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 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:17,薬物数:28(DrugBank:11),標的遺伝子数:11,標的パスウェイ数:11] 

Searched query = "Peroxisomal disease (except Adrenoleukodystrophy)", "Peroxisomal disease", "Peroxisomal disorder", "Peroxisome biogenesis disorder", "Zellweger syndrome", "PEX gene disorder", "Neonatal adrenoleukodystrophy", "Rhizomelic chondrodysplasia punctata type 1", "RCDP type 1", "RCDP1", "Peroxisomal beta-oxidation enzyme deficiency", "Acyl-CoA oxidase deficiency", "AOX deficiency", "D-Bifunctional protein deficiency", "DBP deficiency", "Sterol carrier protein X deficiency", "SCPx deficiency", "2-methylacyl-CoA racemase deficiency", "Alpha-methylacyl-CoA racemase deficiency", "AMACR deficiency", "Refsum disease", "Plasmalogen biosynthesis enzyme deficiency", "Rhizomelic chondrodysplasia punctata type 2", "RCDP type 2", "RCDP2", "Rhizomelic chondrodysplasia punctata type 3", "RCDP type 3", "RCDP3", "Primary hyperoxaluria type 1", "Acatalasemia", "Acatalasia", "Contiguous ABCD1/DXS1357E deletion syndrome", "CADDS"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"    Synonyms (DrugBank) were also searched for.
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04152200November 201911 November 2019A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1ILLUMINATE-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 HyperoxaluriaDrug: LumasiranAlnylam PharmaceuticalsNot recruitingN/AN/AAll16Phase 3
2NCT04125472October 15, 201928 October 2019Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1Primary HyperoxaluriaDrug: LumasiranAlnylam PharmaceuticalsNot recruiting6 YearsN/AAllN/A
3NCT03847909October 20197 October 2019A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2A 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 HyperoxaluriaPrimary Hyperoxaluria Type 1 (PH1);Primary Hyperoxaluria Type 2 (PH2);Kidney Diseases;Urologic Diseases;Genetic DiseaseDrug: DCR-PHXC;Drug: Sterile Normal Saline (0.9% NaCl)Dicerna Pharmaceuticals, Inc.Recruiting6 YearsN/AAll36Phase 2United States;Canada;France;New Zealand;Poland;United Kingdom
4NCT03905694April 22, 201922 October 2019A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1ILLUMINATE-B: An Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1Primary Hyperoxaluria;Primary Hyperoxaluria Type 1 (PH1)Drug: LumasiranAlnylam PharmaceuticalsRecruitingN/A5 YearsAll20Phase 3United States;France;Germany;Israel;United Kingdom
5NCT03856866January 11, 201911 March 2019Hydroxychloroquine Administration for Reduction of PexophagyHydroxychloroquine Administration for Reduction of PexophagyZellweger Syndrome;Peroxisome Biogenesis DisordersDrug: Placebo;Drug: HydroxychloroquineThe Hospital for Sick ChildrenRecruiting6 Months40 YearsAll5Phase 2Canada
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT03681184November 27, 201828 October 2019A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1ILLUMINATE-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 1Primary Hyperoxaluria Type 1 (PH1)Drug: Lumasiran;Drug: Sterile Normal Saline (0.9% NaCl)Alnylam PharmaceuticalsNot recruiting6 YearsN/AAll30Phase 3United States;France;Germany;Israel;Netherlands;Switzerland;United Arab Emirates;United Kingdom
7EUCTR2018-001981-40-DE7 January 2019A Phase 3 Study of an Investigational Drug, Lumasiran (ALN-GO1) with an Extended Dosing Period in Children and Adults with Primary Hyperoxaluria Type 1 DiseaseILLUMINATE-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 1Primary 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 AvailableFemale: yes
Male: yes
30Phase 3France;United States;United Arab Emirates;Jordan;Israel;Netherlands;Germany;Japan;Switzerland
8EUCTR2016-003134-24-DE28/12/201730 April 2019Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients with Primary Hyperoxaluria Type 1Primary 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.AuthorisedFemale: yes
Male: yes
20Phase 2France;United States;United Arab Emirates;Jordan;Israel;Netherlands;Germany;United Kingdom
9NCT03350451November 20, 201728 October 2019An Extension Study of an Investigational Drug, ALN-GO1, in Patients With Primary Hyperoxaluria Type 1A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients With Primary Hyperoxaluria Type 1PH1;Primary Hyperoxaluria;RNAi Therapeutic;siRNA;AGTDrug: ALN-GO1Alnylam PharmaceuticalsRecruiting6 YearsN/AAll20Phase 2France;Germany;Israel;Netherlands;United Kingdom
10NCT02795325May 13, 20169 October 2018A 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 1Drug: DCR-PH1;Other: PlaceboDicerna Pharmaceuticals, Inc.Not recruiting12 YearsN/AAll41Phase 1Germany;Netherlands
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2015-003142-51-GB11/04/201625 April 2016An Early Phase Study of DCR-PH1 in Patients with an Inherited Disorder Resulting in Overproduction of OxalateA 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.AuthorisedFemale: yes
Male: yes
42Phase 1France;United States;Israel;Netherlands;Germany;United Kingdom
12EUCTR2015-004407-23-GB10/02/20167 January 2019The First-in-Human Study of an Investigational Drug, ALN-GO1, in Healthy Adult Subjects and Patients with Primary Hyperoxaluria Type 1 DiseaseA 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 1Primary 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.AuthorisedFemale: yes
Male: yes
52Phase 1;Phase 2France;United States;Jordan;Israel;Netherlands;Germany;United Kingdom
13NCT02171104July 10, 20147 October 2019MT2013-31: Allo HCT for Metabolic Disorders and Severe OsteopetrosisMT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATGMucopolysaccharidosis 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 DisordersBiological: 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 MinnesotaRecruitingN/A55 YearsAll100Phase 2United States
14NCT01626092July 11, 201216 December 2017Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal DisordersTreatment 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 DisorderDrug: Campath-1H;Drug: Clofarabine;Drug: Melphalan;Radiation: Total Body Irradiation with Marrow Boosting;Biological: Hematopoietic stem cell transplantation;Drug: Cyclosporine A;Drug: Mycophenolate mofetilMasonic Cancer Center, University of MinnesotaNot recruitingN/A55 YearsAll3N/AUnited States
15EUCTR2010-022046-25-NL2 October 2017Cholic acid treatment in Peroxisomal Biogenesis Disorders (Zellweger spectrum): biochemical and clinical effects. - Cholic acid in ZellwegerCholic acid treatment in Peroxisomal Biogenesis Disorders (Zellweger spectrum): biochemical and clinical effects. - Cholic acid in ZellwegerPeroxisomal 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 CentreNot AvailableFemale: yes
Male: yes
Phase 2Netherlands
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16NCT00004442September 19977 April 2015Study of Bile Acids in Patients With Peroxisomal DisordersInfantile Refsum's Disease;Zellweger Syndrome;Bifunctional Enzyme Deficiency;AdrenoleukodystrophyDrug: chenodeoxycholic acid;Drug: cholic acid;Drug: ursodiolChildren's Hospital Research Foundation University of CincinnatiChildren's Hospital Medical Center, CincinnatiNot recruitingN/A5 YearsBoth25N/A
17NCT00007020January 199219 February 2015Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic AcidInvestigation 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-M01RR08084-0009Infantile Refsum's Disease;Zellweger Syndrome;Adrenoleukodystrophy;Peroxisomal Disorders;CholestasisDrug: Cholic AcidsAsklepion Pharmaceuticals, LLCChildren's Hospital Medical Center, CincinnatiNot recruitingN/AN/ABoth79Phase 3United States

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