiagnostic criteria as for HLH\n\nNo hemophagocytic disorders secondary to underlying malignancy\n\nPatients 35 years of age and under must have a hematopoietic stem cell donor that is one of the following:\n\n* HLA A and B identical OR\n* Single HLA A or B serologic mismatch with DRB1 identity OR\n* HLA A or B serologic identity with a single DRB1 mismatch\n\nPatients 36 to 55 years of age must have a hematopoietic stem cell donor that is one of the following:\n\n* HLA A and B and HLA DRB1 identical OR\n* Single HLA A or B serologic mismatch with DRB1 identity\n\nPatients receiving umbilical cord blood must have an unrelated donor with no more than two antigen HLA A, B, or DRB1 mismatches\n\n--Patient Characteristics--\n\nPerformance status: Karnofsky 70-100% OR Age less than 16 years: Lansky 50-100%\n\nLife expectancy: Not severly limited by another disease\n\nHepatic: SGOT less than 3 times normal Bilirubin less than 2.5 mg/dL\n\nRenal: Creatinine normal OR Creatinine clearance or glomerular filtration rate greater than 50% normal\n\nCardiovascular: If symptomatic, ventricular ejection fraction must be greater than 40% and must improve with exercise OR Shortening fraction normal on echocardiogram\n\nPulmonary:\n\n* If symptomatic, DLCO greater than 45% predicted (corrected for hemoglobin)\n* In children unable to perform pulmonary function testing, oxygen saturation must be greater than 95%\n\nOther: HIV negative No significant active infections'}, 'identificationModule': {'nctId': 'NCT00006056', 'briefTitle': 'Pilot Study of Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients With Life Threatening Hemophagocytic Disorders', 'organization': {'class': 'NIH', 'fullName': 'Office of Rare Diseases (ORD)'}, 'orgStudyIdInfo': {'id': '199/15106'}, 'secondaryIdInfos': [{'id': 'UMN-MT-1997-08'}, {'id': 'UMN-MT-9708'}]}, 'armsInterventionsModule': {'interventions': [{'name': 'anti-thymocyte globulin', 'type': 'DRUG'}, {'name': 'busulfan', 'type': 'DRUG'}, {'name': 'cyclophosphamide', 'type': 'DRUG'}, {'name': 'cyclosporine', 'type': 'DRUG'}, {'name': 'etoposide', 'type': 'DRUG'}, {'name': 'filgrastim', 'type': 'DRUG'}, {'name': 'methotrexate', 'type': 'DRUG'}, {'name': 'allogeneic hematopoietic stem cell transplantation', 'type': 'PROCEDURE'}]}, 'contactsLocationsModule': {'locations': [{'zip': '55455', 'city': 'Minneapolis', 'state': 'Minnesota', 'country': 'United States', 'facility': 'Fairview University Medical Center', 'geoPoint': {'lat': 44.97997, 'lon': -93.26384}}], 'overallOfficials': [{'name': 'K. Scott Baker', 'role': 'STUDY_CHAIR', 'affiliation': 'Fairview University Medical Center'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Fairview University Medical Center', 'class': 'OTHER'}}}}