Blood draw ( DrugBank: - )

5 diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
6	パーキンソン病	1
19	ライソゾーム病	1
65	原発性免疫不全症候群	3
231	α1−アンチトリプシン欠乏症	2
299	嚢胞性線維症	2

6. パーキンソン病

臨床試験数 : 2,298 / 薬物数 : 2,202 - (DrugBank : 350) / 標的遺伝子数 : 188 - 標的パスウェイ数 : 202

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT01882010 (ClinicalTrials.gov)	September 2013	13/6/2013	Leukine (Sargramostim) for Parkinson's Disease	Leukine (Sargramostim) for Parkinson's Disease	Parkinson's Disease	Procedure: blood draw;Procedure: physical exam and UPDRS part III assessment;Procedure: MEG;Drug: sargramostim;Drug: placebo;Behavioral: physical exam and UPDRS part III assessment;Procedure: Motion Analysis	Howard Gendelman, MD	Sanofi;National Institute of Neurological Disorders and Stroke (NINDS);UNeMed;Nebraska Neuroscience Alliance	Completed	35 Years	85 Years	Both	37	Phase 1	United States

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.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT04002531 (ClinicalTrials.gov)	November 10, 2018	25/3/2019	A One Visit Follow Up of Adults With Fabry Disease Who Started Long-term Enzyme Replacement Therapy As Children	A One Visit Follow Up of Adults With Fabry Disease Who Started Long-term Enzyme Replacement Therapy As Children	Quality of Life;Renal Insufficiency;Cardiac Event	Other: General and Neurological examination;Other: Vital signs;Procedure: 12 lead electrocardiogram;Procedure: Echocardiogram;Procedure: Blood draw;Procedure: Urine collection;Procedure: 2-hour Holter Monitor;Other: Brief Pain Inventory questionnaire;Other: Quality of Life questionnaire	Baylor Research Institute	Shire	Enrolling by invitation	18 Years	N/A	All	12	N/A	United States

65. 原発性免疫不全症候群

臨床試験数 : 482 / 薬物数 : 653 - (DrugBank : 119) / 標的遺伝子数 : 92 - 標的パスウェイ数 : 212

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT01220531 (ClinicalTrials.gov)	November 8, 2010	22/9/2010	Thymus Transplantation Safety-Efficacy	Safety and Efficacy of Thymus Transplantation in Complete DiGeorge Anomaly, IND#9836	Complete DiGeorge Anomaly;DiGeorge Syndrome;DiGeorge Anomaly;Complete DiGeorge Syndrome	Biological: Cultured Thymus Tissue;Procedure: Blood Draw;Drug: Rabbit anti-thymocyte globulin;Drug: Cyclosporine;Drug: Tacrolimus;Drug: Methylprednisolone or Prednisolone;Drug: Mycophenolate mofetil	M. Louise Markert	National Institutes of Health (NIH);National Institute of Allergy and Infectious Diseases (NIAID);Enzyvant Therapeutics GmbH	Available	N/A	N/A	All			United States
2	NCT00909363 (ClinicalTrials.gov)	June 2009	27/5/2009	Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients	Effects Of Eltrombopag On Thrombocytopenia, Platelet Function and Bleeding In Patients With Wiskott-Aldrich Syndrome/X-Linked Thrombocytopenia.	Wiskott-Aldrich Syndrome;Thrombocytopenia;Bleeding	Drug: Promacta;Diagnostic Test: blood drawing in patients with WAS;Diagnostic Test: blood drawing in healthy controls	Weill Medical College of Cornell University	Novartis Pharmaceuticals	Terminated	3 Months	80 Years	Male	24	Phase 2	United States
3	NCT00579527 (ClinicalTrials.gov)	December 19, 2005	20/12/2007	Phase I/II Thymus Transplantation With Immunosuppression #950	Phase I/II Trial of Thymus Transplantation With Immunosuppression, #950	DiGeorge Anomaly;Complete DiGeorge Anomaly;Complete Atypical DiGeorge Anomaly;Complete DiGeorge Syndrome;Complete Atypical DiGeorge Syndrome	Biological: Cultured Thymus Tissue for Implantation (CTTI);Other: Cultured Thymus Tissue Implantation and Parental Parathyroid Transplantation;Procedure: Blood Draw;Drug: Rabbit anti-thymocyte globulin;Drug: Cyclosporine;Drug: Tacrolimus;Drug: Methylprednisolone or Prednisolone;Drug: Daclizumab;Drug: Mycophenolate mofetil	M. Louise Markert	National Institutes of Health (NIH);National Institute of Allergy and Infectious Diseases (NIAID);Enzyvant Therapeutics GmbH;Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	Completed	N/A	N/A	All	14	Phase 1/Phase 2	United States

231. α1−アンチトリプシン欠乏症

臨床試験数 : 89 / 薬物数 : 89 - (DrugBank : 16) / 標的遺伝子数 : 35 - 標的パスウェイ数 : 47

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT01810458 (ClinicalTrials.gov)	October 2013	6/3/2013	Liver Fibrosis in Alpha-1 Antitrypsin Deficiency (Liver AATD)	Clinical Predictors and Epigenetic Markers for Liver Fibrosis in Alpha-1 Antitrypsin Deficiency	Liver Fibrosis;Alpha-1 Antitrypsin Deficiency;AAT Deficiency;AATD	Device: Abdominal ultrasound;Procedure: History and physical;Procedure: Intravenous catheter;Procedure: Blood draw;Other: Liver questionnaire;Procedure: Liver Biopsy;Drug: Midazolam;Drug: Fentanyl;Drug: Lidocaine;Drug: Acetaminophen;Drug: Lorazepam;Drug: Oxycodone/Acetaminophen;Drug: Ondansetron	University of Florida	NULL	Completed	18 Years	70 Years	All	109		United States
2	NCT01851642 (ClinicalTrials.gov)	September 2007	25/4/2013	Lung Disease and Its Affect on the Work of White Blood Cells in the Lungs	The Role of Conformational Diseases on Macrophage Function	Alpha-1 Antitrypsin Deficiency;AAT Deficiency;AATD;Cystic Fibrosis (CF)	Procedure: History and physical exam.;Procedure: Blood draw.;Procedure: Pulmonary function testing.;Drug: Albuterol inhaler.	University of Florida	NULL	Recruiting	18 Years	N/A	All	220		United States

299. 嚢胞性線維症

臨床試験数 : 1,696 / 薬物数 : 1,644 - (DrugBank : 272) / 標的遺伝子数 : 96 - 標的パスウェイ数 : 170

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT02589444 (ClinicalTrials.gov)	December 2015	20/10/2015	Vitamin D and Microbiota in Cystic Fibrosis	Pilot Study Evaluating the Role of Vitamin D Repletion on Gut and Lung Microbiota in Cystic Fibrosis	Vitamin D Deficiency;Cystic Fibrosis	Dietary Supplement: High-Dose Vitamin D3;Other: Stool Sample;Other: Sputum Sample;Other: Sham Comparator;Procedure: Blood draw	Emory University	NULL	Completed	18 Years	N/A	All	41	N/A	United States
2	NCT01851642 (ClinicalTrials.gov)	September 2007	25/4/2013	Lung Disease and Its Affect on the Work of White Blood Cells in the Lungs	The Role of Conformational Diseases on Macrophage Function	Alpha-1 Antitrypsin Deficiency;AAT Deficiency;AATD;Cystic Fibrosis (CF)	Procedure: History and physical exam.;Procedure: Blood draw.;Procedure: Pulmonary function testing.;Drug: Albuterol inhaler.	University of Florida	NULL	Recruiting	18 Years	N/A	All	220		United States

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