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58 studies found for:    "Toni-Debre-Fanconi syndrome"
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Rank Status Study
1 Completed Fanconi Syndrome Due to ARVs in HIV-Infected Persons
Conditions: HIV Infections;   Fanconi Syndrome;   Kidney Disease;   Renal Impairment
Intervention: Procedure: Blood Draws
2 Completed Evaluation of Fanconi Syndrome and Cystinosis
Conditions: Cystinosis;   Fanconi Syndrome
Intervention:
3 Completed Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias
Conditions: Beta-thalassemia;   Myelodysplastic Syndromes;   Fanconi Syndrome;   Anemia, Diamond-Blackfan;   Anemia, Aplastic
Intervention: Drug: Deferasirox
4 Recruiting Cure Cystinosis International Registry
Conditions: Cystinosis;   Nephropathic Cystinosis;   Renal Fanconi Syndrome
Intervention:
5 Completed Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes
Conditions: Shwachman Syndrome;   Fanconi's Anemia;   Dyskeratosis Congenita;   Thrombocytopenia
Intervention: Drug: filgrastim
6 Recruiting HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy
Conditions: Fanconi Anemia;   Severe Marrow Failure;   Myelodysplastic Syndrome (MDS);   Acute Myelogenous Leukemia (AML)
Interventions: Drug: Busulfan;   Drug: Cyclophosphamide;   Drug: Fludarabine;   Drug: rabbit ATG;   Drug: G-CSF;   Biological: Peripheral blood stem cell
7 Terminated
Has Results
Stem Cell Transplantation for Fanconi Anemia
Condition: Fanconi Anemia
Interventions: Procedure: Hematopoietic Stem Cell Transplant;   Procedure: Thymic Shielding During Radiation;   Procedure: Total Body Irradiation;   Drug: Cyclophosphamide, Fludarabine
8 Completed
Has Results
Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis
Condition: Cystinosis
Interventions: Drug: Cystagon® (Cysteamine Bitartrate);   Drug: Cysteamine Bitartrate Delayed-release Capsules (RP103)
9 Completed
Has Results
Study in Healthy Adults to Determine the Effect That Food Has on the Absorption and Delivery of the Drug Cystagon™
Conditions: Cystinosis;   Nephropathic Cystinosis
Intervention: Drug: Cysteamine bitartrate
10 Completed Gene Therapy for the Treatment of Fanconi's Anemia Type C
Conditions: Fanconi's Anemia;   Pancytopenia
Intervention: Drug: Transduced CD34+ Cells
11 Completed Oxandrolone for the Treatment of Bone Marrow Aplasia in Fanconi Anemia
Condition: Fanconi Anemia
Intervention: Drug: Oxandrolone
12 Enrolling by invitation Unrelated HSCT in Patients With Fanconi Anemia
Condition: Fanconi Anemia
Intervention: Biological: CD34+ selected cells
13 Available Use Massive Parallel Sequencing and Exome Capture Technology to Sequence the Exome of Fanconi Anemia Children and Their Patents
Conditions: Fanconi Anemia;   Autosomal or Sex Linked Recessive Genetic Disease;   Bone Marrow Hematopoiesis Failure, Multiple Congenital Abnormalities, and Susceptibility to Neoplastic Diseases.;   Hematopoiesis Maintainance.
Interventions: Genetic: human whole exome;   Genetic: whole genomic
14 Recruiting Gene Therapy for Fanconi Anemia
Condition: Fanconi Anemia
Interventions: Procedure: harvest procedure;   Biological: genetically engineered lymphocyte therapy;   Other: laboratory biomarker analysis
15 Terminated Donor Umbilical Cord Blood Transplant in Treating Patients With Hematologic Cancer
Conditions: Chronic Myeloproliferative Disorders;   Diamond-blackfan Anemia;   Fanconi Anemia;   Graft Versus Host Disease;   Leukemia;   Lymphoma;   Multiple Myeloma and Plasma Cell Neoplasm;   Myelodysplastic Syndromes;   Myelodysplastic/Myeloproliferative Diseases
Interventions: Drug: anti-thymocyte globulin;   Drug: busulfan;   Drug: cyclophosphamide;   Drug: cyclosporine;   Drug: filgrastim;   Drug: melphalan;   Drug: methylprednisolone;   Drug: mycophenolate mofetil;   Procedure: radiation therapy;   Procedure: umbilical cord blood transplantation
16 Suspended Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation
Conditions: Thalassemia;   Sickle Cell Disease;   Glanzmann Thrombasthenia;   Wiskott-Aldrich Syndrome;   Chronic-granulomatous Disease;   Severe Congenital Neutropenia;   Leukocyte Adhesion Deficiency;   Schwachman-Diamond Syndrome;   Diamond-Blackfan Anemia;   Fanconi Anemia;   Dyskeratosis-congenita;   Chediak-Higashi Syndrome;   Severe Aplastic Anemia
Intervention: Drug: Alefacept
17 Completed Cord Blood Stem Cell Transplantation Study (COBLT)
Conditions: Anemia, Aplastic;   Fanconi Anemia;   Hematologic Diseases;   Leukemia;   Neoplasms;   Severe Combined Immunodeficiency;   Hematopoietic Stem Cell Transplantation;   Myelodysplastic Syndromes
Intervention: Procedure: stem cell transplantation
18 Completed The Role of Susceptibility to Thrombosis in the Pseudotumor Cerebri of Nephropathic Cystinosis: A Case-Control Study
Conditions: Pseudotumor Cerebri;   Cystinosis
Intervention:
19 Unknown  Phase I Study of Amifostine in Patients With Bone Marrow Failure Related to Fanconi's Anemia
Condition: Fanconi's Anemia
Intervention: Drug: amifostine
20 Completed Hematopoietic Stem Cell Transplantation for Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using Total Body Irradiation, Cyclophosphamide and Fludarabine
Condition: Fanconi Anemia
Intervention: Drug: Cyclophosphamide and Fludarabine

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