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Bile Acid

Cholic Acid for Smith-Lemli-Opitz Syndrome

Phase 1 & 2
Waitlist Available
Led By William B Rizzo, MD
Research Sponsored by University of Nebraska
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 8 weeks
Awards & highlights

Study Summary

This trial will test whether dietary cholic acid can help people with Smith-Lemli-Opitz syndrome by increasing their cholesterol and reducing harmful cholesterol precursors.

Eligible Conditions
  • Smith-Lemli-Opitz Syndrome

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~8 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 8 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Change in Plasma Cholesterol

Side effects data

From 2016 Phase 3 trial • 53 Patients • NCT01438411
15%
Upper respiratory tract infection
11%
Vitamin D decreased
11%
Diarrhoea
11%
Abdominal pain
9%
Disease progression
8%
Nasopharyngitis
8%
Hepatic enzyme increased
6%
Pneumonia
6%
Coagulopathy
6%
Arthralgia
4%
Encephalopathy
4%
Influenza
4%
Respiratory failure
4%
Fracture
2%
Hypoxia
2%
Cardiac arrest
2%
Completed suicide
2%
Hepatic artery thrombosis
2%
Gastric ulcer
2%
Pyrexia
2%
Hepatic failure
2%
Hyperbilirubinaemia
2%
Bacteraemia
2%
Ear infection
2%
Hypoalbuminaemia
2%
Mental impairment
2%
Orchidopexy
2%
Thrombosis
2%
Respiratory distress
2%
Abdominal distension
2%
Dehydration
2%
Oesophageal carcinoma
2%
Somnolence
2%
Bradykardia
2%
Gastroenteritis viral
2%
Hypokalaemia
2%
Sepsis
2%
Adenoidectomy
100%
80%
60%
40%
20%
0%
Study treatment Arm
Cholic Acid

Trial Design

1Treatment groups
Experimental Treatment
Group I: Cholic acidExperimental Treatment1 Intervention
Participants will be treated with cholic acid 10 mg/kg body weight.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Cholic Acid
2010
Completed Phase 3
~90

Find a Location

Who is running the clinical trial?

Children's Hospital Medical Center, CincinnatiOTHER
815 Previous Clinical Trials
6,530,658 Total Patients Enrolled
University of PittsburghOTHER
1,731 Previous Clinical Trials
16,307,425 Total Patients Enrolled
University of NebraskaLead Sponsor
541 Previous Clinical Trials
1,144,741 Total Patients Enrolled

Media Library

Cholic Acid (Bile Acid) Clinical Trial Eligibility Overview. Trial Name: NCT03720990 — Phase 1 & 2
Smith-Lemli-Opitz Syndrome Research Study Groups: Cholic acid
Smith-Lemli-Opitz Syndrome Clinical Trial 2023: Cholic Acid Highlights & Side Effects. Trial Name: NCT03720990 — Phase 1 & 2
Cholic Acid (Bile Acid) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03720990 — Phase 1 & 2
~3 spots leftby May 2025
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