Clinical Trial Details
— Status: Suspended
Administrative data
| NCT number |
NCT00571272 |
| Other study ID # |
LOGIC Study - ChiLDReN Network |
| Secondary ID |
U01DK103149U01DK |
| Status |
Suspended |
| Phase |
|
| First received |
|
| Last updated |
|
| Start date |
November 30, 2007 |
| Est. completion date |
May 2029 |
Study information
| Verified date |
June 2024 |
| Source |
Arbor Research Collaborative for Health |
| Contact |
n/a |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Observational
|
Clinical Trial Summary
Cholestasis is a condition in which bile is not properly transported from the liver to the
small intestine. Cholestasis can be caused by an array of childhood diseases, including the
genetic diseases Alagille syndrome (ALGS), alpha-1 antitrypsin (a-1AT) deficiency, bile acid
synthesis and metabolism defects, and progressive familial intrahepatic cholestasis (PFIC) or
benign recurrent intrahepatic cholestasis(BRIC). This study will investigate the natural
history and progression of the four previously mentioned cholestatic liver diseases to
provide a better understanding of the causes and effects of the diseases.
Description:
Cholestasis is a rare condition that involves a reduction or obstruction of bile flow from
the liver to the small intestine. When bile flow is hindered, a waste product pigment called
bilirubin can escape into the bloodstream and build up to harmful levels. This may lead to
the easily recognizable cholestatic symptoms of jaundice, itching, and impaired growth and
eventually to more serious health problems. Four rare genetic liver disorders- ALGS, a-1AT,
bile acid synthesis and metabolism defects, and PFIC-account for about 20% to 30% of all
infant cases of cholestasis. These four disorders compose a group of related diseases that
can cause significant growth problems during childhood, serious liver problems, the need for
liver transplantation, and potentially death. More research on these rare liver diseases is
necessary to develop a scientific basis for improvement in diagnostic techniques and
treatments. Current diagnostic procedures are complex, and the development of simpler
diagnostic tests would facilitate early diagnosis and treatment. This study will investigate
the natural history and progression of the four previously mentioned cholestatic liver
diseases to provide a better understanding of the causes and effects of the diseases.
Participation in this study will last 20 years and will consist of a baseline visit and 20
annual follow-up visits. The study will enroll infants through adults 25 years of age who
have, or are suspected of having, one of the four genetic cholestatic liver diseases.
Individuals who are siblings of a-A1T participants and have underlying disease with no
evidence of liver involvement may also be enrolled. Study visits will involve review of
clinical information, family history, and any clinically indicated treatments and their
outcomes; a physical exam; laboratory tests; and radiologic and imaging evaluations. In
addition to these standard of care evaluations, participants will undergo several special
research evaluations, including quality of life questionnaires, neurodevelopmental
evaluations, hearing exams, liver histology studies, and collection of serum, plasma, urine,
and blood for DNA. Serum, plasma, and blood for DNA will also be collected from both
biological parents and from affected siblings of participants with a-A1T or ALGS. Genetic
testing will be performed using the collected specimens.
