MedDataX Logo

Orlistat Treatment of Crigler-Najjar Disease

NCT ID: NCT00461799

Last Updated: 2007-04-18

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

NA

Total Enrollment

16 participants

Study Classification

INTERVENTIONAL

Study Start Date

2003-09-30

Study Completion Date

2004-01-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

The purpose of this study was to determine whether orlistat is effective in decreasing plasma unconjugated bilirubin levels in patients with Crigler-Najjar disease.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Unconjugated hyperbilirubinemia in Crigler-Najjar (CN) disease is conventionally treated with phototherapy and/or phenobarbital. Life-long daily phototherapy has considerable disadvantages. Main problems are a decreasing efficacy with age and a profound impact of the intensive phototherapy regimen on the quality of (social) life. An alternative treatment option for unconjugated hyperbilirubinemia is based on intestinal capture of UCB by oral treatment. Particularly when plasma UCB concentrations are high as in CN disease, UCB can diffuse from the blood into the intestinal lumen across the mucosa. Intestinal capture of UCB followed by fecal excretion reduces the enterohepatic circulation of UCB and subsequently decreases plasma UCB concentration. We demonstrated in Gunn rats, the animal model for CN disease, that orlistat treatment decreases plasma UCB concentrations parallel with increased fecal fat excretion, and induces net transmucosal excretion of UCB from the blood into the intestinal lumen. In human adults, orlistat has been widely applied for treatment of obesity, without serious side effects. Recent studies in obese adolescents and prepubertal children indicate that short-term orlistat treatment is well-tolerated by children and generally has only mild side effects. In the present randomized, placebo-controlled trial we determined in patients with CN disease the effects of orlistat treatment on plasma UCB concentrations, and on fecal excretion of fat and UCB.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Crigler-Najjar Syndrome

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

orlistat

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* patients with Crigler-Najjar disease above the age of 7 years

Exclusion Criteria

* cholestasis, chronic malabsorption syndrome, pregnancy
Minimum Eligible Age

8 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Erasmus Medical Center

OTHER

Sponsor Role collaborator

De Najjar Stichting

OTHER

Sponsor Role collaborator

University Medical Center Groningen

OTHER

Sponsor Role lead

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Anja M. Hafkamp, MD

Role: PRINCIPAL_INVESTIGATOR

University Medical Center Groningen and Erasmus University Medical Center

Maarten Sinaasappel, MD

Role: STUDY_CHAIR

Erasmus Medical Center

Henkjan J. Verkade, MD, PhD

Role: STUDY_DIRECTOR

University Medical Center Groningen

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Erasmus University Medical Center

Rotterdam, , Netherlands

Site Status

Countries

Review the countries where the study has at least one active or historical site.

Netherlands

References

Explore related publications, articles, or registry entries linked to this study.

Hafkamp AM, Havinga R, Sinaasappel M, Verkade HJ. Effective oral treatment of unconjugated hyperbilirubinemia in Gunn rats. Hepatology. 2005 Mar;41(3):526-34. doi: 10.1002/hep.20589.

Reference Type BACKGROUND
PMID: 15726662 (View on PubMed)

Hafkamp AM, Havinga R, Ostrow JD, Tiribelli C, Pascolo L, Sinaasappel M, Verkade HJ. Novel kinetic insights into treatment of unconjugated hyperbilirubinemia: phototherapy and orlistat treatment in Gunn rats. Pediatr Res. 2006 Apr;59(4 Pt 1):506-12. doi: 10.1203/01.pdr.0000203180.79636.98.

Reference Type BACKGROUND
PMID: 16549520 (View on PubMed)

Nishioka T, Hafkamp AM, Havinga R, vn Lierop PP, Velvis H, Verkade HJ. Orlistat treatment increases fecal bilirubin excretion and decreases plasma bilirubin concentrations in hyperbilirubinemic Gunn rats. J Pediatr. 2003 Sep;143(3):327-34. doi: 10.1067/s0022-3476(03)00298-1.

Reference Type BACKGROUND
PMID: 14517515 (View on PubMed)

Verkade HJ. A novel hypothesis on the pathophysiology of neonatal jaundice. J Pediatr. 2002 Oct;141(4):594-5. No abstract available.

Reference Type BACKGROUND
PMID: 12378205 (View on PubMed)

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

CN-01

Identifier Type: -

Identifier Source: org_study_id