| 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: 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: 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 |
| 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 |
| 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 |
| Enrolling by invitation |
Study Name: Unrelated HSCT in Patients With Fanconi Anemia Condition: Fanconi Anemia Date: 2012-10-19 Interventions: Biological: CD34+ selected cells Compassionate treatment of Fanconi Anemia patients with unrelated bone |
| Recruiting |
Study Name: Quercetin in Children With Fanconi Anemia; a Pilot Study Condition: Fanconi Anemia Date: 2012-07-25 Interventions: Drug: Quercetin (dietary supplement) Quercetin will be given orally on a twice a day schedule starting w |
| Recruiting |
Study Name: Microcephaly Genetic Deficiency in Neural Progenitors Condition: Microcephaly Date: 2012-02-29 |
| Recruiting |
Study Name: Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia Condition: Diamond Blackfan Anemia Date: 2011-10-31 Interventions: Drug: Sotatercept |
| Active, not recruiting |
Study Name: Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia Condition: Diamond Blackfan Anemia Blackfan Diamond Syndrome DBA Date: 2011-05-20 Interventions: Drug: leucine Dosage of leucine will be dependent on body |