19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details


臨床試験数 : 854 薬物数 : 727 - (DrugBank : 105) / 標的遺伝子数 : 69 - 標的パスウェイ数 : 190

  
2 trials found
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1NCT04411654
(ClinicalTrials.gov)
June 29, 202111/5/2020Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)An Open-label, Phase 1/2 Study to Evaluate the Safety and Efficacy of Single-dose PR001A in Infants With Type 2 Gaucher DiseaseGaucher Disease, Type 2Biological: PR001;Drug: Methylprednisolone;Drug: Sirolimus;Drug: PrednisonePrevail TherapeuticsEli Lilly and CompanyRecruitingN/A24 MonthsAll15Phase 1/Phase 2United States;United Kingdom
2NCT03952637
(ClinicalTrials.gov)
August 19, 201915/5/2019A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 GangliosidosisA Phase 1-2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 GangliosidosisLysosomal Diseases;Gangliosidosis;GM1Biological: AAV9-GLB1;Drug: Rituximab;Drug: Sirolimus;Drug: Methylprednisolone;Drug: Prednisone;Diagnostic Test: Audiology assessmentwith ABR;Diagnostic Test: Bone density scan (DEXA;Diagnostic Test: Electrocardiogram (EKG);Diagnostic Test: Echocardiogram;Other: Electroencephalogram (EEG) awake andextended overnight;Diagnostic Test: Laboratory tests;Procedure: Lumbar puncture;Procedure: Brain MRI/MRS/fMRI;Behavioral: Neurocognitive testing;Other: Neurology exam;Behavioral: PICC line placement;Procedure: Skeletal survey;Procedure: Skin biopsy;Procedure: Speech and modified barium swallow study;Procedure: Ophthalmology examNational Human Genome Research Institute (NHGRI)Axovant Sciences, Inc.;Sio Gene Therapies (Sponsor)Recruiting6 Months12 MonthsAll45Phase 1/Phase 2United States