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。） | 7 |
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 : 41 / 薬物数 : 37 - (DrugBank : 12) / 標的遺伝子数 : 13 - 標的パスウェイ数 : 44
| 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 | NCT03847909 (ClinicalTrials.gov) | October 28, 2019 | 15/2/2019 | A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2 | A Phase 2 Placebo-Controlled, Double-Blind, Multicenter Study to Evaluate the Efficacy, Safety, and Tolerability of DCR-PHXC Solution for Injection (Subcutaneous Use) in Patients With Primary Hyperoxaluria | Primary Hyperoxaluria Type 1 (PH1);Primary Hyperoxaluria Type 2 (PH2);Kidney Diseases;Urologic Diseases;Genetic Disease | Drug: DCR-PHXC;Drug: Sterile Normal Saline (0.9% NaCl) | Dicerna Pharmaceuticals, Inc. | NULL | Completed | 6 Years | N/A | All | 35 | Phase 2 | United States;Australia;Canada;France;Germany;Israel;Italy;Japan;Lebanon;Netherlands;New Zealand;Poland;Romania;Spain;United Kingdom |
| 2 | NCT04125472 (ClinicalTrials.gov) | October 15, 2019 | 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;Italy;Netherlands;Spain;France;French Polynesia;Germany;Switzerland;United Kingdom;United States | ||
| 3 | NCT04042402 (ClinicalTrials.gov) | July 9, 2019 | 10/7/2019 | Long Term Extension Study in Patients With Primary Hyperoxaluria | An Open-Label Roll-Over Study to Evaluate the Long-Term Safety and Efficacy of DCR-PHXC Solution for Injection (Subcutaneous Use) in Patients With Primary Hyperoxaluria | Primary Hyperoxaluria Type 1 (PH1);Primary Hyperoxaluria Type 2 (PH2);Kidney Diseases;Urologic Diseases;Genetic Disease | Drug: DCR-PHXC | Dicerna Pharmaceuticals, Inc. | NULL | Enrolling by invitation | 6 Years | N/A | All | 50 | Phase 3 | United States;Australia;Canada;France;Germany;Italy;Japan;Lebanon;Netherlands;Spain;United Kingdom |
| 4 | 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 |
| 5 | 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 Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG | Mucopolysaccharidosis Disorders;Hurler Syndrome;Hunter Syndrome;Maroteaux Lamy Syndrome;Sly Syndrome;Alpha-Mannosidosis;Fucosidosis;Aspartylglucosaminuria;Glycoprotein Metabolic Disorders;Sphingolipidoses;Recessive Leukodystrophies;Globoid Cell Leukodystrophy;Metachromatic Leukodystrophy;Niemann-Pick B;Niemann-Pick C Subtype 2;Sphingomyelin Deficiency;Peroxisomal Disorders;Adrenoleukodystrophy With Cerebral Involvement;Zellweger Syndrome;Neonatal Adrenoleukodystrophy;Infantile Refsum Disease;Acyl-CoA Oxidase Deficiency;D-Bifunctional Enzyme Deficiency;Multifunctional Enzyme Deficiency;Alpha-methylacyl-CoA Racmase Deficiency;Mitochondrial Neurogastrointestingal Encephalopathy;Severe Osteopetrosis;Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation);Inherited Metabolic Disorders | Biological: Stem Cell Transplantation;Drug: IMD Preparative Regimen;Drug: Osteopetrosis Only Preparative Regimen;Drug: Osteopetrosis Haploidentical Only Preparative Regimen;Drug: cALD SR-A (Standard-Risk, Regimen A);Drug: cALD SR-B (Standard-Risk, Regimen B);Drug: cALD HR-D (High-Risk, Regimen C);Drug: cALD HR-D (High-Risk, Regimen D) | Masonic Cancer Center, University of Minnesota | NULL | Recruiting | N/A | 55 Years | All | 100 | Phase 2 | United States |
| 6 | NCT01626092 (ClinicalTrials.gov) | July 11, 2012 | 20/6/2012 | Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders | Treatment of High Risk, Inherited Lysosomal and Peroxisomal Disorders by Reduced-Intensity Hematopoietic Cell Transplantation and Low-Dose Total Body Irradiation With Marrow Boosting by Volumetric-Modulated Arc Therapy (VMAT) | Lysosomal Storage Disease;Peroxisomal Disorder | Drug: Campath-1H;Drug: Clofarabine;Drug: Melphalan;Radiation: Total Body Irradiation with Marrow Boosting;Biological: Hematopoietic stem cell transplantation;Drug: Cyclosporine A;Drug: Mycophenolate mofetil | Masonic Cancer Center, University of Minnesota | NULL | Completed | N/A | 55 Years | All | 3 | N/A | United States |
| 7 | EUCTR2010-022046-25-NL (EUCTR) | 27/10/2010 | Cholic acid treatment in Peroxisomal Biogenesis Disorders (Zellweger spectrum): biochemical and clinical effects. - Cholic acid in Zellweger | Cholic acid treatment in Peroxisomal Biogenesis Disorders (Zellweger spectrum): biochemical and clinical effects. - Cholic acid in Zellweger | Peroxisomal biogenesis disorder Synonym: Zellweger spectrum disorderThis syndrome encompasses a spectrum of severity in symptomsPreviously the disorder was subdivided into three disorders based on the severity of symptoms; Infantile Refsum disease (IRD) is the mildest form, neonatal adrenoleucodystrofia (NALD) has more severe symptoms and Zellweger syndrome has the most severe clinical symptoms. MedDRA version: 12.1;Level: LLT;Classification code 10053706;Term: Zellweger syndrome MedDRA version: 12.1;Classification code 10038275;Term: Refsum's disease | Product Name: cholic acid Product Code: CA INN or Proposed INN: Cholic Acid Other descriptive name: CHOLIC ACID | Department of Pediatric Gastroenterology, Academic Medical Centre | NULL | NA | Female: yes Male: yes | Phase 2 | Netherlands |