drugset / Trial / NCT03720990
Smith-Lemli-Opitz Syndrome and Cholic Acid
Phase 1/2
Completed
12 enrolled
University of Nebraska
Children's Hospital Medical Center, Cincinnati · collabUniversity of Colorado, Denver · collabUniversity of Pittsburgh · collab
NaSingle-groupOpen-labelTreatment
Summary
The purpose of this study is to determine whether dietary cholic acid therapy benefits people with Smith-Lemli-Opitz syndrome (SLOS) by leading to an increase in plasma cholesterol and reduction in harmful cholesterol precursors. SLOS participants will be treated with dietary cholic acid for 8 weeks and plasma cholesterol and cholesterol precursor metabolites will be measured.
Timeline
- Start
- 2021-03-27
- Primary completion
- 2022-08-30
- Completion
- 2023-09-30
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Cholic Acid | Small molecule | 10 mg/kg | — |