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_
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PhaseCountries
1NCT00004442
(ClinicalTrials.gov)
September 199718/10/1999Study of Bile Acids in Patients With Peroxisomal DisordersInfantile Refsum's Disease;Zellweger Syndrome;Bifunctional Enzyme Deficiency;AdrenoleukodystrophyDrug: chenodeoxycholic acid;Drug: cholic acid;Drug: ursodiolChildren's Hospital Research Foundation University of CincinnatiChildren's Hospital Medical Center, CincinnatiTerminatedN/A5 YearsBoth25N/ANULL
2NCT00007020
(ClinicalTrials.gov)
January 19926/12/2000Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic AcidInvestigation in the Pathogenesis of Liver Disease in Patients With Inborn Errors of Bile Acid MetabolismInfantile Refsum's Disease;Zellweger Syndrome;Adrenoleukodystrophy;Peroxisomal Disorders;CholestasisDrug: Cholic AcidsTravere Therapeutics, Inc.Children's Hospital Medical Center, CincinnatiCompletedN/AN/AAll85Phase 3United States
3EUCTR2010-022046-25-NL
(EUCTR)
27/10/2010Cholic acid treatment in Peroxisomal Biogenesis Disorders (Zellweger spectrum): biochemical and clinical effects. - Cholic acid in ZellwegerCholic acid treatment in Peroxisomal Biogenesis Disorders (Zellweger spectrum): biochemical and clinical effects. - Cholic acid in Zellweger Peroxisomal biogenesis disorder Synonym: Zellweger spectrum disorderThis syndrome encompasses a spectrum of severity in symptomsPreviously the disorder was subdivided into three disorders based on the severity of symptoms; Infantile Refsum disease (IRD) is the mildest form, neonatal adrenoleucodystrofia (NALD) has more severe symptoms and Zellweger syndrome has the most severe clinical symptoms.
MedDRA version: 12.1;Level: LLT;Classification code 10053706;Term: Zellweger syndrome
MedDRA version: 12.1;Classification code 10038275;Term: Refsum's disease
Product Name: cholic acid
Product Code: CA
INN or Proposed INN: Cholic Acid
Other descriptive name: CHOLIC ACID
Department of Pediatric Gastroenterology, Academic Medical CentreNULLNAFemale: yes
Male: yes
Phase 2Netherlands