Cholic acid treatment in Peroxisomal Biogenesis Disorders (Zellweger spectrum)

Not Applicable

Completed

• Conditions: Zellweger spectrum disorder; Neonatal Diseases

• Registration Number: ISRCTN96480891
• Lead Sponsor: Emma Paeditric Hosptial (Netherlands)

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2012-01-30
• Last Update Posted: 17 October 2016

Recruitment & Eligibility

• Status: Completed
• Sex: All
• Target Recruitment: 25

Inclusion Criteria

1. Zellweger spectrum disorder
2. At least one of the following hallmarks:
2.1. Steatorrhea
2.2. Elevated transaminases
2.3. Growth retardation
2.4. Neurological symptoms

Exclusion Criteria

Short life expectancy (severe multiple organ dysfunction at the time of diagnosis)

Study & Design

• Study Type: Interventional
• Study Design: Not specified