Biopsy ( DrugBank: - )


4 diseases
IDDisease name (Link within this page)Number of trials
19Lysosomal storage disease2
51Scleroderma3
78Hypopituitarism2
193Prader-Willi syndrome1

19. Lysosomal storage disease


Clinical trials : 854 Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 191
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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Status
Inclusion_
agemin
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agemax
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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. Scleroderma


Clinical trials : 523 Drugs : 608 - (DrugBank : 156) / Drug target genes : 114 - Drug target pathways : 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. Hypopituitarism


Clinical trials : 494 Drugs : 385 - (DrugBank : 49) / Drug target genes : 44 - Drug target pathways : 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. Prader-Willi syndrome


Clinical trials : 111 Drugs : 120 - (DrugBank : 30) / Drug target genes : 51 - Drug target pathways : 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