Cholic acid treatment in Peroxisomal Biogenesis Disorders (Zellweger spectrum): biochemical and clinical effects. - Cholic acid in Zellweger

Phase 1

• Conditions: 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: 12.1Level: LLTClassification code 10053706Term: Zellweger syndrome; MedDRA version: 12.1Level: LLTClassification code 10038275Term: Refsum's disease

• Registration Number: EUCTR2010-022046-25-NL
• Lead Sponsor: Department of Pediatric Gastroenterology, Academic Medical Centre

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2010-10-27
• Last Update Posted: 2 October 2017

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

Zellweger spectrum disorder
At least one of the following hallmarks: steatorrhea, elevated transaminases, growth retardation, neurological symptoms

Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Short life expectancy based on severe multiple organ dysfunction in the first 6 months of life

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified