Biopsy ( DrugBank: - )

4 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2
51全身性強皮症3
78下垂体前葉機能低下症2
193プラダー・ウィリ症候群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
2NCT02841358
(ClinicalTrials.gov)		December 201311/7/2016Screening of Niemann-Pick Disease, Type C in a Psychiatric PopulationStudy Qbout the Screening of Niemann-Pick Disease, Type C in a Psychiatric PopulationPsychiatric Adults PatientsBiological: Blood sampling;Biological: BiopsyUniversity Hospital, GrenobleNULLTerminated18 YearsN/AAll22N/AFrance

51. 全身性強皮症

臨床試験数 : 523 /  薬物数 : 608 - (DrugBank : 156) / 標的遺伝子数 : 114 - 標的パスウェイ数 : 215
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT04265144
(ClinicalTrials.gov)		June 8, 20205/2/2020Cohort of Patients With Systemic Sclerosis Within the Framework of the RESO Reference CentreCohort of Patients With Systemic Sclerosis and Associated Biological Collection Within the Framework of the RESO Reference Centre for Rare Systemic Autoimmune DiseasesScleroderma;Systemic SclerosisBiological: Blood samples;Other: Biopsy;Other: Bronchoalveolar samplesUniversity Hospital, BordeauxNULLRecruiting18 YearsN/AAll500N/AFrance
2NCT02562079
(ClinicalTrials.gov)		March 201229/12/2014Vasculopathy, Inflammation and Systemic SclerosisVasculopathy, Inflammation and Systemic Sclerosis: The Role of Endothelial Cell Activation and OX40/OX40L in Modulation of T Lymphocyte ActivationSystemic SclerosisBiological: Blood samples;Biological: BiopsyUniversity Hospital, BordeauxSociété Française de RhumatologieCompleted18 Years75 YearsAll350N/AFrance
3NCT00622895
(ClinicalTrials.gov)		September 1, 200622/2/2008Allogeneic Hematopoietic Cell Transplantation for Severe Systemic SclerosisAllogeneic Hematopoietic Cell Transplantation After Nonmyeloablative Conditioning for Patients With Severe Systemic SclerosisSystemic Scleroderma;Severe Systemic SclerosisDrug: fludarabine phosphate;Drug: Mycophenolic Acid;Drug: tacrolimus;Radiation: total-body irradiation;Procedure: bone marrow transplantation;Procedure: reduced intensity allogeneic hematopoietic stem cell transplantation;Procedure: quality-of-life assessment;Other: laboratory biomarker analysis;Other: flow cytometry;Procedure: biopsyFred Hutchinson Cancer Research CenterNational Institute of Allergy and Infectious Diseases (NIAID)CompletedN/A70 YearsAll3Phase 1/Phase 2United States

78. 下垂体前葉機能低下症

臨床試験数 : 494 /  薬物数 : 385 - (DrugBank : 49) / 標的遺伝子数 : 44 - 標的パスウェイ数 : 100
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT01298180
(ClinicalTrials.gov)		January 20096/11/2009Is There a Sensibility Increased in the Growth Hormone at Child With Prader-Willi Syndrome?Is There a Sensibility Increased in the Growth Hormone at Child With Prader-Willi Syndrome?Prader-Willi Syndrome;Growth Hormone DeficiencyDrug: Growth hormone (Genotonorm® or Omnitrope®);Procedure: DEXA, blood tests, H.G.P.O, osseous age.;Procedure: biopsyUniversity Hospital, ToulouseNULLCompleted1 Year5 YearsAll111Phase 4France
2NCT00720902
(ClinicalTrials.gov)		February 200716/6/2008Adult Growth Hormone Deficiency and Cardiovascular RiskAssessment of Cardiovascular Risk Markers in GH Deficient Patients With Nonsecreting Pituitary AdenomasGrowth Hormone DeficiencyProcedure: Blood draws;Drug: Growth hormone releasing hormone (GHRH) and arginine;Procedure: Carotid ultrasound;Procedure: MRI;Procedure: Endothelial cell biopsyColumbia UniversityNULLTerminated19 Years65 YearsBoth8N/AUnited States

193. プラダー・ウィリ症候群

臨床試験数 : 111 /  薬物数 : 120 - (DrugBank : 30) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 103
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT01298180
(ClinicalTrials.gov)		January 20096/11/2009Is There a Sensibility Increased in the Growth Hormone at Child With Prader-Willi Syndrome?Is There a Sensibility Increased in the Growth Hormone at Child With Prader-Willi Syndrome?Prader-Willi Syndrome;Growth Hormone DeficiencyDrug: Growth hormone (Genotonorm® or Omnitrope®);Procedure: DEXA, blood tests, H.G.P.O, osseous age.;Procedure: biopsyUniversity Hospital, ToulouseNULLCompleted1 Year5 YearsAll111Phase 4France

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