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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
3 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2015-003142-51-NL
(EUCTR)
21/07/201617/11/2015An 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: 18.1;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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
42Phase 1France;United States;Israel;Germany;Netherlands;United Kingdom
2NCT02795325
(ClinicalTrials.gov)
May 13, 201618/5/2016A 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.NULLTerminated12 YearsN/AAll41Phase 1Germany;Netherlands
3EUCTR2015-003142-51-GB
(EUCTR)
11/04/201606/10/2015An 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]
Dicerna Pharmaceuticals, Inc.NULLNot Recruiting Female: yes
Male: yes
42Phase 1France;United States;Israel;Netherlands;Germany;United Kingdom