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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?

University of PittsburghOTHER
1,719 Previous Clinical Trials
16,342,329 Total Patients Enrolled
Children's Hospital Medical Center, CincinnatiOTHER
815 Previous Clinical Trials
6,531,584 Total Patients Enrolled
University of NebraskaLead Sponsor
536 Previous Clinical Trials
1,143,910 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What is the cap on participants in this medical experiment?

"This trial is no longer recruiting. It was initially posted on March 27th 2021 and the last update was November 7th 2022. For those seeking to join other studies, 1412 clinical trials are actively searching for participants diagnosed with Smith-Lemli-Opitz Syndrome while 1 trail seeks patients affected by Cholic Acid."

Answered by AI

Does this medical experiment have any available openings?

"Per information on clinicaltrials.gov, this study is not currently enrolling patients. Initially posted in March 2021 and last edited in November of 2022, the research team are no longer recruiting for their trial; however, there are 1,413 other studies actively seeking participants."

Answered by AI

Are there prior investigations that have featured Cholic Acid?

"Currently, 1 clinical trial related to Cholic Acid is underway with none in the advanced Phase 3 stage. Omaha, Nebraska has multiple studies concerning this substance while two other sites are also conducting research into it."

Answered by AI

For what type of patient is enrollment in this trial appropriate?

"To be considered for inclusion in this clinical trial, potential participants must possess smith-lemli-opitz syndrome and fall between the ages of 2 to 25. The study is currently searching for approximately 12 people with these credentials."

Answered by AI

What afflictions can Cholic Acid be used to palliate?

"Cholic Acid is often prescribed to patients with fat malabsorption disorder, in addition to those suffering from Zellweger spectrum, single enzyme deficits and vitamin shortages."

Answered by AI

Does this research trial allow for individuals under 35 to take part?

"This clinical trial has a minimum age requirement of 2 and an upper limit of 25 years old, as stated in the inclusion criteria."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
Smith-Lemli-Opitz Syndrome and Cholic Acid
William Rizzo, MD 2021. "Smith-Lemli-Opitz Syndrome and Cholic Acid". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03720990.
~3 spots leftby Apr 2025
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