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 |
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 |
Completed |
Study Name: Study in Healthy Adults to Determine the Effect That Food Has on the Absorption and Delivery of the Drug Cystagon™ Condition: Cystinosis Nephropathic Cystinosis Date: 2011-09-09 Interventions: Drug: Cysteamine bitartrate 500 mg total, single dose taken orally on visits 2, 3 & 4 which must occ |
Recruiting |
Study Name: Cure Cystinosis International Registry Condition: Cystinosis Nephropathic Cystinosis Renal Date: 2011-03-29 |
Recruiting |
Study Name: Gene Therapy for Fanconi Anemia Condition: Fanconi Anemia Date: 2011-03-16 Interventions: Procedure: Bone Marrow Aspiration |
Completed |
Study Name: Multicenter Transplant Study for Fanconi Anemia Condition: Fanconi Anemia Date: 2010-03-05 Interventions: Drug: Chemotherapy Busulfan 0. |
Completed |
Study Name: Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis Condition: Cystinosis Date: 2009-10-22 Interventions: Drug: Cystagon® (Cysteamine Bitartrate) |