| Status | Study |
| Recruiting |
Study Name: Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype A Condition: Fanconi Anemia Date: 2017-04-26 Interventions: Procedure: IV administration of Genetically Engineered Hematopoietic Stem/Pr |
| Recruiting |
Study Name: Familial Investigations of Childhood Cancer Predisposition Condition: Acute Leukemia Adenomatous Polyposis Adrenocortical Carcin Date: 2017-02-08 |
| Recruiting |
Study Name: 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 Date: 2016-09-06 Interventions: Drug: filgrastim G-CSF (12 |
| Recruiting |
Study Name: Investigation of the Genetics of Hematologic Diseases Condition: Bone Marrow Failure Syndromes Erythrocyte Disorder Leukocy Date: 2016-03-16 |
| Recruiting |
Study Name: Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and Plerixafor Condition: Fanconi Anemia Date: 2016-02-05 Interventions: Drug: G-CSF D1 to D4 : Injecti |
| Terminated |
Study Name: Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells Condition: Stem Cell Transplantation Bone Marrow Transplantation Peri Date: 2015-05-21 Interventions: Drug: Cyclophosphamide Dose Level 1 |
| Recruiting |
Study Name: L-leucine in Diamond Blackfan Anemia Patients Condition: Diamond Blackfan Anemia Date: 2015-02-18 Interventions: Drug: L-leucine L-leucine pills per os for 6 months |
| Recruiting |
Study Name: Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies Condition: Sickle Cell Disease Transfusion Dependent Alpha- or Beta- Thalassemia Date: 2014-06-27 Interventions: Drug: Reduced Toxicity Ablative Regimen |
| Recruiting |
Study Name: HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy Condition: Fanconi Anemia Severe Marrow Failure Myelodysplastic Syndr Date: 2014-04-28 Interventions: Drug: Busulfan |
| Available |
Study Name: Use Massive Parallel Sequencing and Exome Capture Technology to Sequence the Exome of Fanconi Anemia Children and Their Patents Condition: Fanconi Anemia Autosomal or Sex Linked Recessive Genetic Disease Date: 2013-11-21 Interventions: Genetic: human whole exome he |