20. 副腎白質ジストロフィー Adrenoleukodystrophy Clinical trials / Disease details


臨床試験数 : 61 薬物数 : 90 - (DrugBank : 31) / 標的遺伝子数 : 23 - 標的パスウェイ数 : 126

  
1 trial found
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1NCT02559830
(ClinicalTrials.gov)
January 201512/8/2015Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and AdrenoleukodystrophyA Phase I/II Clinical Trial of Lentiviral Hematopoietic Stem Cell Gene Therapy for Treatment of Developed Metachromatic Leukodystrophy and AdrenoleukodystrophyMetachromatic Leukodystrophy;AdrenoleukodystrophyGenetic: transduced CD34+ hematopoietic stem cellShenzhen Second People's HospitalShenzhen University;Guangzhou Women and Children's Medical CenterRecruiting1 Year16 YearsAll50Phase 1/Phase 2China