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Smith-Lemli-Opitz Syndrome and Cholic Acid

NCT03720990 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2024-04-30

Study results available
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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.

Conditions

  • Smith-Lemli-Opitz Syndrome

Interventions

DRUG

Cholic Acid

Participants will be treated with cholic acid for 8 weeks

Sponsors & Collaborators

  • University of Colorado, Denver

    collaborator OTHER

  • Children's Hospital Medical Center, Cincinnati

    collaborator OTHER

  • University of Pittsburgh

    collaborator OTHER

  • University of Nebraska

    lead OTHER

Principal Investigators

  • Ellen R Elias, MD · University of Colorado - Colorado Children's Hospital

  • William B Rizzo, MD · University of Nebraska

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
25 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-03-27
Primary Completion
2022-08-30
Completion
2023-09-30
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03720990 on ClinicalTrials.gov