234. Peroxisomal disease (except Adrenoleukodystrophy) Clinical trials / Disease details
Clinical trials : 39 / Drugs : 35 - (DrugBank : 12) / Drug target genes : 13 - Drug target pathways : 45
| 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 | NCT00004442 (ClinicalTrials.gov) | September 1997 | 18/10/1999 | Study of Bile Acids in Patients With Peroxisomal Disorders | Infantile Refsum's Disease;Zellweger Syndrome;Bifunctional Enzyme Deficiency;Adrenoleukodystrophy | Drug: chenodeoxycholic acid;Drug: cholic acid;Drug: ursodiol | Children's Hospital Research Foundation University of Cincinnati | Children's Hospital Medical Center, Cincinnati | Terminated | N/A | 5 Years | Both | 25 | N/A | NULL | |
| 2 | NCT00007020 (ClinicalTrials.gov) | January 1992 | 6/12/2000 | Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid | Investigation in the Pathogenesis of Liver Disease in Patients With Inborn Errors of Bile Acid Metabolism | Infantile Refsum's Disease;Zellweger Syndrome;Adrenoleukodystrophy;Peroxisomal Disorders;Cholestasis | Drug: Cholic Acids | Travere Therapeutics, Inc. | Children's Hospital Medical Center, Cincinnati | Completed | N/A | N/A | All | 85 | Phase 3 | United States |
| 3 | EUCTR2010-022046-25-NL (EUCTR) | 27/10/2010 | Cholic acid treatment in Peroxisomal Biogenesis Disorders (Zellweger spectrum): biochemical and clinical effects. - Cholic acid in Zellweger | Cholic 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 Centre | NULL | NA | Female: yes Male: yes | Phase 2 | Netherlands |