234. Peroxisomal disease (except Adrenoleukodystrophy)
38 clinical trials,   37 drugs   (DrugBank: 12 drugs),   12 drug target genes,   42 drug target pathways
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.
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 |
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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 |