CA ( DrugBank: - )

1 disease

告示番号	疾患名（ページ内リンク）	臨床試験数
234	ペルオキシソーム病（副腎白質ジストロフィーを除く。）	6

234. ペルオキシソーム病（副腎白質ジストロフィーを除く。）

臨床試験数 : 39 / 薬物数 : 35 - (DrugBank : 12) / 標的遺伝子数 : 13 - 標的パスウェイ数 : 45

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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	NCT05001269 (ClinicalTrials.gov)	February 22, 2022	30/6/2021	Nedosiran in Pediatric Patients From Birth to 11 Years of Age With PH and Relatively Intact Renal Fu ...	A Phase 2 Open-Label Multicenter Study to Evaluate the Safety, Pharmacokinetics, and Efficacy of Ned ...	Primary Hyperoxaluria;Primary Hyperoxaluria Type 1;Primary Hyperoxaluria Type 2;Primary Hyperoxaluri ...	Drug: nedosiran	Dicerna Pharmaceuticals, Inc.	NULL	Recruiting	N/A	11 Years	All	20	Phase 2	United States;Canada;France;Germany;Italy;Japan;Lebanon;Morocco;Poland;Spain;Turkey;United Arab Emir ...
2	NCT04125472 (ClinicalTrials.gov)	June 2, 2021	10/10/2019	Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1	Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1	Primary Hyperoxaluria	Drug: Lumasiran	Alnylam Pharmaceuticals	NULL	Approved for marketing	N/A	N/A	All			Belgium;Canada;France;French Polynesia;Germany;Italy;Netherlands;Spain;Switzerland;United Kingdom;Un ...
3	NCT03856866 (ClinicalTrials.gov)	January 11, 2019	5/11/2018	Hydroxychloroquine Administration for Reduction of Pexophagy	Hydroxychloroquine Administration for Reduction of Pexophagy	Zellweger Syndrome;Peroxisome Biogenesis Disorders	Drug: Hydroxychloroquine;Drug: Placebo	The Hospital for Sick Children	NULL	Recruiting	6 Months	40 Years	All	5	Phase 2	Canada
4	NCT02171104 (ClinicalTrials.gov)	July 10, 2014	20/6/2014	MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis	MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Sever ...	Mucopolysaccharidosis Disorders;Hurler Syndrome;Hunter Syndrome;Maroteaux Lamy Syndrome;Sly Syndrome ...	Biological: Stem Cell Transplantation;Drug: IMD Preparative Regimen;Drug: Osteopetrosis Only Prepara ...	Masonic Cancer Center, University of Minnesota	NULL	Recruiting	N/A	55 Years	All	100	Phase 2	United States
5	NCT01626092 (ClinicalTrials.gov)	July 11, 2012	20/6/2012	Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorde ...	Treatment of High Risk, Inherited Lysosomal and Peroxisomal Disorders by Reduced-Intensity Hematopoi ...	Lysosomal Storage Disease;Peroxisomal Disorder	Drug: Campath-1H;Drug: Clofarabine;Drug: Melphalan;Radiation: Total Body Irradiation with Marrow Boo ...	Masonic Cancer Center, University of Minnesota	NULL	Completed	N/A	55 Years	All	3	N/A	United States
6	EUCTR2010-022046-25-NL (EUCTR)		27/10/2010	Cholic acid treatment in Peroxisomal Biogenesis Disorders (Zellweger spectrum): biochemical and clin ...	Cholic acid treatment in Peroxisomal Biogenesis Disorders (Zellweger spectrum): biochemical and clin ...	Peroxisomal biogenesis disorder Synonym: Zellweger spectrum disorderThis syndrome encompasses a spec ...	Product Name: cholic acid Product Code: CA INN or Proposed INN: Cholic Acid Other descriptive name: CHO ...	Department of Pediatric Gastroenterology, Academic Medical Centre	NULL	NA			Female: yes Male: yes		Phase 2	Netherlands

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