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Anemia, Hypoplastic, Congenital - 25 Studies Found

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
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