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61 studies found for:    "Toni-Debre-Fanconi syndrome"
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Rank Status Study
1 Completed Evaluation of Fanconi Syndrome and Cystinosis
Conditions: Cystinosis;   Fanconi Syndrome
Intervention:
2 Completed Fanconi Syndrome Due to ARVs in HIV-Infected Persons
Conditions: HIV Infections;   Fanconi Syndrome;   Kidney Disease;   Renal Impairment
Intervention: Procedure: Blood Draws
3 Recruiting Cure Cystinosis International Registry
Conditions: Cystinosis;   Nephropathic Cystinosis;   Renal Fanconi Syndrome
Intervention:
4 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
5 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
6 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)
7 Completed
Has Results
Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis
Condition: Cystinosis
Interventions: Drug: Cystagon®;   Drug: RP103
8 Recruiting Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype A
Condition: Fanconi Anemia
Interventions: Procedure: IV administration of Genetically Engineered Hematopoietic Stem/Progenitors Cells (HSPCs);   Biological: Genetically Engineered Hematopoietic Stem/Progenitor Cells;   Other: Laboratory Biomarker Analysis;   Biological: Filgrastim;   Drug: Plerixafor;   Procedure: Bone Marrow Aspiration
9 Recruiting Clinical Phase II Trial to Evaluate CD34+ Cells Mobilization and Collection in Patients With Fanconi Anemia for Subsequent Transduction With a Lentiviral Vector Carring FANCA Gene. FANCOSTEM-1
Condition: Fanconi Anemia
Interventions: Drug: filgrastim;   Drug: plerixafor
10 Recruiting Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and Plerixafor
Condition: Fanconi Anemia
Interventions: Drug: G-CSF;   Drug: Plerixafor
11 Enrolling by invitation Unrelated HSCT in Patients With Fanconi Anemia
Condition: Fanconi Anemia
Intervention: Biological: CD34+ selected cells
12 Recruiting Quercetin in Children With Fanconi Anemia; a Pilot Study
Condition: Fanconi Anemia
Intervention: Drug: Quercetin (dietary supplement)
13 Recruiting Gene Therapy for Fanconi Anemia
Condition: Fanconi Anemia
Interventions: Procedure: Bone Marrow Aspiration;   Biological: Filgrastim;   Biological: Genetically Engineered Hematopoietic Stem Progenitor Cells;   Other: Laboratory Biomarker Analysis;   Drug: Methylprednisolone;   Drug: Plerixafor;   Drug: Prednisone
14 Completed Multicenter Transplant Study for Fanconi Anemia
Condition: Fanconi Anemia
Interventions: Drug: Chemotherapy;   Biological: Miltenyi CliniMACS
15 Active, not recruiting Hematopoietic Stem Cell Transplant for Fanconi Anemia
Condition: Fanconi Anemia
Intervention: Device: CliniMACs device
16 Completed Pilot Study of Etanercept (Enbrel) in Children With Fanconi Anemia
Condition: Fanconi Anemia
Intervention: Drug: Etanercept
17 Terminated Gene Function in Bone Marrow Cells From Patients With Fanconi Anemia and From Healthy Participants
Condition: Fanconi Anemia
Interventions: Genetic: microarray analysis;   Procedure: biopsy
18 Recruiting Cytoxan, Fludara, and Antithymocyte Globulin Conditioning Followed By Stem Cell Transplant in Treating Fanconi Anemia
Condition: Fanconi Anemia
Interventions: Biological: Anti-Thymocyte Globulin;   Drug: Cyclophosphamide;   Drug: Fludarabine;   Procedure: Hematopoietic Stem Cell Transplantation;   Drug: Methylprednisolone;   Drug: Filgrastim;   Drug: Cyclosporine;   Drug: Mycophenolate Mofetil
19 Completed
Has Results
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
Interventions: Drug: Cyclophosphamide;   Drug: Fludarabine;   Radiation: Total body irradiation (TBI)
20 Terminated Use of Rft5-Dga to Deplete Alloreactive Cells for Pts With Fanconi Anemia After Haploidentical SCT
Condition: FANCONI ANEMIA
Interventions: Procedure: CD34 selected haploidentical PBSCT;   Drug: Fludarabine;   Biological: T cell infusion;   Biological: Campath 1h;   Biological: anti-CD45

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Study has passed its completion date and status has not been verified in more than two years.