234. ペルオキシソーム病（副腎白質ジストロフィーを除く。） Peroxisomal disease (except Adrenoleukodystrophy) Clinical trials / Disease details

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

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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)	October 2021	30/6/2021	Nedosiran in Pediatric Patients From Birth to 5 Years of Age With PH and Relatively Intact Renal Fun ...	A Phase 2 Open-Label Multicenter Study to Evaluate the Safety, Pharmacokinetics, and Efficacy of Ned ...	Primary Hyperoxaluria;Primary Hyperoxaluria Type 2;Primary Hyperoxaluria Type 1	Drug: nedosiran	Dicerna Pharmaceuticals, Inc.	NULL	Not yet recruiting	N/A	5 Years	All	10	Phase 2	NULL
2	EUCTR2019-001346-17-IT (EUCTR)	14/05/2021	07/06/2021	A Phase 3 study of an Investigational Drug, Lumasiran (ALN-GO1) in patients with advanced primary hy ...	ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamic ...	Primary Hyperoxaluria Type 1(PH1) MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: ...	Product Name: lumasiran Product Code: [ALN-GO1] INN or Proposed INN: Lumasiran	ALNYLAM PHARMACEUTICALS, INC.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	20	Phase 3	United Arab Emirates;United States;Lebanon;Turkey;Israel;United Kingdom;Switzerland;Italy;India;Egyp ...
3	EUCTR2020-002826-97-IT (EUCTR)	12/05/2021	07/06/2021	Study for patients with Primary Hyperoxaluria and Severe Renal Impairment to evaluate the efficacy, ...	A Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of DCRPHXC in Patients With Primary H ...	Primary Hyperoxaluria MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluri ...	Product Name: Nedosiran Product Code: [DCR-PHXC] INN or Proposed INN: DCR-L1360	Dicerna Pharmaceuticals Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	24	Phase 2	France;United States;Spain;Romania;Australia;Germany;United Kingdom;Italy
4	EUCTR2020-002826-97-DE (EUCTR)	20/04/2021	08/12/2020	Study for patients with Primary Hyperoxaluria and Severe Renal Impairment to evaluate the efficacy, ...	A Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of DCR-PHXC in Patients With Primary ...	Primary Hyperoxaluria MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluri ...	Product Name: Nedosiran Product Code: DCR-PHXC INN or Proposed INN: DCR-L1360 Other descriptive name: N ...	Dicerna Pharmaceuticals Inc	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	30	Phase 2	United States;France;Spain;Romania;Germany;United Kingdom;Italy
5	NCT04580420 (ClinicalTrials.gov)	April 15, 2021	24/9/2020	Safety & Efficacy of DCR-PHXC in Patients With PH1/2 and ESRD	A Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of DCR-PHXC in Patients With Primary ...	Primary Hyperoxaluria;Primary Hyperoxaluria Type 2;End Stage Renal Disease	Drug: DCR-PHXC	Dicerna Pharmaceuticals, Inc.	NULL	Recruiting	N/A	N/A	All	12	Phase 2	United States;Spain;United Kingdom
6	EUCTR2020-002826-97-FR (EUCTR)	29/01/2021	30/09/2020	Study for patients with Primary Hyperoxaluria and Severe Renal Impairment to evaluate the efficacy, ...	A Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of DCR-PHXC in Patients With Primary ...	Primary Hyperoxaluria MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: Hyperoxaluri ...	Product Name: Nedosiran Product Code: DCR-PHXC INN or Proposed INN: DCR-L1360 Other descriptive name: N ...	Dicerna Pharmaceuticals Inc	NULL	Not Recruiting			Female: yes Male: yes	24	Phase 2	United States;France;Spain;Romania;Australia;Germany;United Kingdom;Japan;Italy
7	EUCTR2019-001346-17-GB (EUCTR)	22/01/2020	23/07/2019	A Phase 3 study of an Investigational Drug, Lumasiran (ALN-GO1) in patients with advanced primary hy ...	ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamic ...	Primary Hyperoxaluria Type 1(PH1) MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: ...	Product Name: lumasiran Product Code: ALN-GO1 INN or Proposed INN: Lumasiran	Alnylam Pharmaceuticals, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	16	Phase 3	United Arab Emirates;United States;Lebanon;Turkey;Israel;United Kingdom;Switzerland;India;Egypt;Fran ...
8	NCT04152200 (ClinicalTrials.gov)	January 21, 2020	31/10/2019	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 Pharmacodynamic ...	Primary Hyperoxaluria Type 1;Primary Hyperoxaluria	Drug: Lumasiran	Alnylam Pharmaceuticals	NULL	Active, not recruiting	N/A	N/A	All	21	Phase 3	United States;Australia;Belgium;France;Israel;Italy;Jordan;Lebanon;Netherlands;Turkey;United Arab Em ...
9	EUCTR2019-001346-17-NL (EUCTR)	05/12/2019	22/08/2019	A Phase 3 study of an Investigational Drug, Lumasiran (ALN-GO1) in patients with advanced primary hy ...	ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamic ...	Primary Hyperoxaluria Type 1(PH1) MedDRA version: 20.1;Level: PT;Classification code 10020703;Term: ...	Product Name: lumasiran Product Code: ALN-GO1 INN or Proposed INN: LUMASIRAN	Alnylam Pharmaceuticals, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	16	Phase 3	United States;United Arab Emirates;Lebanon;Turkey;Israel;Switzerland;United Kingdom;India;Egypt;Fran ...
10	NCT03847909 (ClinicalTrials.gov)	October 28, 2019	15/2/2019	A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1and Primary Hyp ...	A Phase 2 Placebo-Controlled, Double-Blind, Multicenter Study to Evaluate the Efficacy, Safety, and ...	Primary Hyperoxaluria Type 1(PH1);Primary Hyperoxaluria Type 2 (PH2);Kidney Diseases;Urologic Diseas ...	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;Pol ...

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