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Cholic Acid trial
EU CTIS ID: 2024-518652-23-00
What this study is testing
To investigate the long-term safety of personalized cholic acid capsules treatment of patients with bile acid synthesis defects based on clinical and biochemical parameters.
- Phase III and phase IV (Integrated)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Bile acid synthesis defect due to 3β-hydroxy-Δ5-C27-steroid oxidoreductase-, Δ4-3-oxosteroid-5β-reductase-, α-methylacyl-CoA racemase (AMACR)-, cholesterol 7a-hydroxylase (CYP7A1) deficiency OR Zellweger spectrum disorder
- At least one of the following hallmarks: steatorrhea (confirmed per local protocol), elevated transaminases (ALAT and/or ASAT), developmental delay, neurological symptoms
You likely can't join if
- Short life expectancy of < 12 months (severe multiple organ dysfunction)
- Decompensated liver cirrhosis
- High bilirubin serum levels (conjugated bilirubin > 20 μmol/L)
- Prolonged prothrombin time (PT > 15s not due to vitamin K deficiency)
- Pregnancy and high total bile acid serum level ( > 40μmol/L)
- Allergy to one of the components of CA capsules.
See the full eligibility criteria
- Bile acid synthesis defect due to 3β-hydroxy-Δ5-C27-steroid oxidoreductase-, Δ4-3-oxosteroid-5β-reductase-, α-methylacyl-CoA racemase (AMACR)-, cholesterol 7a-hydroxylase (CYP7A1) deficiency OR Zellweger spectrum disorder
- At least one of the following hallmarks: steatorrhea (confirmed per local protocol), elevated transaminases (ALAT and/or ASAT), developmental delay, neurological symptoms
- Short life expectancy of < 12 months (severe multiple organ dysfunction)
- Decompensated liver cirrhosis
- High bilirubin serum levels (conjugated bilirubin > 20 μmol/L)
- Prolonged prothrombin time (PT > 15s not due to vitamin K deficiency)
- Pregnancy and high total bile acid serum level ( > 40μmol/L)
- Allergy to one of the components of CA capsules.
- Increased liver enzymes during previous CA treatment (for ZSD)
- Normal biochemical parameters (THCA and/or DHCA ≤1.0 μmol/L) (for ZSD)
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years, 65+ years, 18-64 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.