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

• Register: EUCTR
• Last refreshed on: 2 October 2017
• Main ID: EUCTR2010-022046-25-NL
• Date of registration: 27/10/2010
• Prospective Registration: Yes
• Primary sponsor: Department of Pediatric Gastroenterology, Academic Medical Centre
• Public title: Cholic acid treatment in Peroxisomal Biogenesis Disorders (Zellweger spectrum): biochemical and clinical effects. - Cholic acid in Zellweger
• Scientific title: Cholic acid treatment in Peroxisomal Biogenesis Disorders (Zellweger spectrum): biochemical and clinical effects. - Cholic acid in Zellweger
• Recruitment status: NA
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2010-022046-25
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: no Open: no Single blind: no Double blind: no Parallel group: no Cross over: yes Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: yes Other specify the comparator: no medication
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Netherlands

Key inclusion & exclusion criteria

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


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Peroxisomal biogenesis disorder Synonym: Zellweger spectrum disorder This syndrome encompasses a spectrum of severity in symptoms Previously 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 Level: LLT Classification code 10038275 Term: Refsum's disease

Intervention(s)

Product Name: cholic acid
Product Code: CA
Pharmaceutical Form: Capsule*
INN or Proposed INN: Cholic Acid
CAS Number: 81254
Current Sponsor code: CA
Other descriptive name: CHOLIC ACID
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: up to
Concentration number: 10-20

Primary Outcome(s)

Secondary Objective: Other biochemical and clinical effects of cholic acid supplementation in Zellweger spectrum disorder:
- changes in liver abnormalities
- change in neurological abnormalities

Main Objective: Biochemical and clinical effects of cholic acid supplementation in Zellweger spectrum disorder:
- changes in bile acid pool
- change in growth
- change in fat soluble vitamines

Feasibility and safety of cholic acid supplementation

Primary end point(s): 1- - Degree of suppression of endogenous bile acid synthesis
- Increase in normal primary bile acids (increase in urine CA)

2- - Change in fat soluble vitamines levels

3- - Change in weight gain (weight-for-height percentile)
- Change total body length growth rate (cm/year; only in those with
remaining growth potential).

Secondary ID(s)

Zellweger