Long-term Safety Study of Cholic Acid for Patients with Bile Acid Synthesis Defects

3 1 1 1

What is this study about?

This clinical trial is focused on studying a group of rare conditions known as bile acid synthesis defects. These are disorders where the body has trouble producing bile acids, which are important for digestion and absorbing fats and vitamins. The study is testing a treatment using cholic acid, a substance derived from bovine bile, which is given in the form of capsules. The purpose of the study is to investigate the long-term safety of this personalized treatment for patients with these defects.

Participants in the study will receive cholic acid capsules tailored to their specific needs. The study will monitor various health indicators over time, such as liver function and growth, to ensure the treatment is safe and effective. The study will also look at any side effects or adverse events that may occur during the treatment period.

The trial will last for several years, allowing researchers to gather comprehensive data on the safety of cholic acid treatment in individuals with bile acid synthesis defects. This information will help determine if this treatment can be a reliable option for managing these rare conditions in the long term.

1 joining the study

Upon joining the study, the patient will begin a long-term safety study focused on the use of cholic acid for treating bile acid synthesis defects.

The study aims to assess the safety of personalized cholic acid treatment based on clinical and biochemical parameters.

2 medication administration

The patient will receive cholic acid in capsule form, administered orally.

The dosage and frequency of administration will be personalized based on the patient’s specific needs and medical condition.

3 monitoring and assessments

Throughout the study, the patient will undergo regular monitoring to evaluate the suppression of endogenous bile acid synthesis. This involves measuring the decrease in specific bile acid intermediates in serum and urine.

The study will also track any adverse events or side effects experienced by the patient.

4 secondary assessments

Additional assessments will include monitoring changes in normal primary bile acids, liver function tests, and levels of various enzymes and proteins.

The study will also evaluate changes in weight, growth rate, vitamin levels, and neurological development.

5 study duration

The study is expected to continue until December 30, 2027, providing a comprehensive evaluation of the long-term safety of cholic acid treatment.

Who Can Join the Study?

The patient must have a bile acid synthesis defect. This means there is a problem with how the body makes bile acids, which are important for digestion.
The defect can be due to specific enzyme deficiencies or a condition called Zellweger spectrum disorder. Enzymes are proteins that help chemical reactions happen in the body.
The patient must have at least one of the following signs:
- Steatorrhea: This is when there is too much fat in the stool, making it look greasy.
- Elevated transaminases: These are liver enzymes, and high levels can indicate liver problems. They are often checked with blood tests.
- Developmental delay: This means the patient is not reaching milestones in learning or physical skills as expected for their age.
- Neurological symptoms: These are problems related to the brain and nerves, like difficulty with movement or coordination.
The patient can be of any gender, meaning both males and females are eligible.
The patient must be within certain age ranges, which include children and adults.

Who Cannot Join the Study?

There are no specific reasons listed that would prevent a patient from participating in this study.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

No sites found in this category

Other Sites

Site Name	City	Country	Status
Agjnhwnlm Uyy	Amsterdam	The Netherlands

Trial status

Country	Status	Recruitment Start
The Netherlands	Not recruiting	01.06.2020

Trial locations

Investigated drugs:

Cholic Acid

Cholic Acid is a medication used in this clinical trial to treat patients with bile acid synthesis defects. It is a type of bile acid that helps the body digest fats and absorb fat-soluble vitamins. In this study, the focus is on assessing the long-term safety of using cholic acid capsules, which are personalized for each patient based on their specific clinical and biochemical needs.

Investigated diseases:

Bile acid synthesis disorder

Bile Acid Synthesis Defects – These are rare genetic disorders that affect the body’s ability to produce bile acids, which are crucial for digesting fats and absorbing fat-soluble vitamins. The condition can lead to the accumulation of toxic bile acid intermediates in the liver and bloodstream, potentially causing liver damage. Symptoms may include jaundice, poor growth, and developmental delays. Over time, individuals may experience liver dysfunction, which can manifest as changes in liver enzyme levels and protein synthesis. The disease can also affect the body’s ability to clot blood properly, leading to coagulopathy. Monitoring of growth, vitamin levels, and liver health is essential in managing the progression of this condition.

Trial ID:

2024-518652-23-00

Trial Phase:

Human Pharmacology (Phase I) – Other

Other Trials to Consider

#1 Clinical Trials Search in Europe

Long-term Safety Study of Cholic Acid for Patients with Bile Acid Synthesis Defects

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?