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 |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2015-003142-51-NL (EUCTR) | 21/07/2016 | 17/11/2015 | 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: 18.1;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: DCR-PH1 INN or Proposed INN: Not Yet Assigned Other descriptive name: DCR-1171X | Dicerna Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 42 | Phase 1 | France;United States;Israel;Germany;Netherlands;United Kingdom | ||
| 2 | NCT02795325 (ClinicalTrials.gov) | May 13, 2016 | 18/5/2016 | 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. | NULL | Terminated | 12 Years | N/A | All | 41 | Phase 1 | Germany;Netherlands |
| 3 | EUCTR2015-003142-51-GB (EUCTR) | 11/04/2016 | 06/10/2015 | 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] | Dicerna Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 42 | Phase 1 | France;United States;Israel;Netherlands;Germany;United Kingdom |