234. Peroxisomal disease (except Adrenoleukodystrophy)
[
17 clinical trials,
28 drugs（DrugBank:
11 drugs),
11 target genes / 37 target pathways ]
Searched query = "Peroxisomal disease (except Adrenoleukodystrophy)", "Peroxisomal disease", "Peroxisomal disorder", "Peroxisome biogenesis disorder", "Zellweger syndr ... show all
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04152200 | November 2019 | 11 November 2019 | A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1 | ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1 (PH1) | Primary Hyperoxaluria Type 1;Primary Hyperoxaluria | Drug: Lumasiran | Alnylam Pharmaceuticals | Not recruiting | N/A | N/A | All | 16 | Phase 3 | ||
| 2 | NCT04125472 | October 15, 2019 | 28 October 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 | Not recruiting | 6 Years | N/A | All | N/A | |||
| 3 | NCT03847909 | October 2019 | 7 October 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. | Recruiting | 6 Years | N/A | All | 36 | Phase 2 | United States;Canada;France;New Zealand;Poland;United Kingdom | |
| 4 | NCT03905694 | April 22, 2019 | 22 October 2019 | A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1 | ILLUMINATE-B: An Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1 | Primary Hyperoxaluria;Primary Hyperoxaluria Type 1 (PH1) | Drug: Lumasiran | Alnylam Pharmaceuticals | Recruiting | N/A | 5 Years | All | 20 | Phase 3 | United States;France;Germany;Israel;United Kingdom | |
| 5 | NCT03856866 | January 11, 2019 | 11 March 2019 | Hydroxychloroquine Administration for Reduction of Pexophagy | Hydroxychloroquine Administration for Reduction of Pexophagy | Zellweger Syndrome;Peroxisome Biogenesis Disorders | Drug: Placebo;Drug: Hydroxychloroquine | The Hospital for Sick Children | Recruiting | 6 Months | 40 Years | All | 5 | Phase 2 | Canada | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | NCT03681184 | November 27, 2018 | 28 October 2019 | A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1 | ILLUMINATE-A: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study With an Extended Dosing Period to Evaluate the Efficacy and Safety of Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1 | Primary Hyperoxaluria Type 1 (PH1) | Drug: Lumasiran;Drug: Sterile Normal Saline (0.9% NaCl) | Alnylam Pharmaceuticals | Not recruiting | 6 Years | N/A | All | 30 | Phase 3 | United States;France;Germany;Israel;Netherlands;Switzerland;United Arab Emirates;United Kingdom | |
| 7 | EUCTR2018-001981-40-DE | 7 January 2019 | A Phase 3 Study of an Investigational Drug, Lumasiran (ALN-GO1) with an Extended Dosing Period in Children and Adults with Primary Hyperoxaluria Type 1 Disease | ILLUMINATE-A: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study with an Extended Dosing Period to Evaluate the Efficacy and Safety of Lumasiran in Children and Adults with Primary Hyperoxaluria Type 1 | Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1 Level: PT Classification code 10020703 Term: Hyperoxaluria System Organ Class: 10038359 - Renal and urinary disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: lumasiran Product Code: ALN-GO1 Pharmaceutical Form: Solution for injection INN or Proposed INN: lumasiran Current Sponsor code: ALN-GO1 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 189- Pharmaceutical form of the placebo: Solution for injection Route of administration of the placebo: Subcutaneous use | Alnylam Pharmaceuticals, Inc. | Not Available | Female: yes Male: yes | 30 | Phase 3 | France;United States;United Arab Emirates;Jordan;Israel;Netherlands;Germany;Japan;Switzerland | ||||
| 8 | EUCTR2016-003134-24-DE | 28/12/2017 | 30 April 2019 | Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1 | A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1 | Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1 Level: PT Classification code 10020703 Term: Hyperoxaluria System Organ Class: 10038359 - Renal and urinary disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: ALN-GO1 Product Code: ALN-GO1 Pharmaceutical Form: Solution for injection INN or Proposed INN: ALN-65585 CAS Number: 1834610-13-7 Other descriptive name: ALN-65585 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 200- | Alnylam Pharmaceuticals, Inc. | Authorised | Female: yes Male: yes | 20 | Phase 2 | France;United States;United Arab Emirates;Jordan;Israel;Netherlands;Germany;United Kingdom | |||
| 9 | NCT03350451 | November 20, 2017 | 28 October 2019 | An Extension Study of an Investigational Drug, ALN-GO1, in Patients With Primary Hyperoxaluria Type 1 | A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients With Primary Hyperoxaluria Type 1 | PH1;Primary Hyperoxaluria;RNAi Therapeutic;siRNA;AGT | Drug: ALN-GO1 | Alnylam Pharmaceuticals | Recruiting | 6 Years | N/A | All | 20 | Phase 2 | France;Germany;Israel;Netherlands;United Kingdom | |
| 10 | NCT02795325 | May 13, 2016 | 9 October 2018 | A Study of DCR-PH1 in Patients With Primary Hyperoxaluria Type 1 (PH1) | A Phase 1 Study of DCR-PH1 in Patients With Primary Hyperoxaluria Type 1 (PH1) | Primary Hyperoxaluria Type 1 | Drug: DCR-PH1;Other: Placebo | Dicerna Pharmaceuticals, Inc. | Not recruiting | 12 Years | N/A | All | 41 | Phase 1 | Germany;Netherlands | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 11 | EUCTR2015-003142-51-GB | 11/04/2016 | 25 April 2016 | An Early Phase Study of DCR-PH1 in Patients with an Inherited Disorder Resulting in Overproduction of Oxalate | A Phase 1 Study of DCR-PH1 in Patients with Primary Hyperoxaluria Type 1 (PH1) | Primary Hyperoxaluria Type 1 MedDRA version: 18.0 Level: PT Classification code 10020703 Term: Hyperoxaluria System Organ Class: 10038359 - Renal and urinary disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: DCR-PH1 Pharmaceutical Form: Concentrate for solution for injection INN or Proposed INN: Not Yet Assigned CAS Number: NA Current Sponsor code: DCR-1171X Other descriptive name: DCR-1171X Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 2.0- | Dicerna Pharmaceuticals, Inc. | Authorised | Female: yes Male: yes | 42 | Phase 1 | France;United States;Israel;Netherlands;Germany;United Kingdom | |||
| 12 | EUCTR2015-004407-23-GB | 10/02/2016 | 7 January 2019 | The First-in-Human Study of an Investigational Drug, ALN-GO1, in Healthy Adult Subjects and Patients with Primary Hyperoxaluria Type 1 Disease | A Phase 1/2, Single-Blind, Placebo-Controlled, Single and Multiple Ascending Dose Safety, Tolerability, Pharmacokinetic and Pharmacodynamics Study of Subcutaneously Administered ALN-GO1 in Healthy Adult Subjects, and Patients with Primary Hyperoxaluria Type 1 | Primary Hyperoxaluria Type 1 (PH1) MedDRA version: 20.1 Level: PT Classification code 10020703 Term: Hyperoxaluria System Organ Class: 10038359 - Renal and urinary disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: ALN-GO1 Product Code: ALN-GO1 Pharmaceutical Form: Solution for injection INN or Proposed INN: ALN-65585 Current Sponsor code: ALN-65585 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 200- Pharmaceutical form of the placebo: Solution for injection Route of administration of the placebo: Subcutaneous use | Alnylam Pharmaceuticals, Inc. | Authorised | Female: yes Male: yes | 52 | Phase 1;Phase 2 | France;United States;Jordan;Israel;Netherlands;Germany;United Kingdom | |||
| 13 | NCT02171104 | July 10, 2014 | 7 October 2019 | 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 | Recruiting | N/A | 55 Years | All | 100 | Phase 2 | United States | |
| 14 | NCT01626092 | July 11, 2012 | 16 December 2017 | 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 | Not recruiting | N/A | 55 Years | All | 3 | N/A | United States | |
| 15 | EUCTR2010-022046-25-NL | 2 October 2017 | 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 disorder This syndrome encompasses a spectrum of severity in symptoms Previously 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 Level: LLT Classification code 10038275 Term: Refsum's disease | Product Name: cholic acid Product Code: CA Pharmaceutical Form: Capsule* INN or Proposed INN: Cholic Acid CAS Number: 81254 Current Sponsor code: CA Other descriptive name: CHOLIC ACID Concentration unit: mg/kg milligram(s)/kilogram Concentration type: up to Concentration number: 10-20 | Department of Pediatric Gastroenterology, Academic Medical Centre | Not Available | Female: yes Male: yes | Phase 2 | Netherlands | |||||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 16 | NCT00004442 | September 1997 | 7 April 2015 | Study of Bile Acids in Patients With Peroxisomal Disorders | Infantile Refsum's Disease;Zellweger Syndrome;Bifunctional Enzyme Deficiency;Adrenoleukodystrophy | Drug: chenodeoxycholic acid;Drug: cholic acid;Drug: ursodiol | Children's Hospital Research Foundation University of Cincinnati | Children's Hospital Medical Center, Cincinnati | Not recruiting | N/A | 5 Years | Both | 25 | N/A | ||
| 17 | NCT00007020 | January 1992 | 19 February 2015 | Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid | Investigation in the Pathogenesis of Liver Disease in Patients With Inborn Errors of Bile Acid Metabolism. This Study Was Previously Registered by the NCRR and Identified as NCRR-M01RR08084-0009 | Infantile Refsum's Disease;Zellweger Syndrome;Adrenoleukodystrophy;Peroxisomal Disorders;Cholestasis | Drug: Cholic Acids | Asklepion Pharmaceuticals, LLC | Children's Hospital Medical Center, Cincinnati | Not recruiting | N/A | N/A | Both | 79 | Phase 3 | United States |