Skin biopsy ( DrugBank: - )


2 diseases
告示番号疾患名(ページ内リンク)臨床試験数
19ライソゾーム病1
84サルコイドーシス1

19. ライソゾーム病


臨床試験数 : 854 薬物数 : 716 - (DrugBank : 105) / 標的遺伝子数 : 70 - 標的パスウェイ数 : 191
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03952637
(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

84. サルコイドーシス


臨床試験数 : 149 薬物数 : 227 - (DrugBank : 81) / 標的遺伝子数 : 82 - 標的パスウェイ数 : 167
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT02134717
(ClinicalTrials.gov)
January 201430/4/2014Impact of Chemokine Receptor 5 (CCR5) Inhibition on Sarcoidosis ImmunophenotypesAn Interventional Study of the Effect of CCR5 Inhibition With Maraviroc on Immune Cells in the the Lung and in Peripheral Blood of Patients With SarcoidosisSarcoidosisDrug: all subjects will receive maraviroc 300mg orally twice a day for 6 weeks;Procedure: Bronchoscopy with bronchoalveolar lavage;Procedure: venipunctures;Procedure: Skin biopsyKevin F. GibsonUniversity of PittsburghTerminated18 Years80 YearsAll3N/AUnited States