DDrare: Database of Drug Development for Rare Diseases

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：38，薬物数：37（DrugBank：12），標的遺伝子数：12，標的パスウェイ数：42］

Searched query = "Peroxisomal disease (except Adrenoleukodystrophy)", "Peroxisomal disease", "Peroxisomal disorder", "Peroxisome biogenesis disorder", "PEX gene disorder", "Zellweger syndrome", "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", "PH1", "Acatalasemia", "Acatalasia", "Contiguous ABCD1/DXS1357E deletion syndrome", "CADDS"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Drug = ALN-65585; ALN-GO1; CA; CALD HR-D (High-Risk, Regimen C); CALD HR-D (High-Risk, Regimen D); CALD SR-A (Standard-Risk, Regimen A); CALD SR-B (Standard-Risk, Regimen B); CHOLIC ACID; Campath; Campath-1H; Chenodeoxycholic acid; Cholic Acid; Cholic Acids; Cholic acid; Clofarabine; Cyclosporine; Cyclosporine A; DCR-1171X; DCR-L1360; DCR-PH1; DCR-PHXC; Hematopoietic stem cell transplantation; Hydroxychloroquine; IMD; IMD Preparative Regimen; LUMASIRAN; Lumasiran; Melphalan; Mycophenolate; Mycophenolate mofetil; Nedosiran; Osteopetrosis Haploidentical Only Preparative Regimen; Osteopetrosis Only Preparative Regimen; Radiation: Total Body Irradiation with Marrow Boosting; Stem Cell Transplantation; Sterile Normal Saline (0.9% NaCl); Ursodiol

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	Recruiting	6 Years	N/A	All	36	Phase 2	United States;Australia;Canada;France;Germany;Israel;Italy;Japan;Lebanon;Netherlands;New Zealand;Poland;Romania;Spain;United Kingdom
2	NCT03681184 (ClinicalTrials.gov)	November 27, 2018	19/9/2018	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	NULL	Active, not recruiting	6 Years	N/A	All	39	Phase 3	United States;France;Germany;Israel;Netherlands;Switzerland;United Arab Emirates;United Kingdom

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

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

Recruiting

6 Years

N/A

All

Phase 2

United States;Australia;Canada;France;Germany;Israel;Italy;Japan;Lebanon;Netherlands;New Zealand;Poland;Romania;Spain;United Kingdom

NCT03681184
(ClinicalTrials.gov)

November 27, 2018

19/9/2018

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

NULL

Active, not recruiting

6 Years

N/A

All

Phase 3

United States;France;Germany;Israel;Netherlands;Switzerland;United Arab Emirates;United Kingdom