Clinical trials: Bile Acid Synthesis Disorders
Inborn Errors of Bile Acid Synthesis
Efficacy of To Be Marketed (TBM) Cholic Acid Capsules Used to Treat Children With Inborn Errors of Bile Acid Synthesis
This is a study in a small population of children who have inborn errors of bile acid synthesis who are currently taking established doses of the currently used cholic acid capsules prepared at the Cincinnati Children's Hospital…
- Trial name
- NCT01115582
- Testing
- Cholic acid
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Hypercholesterolemia
The Efficacy of Cholesterol-lowering Probiotic Lactobacillus Plantarum LPLDL® in Hypercholesterolemic Adults.
Coronary heart disease (CHD) is one of the major causes of death and disability in industrialised countries. Results from several epidemiological and clinical studies indicate a positive correlation between elevated total serum…
- Trial name
- ProLoChol (NCT03540108)
- Testing
- Lactobacillus plantarum ECGC 13110402
- Where
- United Kingdom
- Volunteers
- 16 people
- Sponsor
- University of Roehampton
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Bile Acid Synthesis Disorders
The REPLACE Registry for Cholbam® (Cholic Acid)
This is a prospective, observational, non-interventional patient registry study designed to document product safety and effectiveness outcomes for 10 years in patients treated with Cholbam, including those who have been using…
- Trial name
- NCT03115086
- Testing
- Cholbam
- Where
- 24 sites in United States
- Volunteers
- 55 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Cerebrotendinous Xanthomatosis
Cholestanol in Humans
The treatment of cerebrotendinous xanthomatosis an in born error of bile acid synthesis with chenodeoxycholic acid. Patients with this disease over produce cholestanol and bile acid precursors because of the block in synthesis.
- Trial name
- NCT00018694
- Testing
- Chenodeoxycholic Acid
- Where
- United States
- Sponsor
- US Department of Veterans Affairs
Health, Bile Acid Synthesis Disorders
Bile Acids As Determinants of Postprandial Metabolism
This study aims to understand how bile acids (BAs) appear in the bloodstream after eating and how this might affect inflammation and metabolism.
- Conditions
- Health, Bile Acid Synthesis Disorders, Postprandial Metabolism, Postprandial Lipids Metabolism
- Trial name
- PRIME (NCT06758453)
- Testing
- Dietary Challenge
- Where
- Brazil
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- University of Sao Paulo
Liver Diseases, Alagille Syndrome
Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis (LOGIC)
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)…
- Conditions
- Liver Diseases, Alagille Syndrome, Alpha 1-Antitrypsin Deficiency
- Trial name
- LOGIC (NCT00571272)
- Where
- 17 sites in United States, Canada
- Volunteers
- 1,675 people
- Sponsor
- Arbor Research Collaborative for Health
Chronic Pancreatitis, Inflammatory Bowel Diseases
Genomic Resources for Enhancing Available Therapies (GREAT1.0) Study
This is a prospective, descriptive, observational research study designed to observe and document the clinical practice by domain experts, and how the knowledge of new findings that are published in the medical literature affect…
- Conditions
- Chronic Pancreatitis, Inflammatory Bowel Diseases, Hepatitis, NASH - Nonalcoholic Steatohepatitis
- Trial name
- GREAT1 (NCT04306939)
- Testing
- venipuncture, Questionnaires, Additional Sample Collections
- Where
- United States
- Volunteers
- 120,000 people, healthy volunteers welcome
- Sponsor
- University of Pittsburgh