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Cholesterol + Antioxidants for Smith-Lemli-Opitz Syndrome

Phase 2
Recruiting
Led By Ellen R Elias, MD
Research Sponsored by University of Colorado, Denver
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Clinical diagnosis of Smith-Lemli-Opitz Syndrome
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1-2 year
Awards & highlights

Study Summary

This trial is testing a treatment for patients with a rare genetic disease called Smith-Lemli-Opitz syndrome. The treatment involves giving the patients cholesterol and antioxidant medication, and then carefully monitoring them with regular clinic visits and lab tests. Once a year, the patients also undergo a series of tests under anesthesia, to check for any progression of their disease.

Who is the study for?
This trial is for patients with Smith-Lemli-Opitz Syndrome (SLOS), who have high levels of specific cholesterol byproducts and can visit Children's Hospital Colorado yearly. They must also have insurance that covers certain eye and ear tests. People allergic to antioxidants or without detectable cholesterol byproducts cannot join.Check my eligibility
What is being tested?
The study is testing the effects of giving cholesterol and antioxidant supplements to SLOS patients. Their health is tracked through regular clinic visits, blood tests, vitamin level checks, and annual under-anesthesia exams including vision and hearing assessments.See study design
What are the potential side effects?
Potential side effects may include reactions to the antioxidant medications such as rashes or digestive issues since allergies are an exclusion criterion; however, detailed side effect profiles are not provided in this summary.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have been diagnosed with Smith-Lemli-Opitz Syndrome.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1-2 year
This trial's timeline: 3 weeks for screening, Varies for treatment, and 1-2 year 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 Electroretinogram (ERG) results over time
Secondary outcome measures
Change in ABR (Auditory Brainstem response) testing over time
Other outcome measures
Change in Blood Oxysterol measurements over time

Trial Design

3Treatment groups
Experimental Treatment
Group I: antioxidant effects on retinal functionExperimental Treatment2 Interventions
Patients with SLOS will be treated with both cholesterol supplementation and antioxidants. Retinal function will be followed by serial electroretinogram (ERG) testing and pigmentary retinopathy will be followed by Serial Ophthalmologic exams under anesthesia
Group II: antioxidant effects on hearingExperimental Treatment2 Interventions
Patients with SLOS will be treated with cholesterol and antioxidant medication and their hearing will be followed by serial brainstem audiometry (ABR)
Group III: Antioxidant effect on OxysterolsExperimental Treatment2 Interventions
Patients with SLOS will be treated with antioxidants and cholesterol. Blood oxysterol levels will be measured. Future focus will be on being able to use oxysterol levels to regulate antioxidant doses, and to determine which particular antioxidants might have the most benefit in lowering oxysterols
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Cholesterol
1998
N/A
~130
Antioxidants
2005
Completed Phase 4
~7310

Find a Location

Who is running the clinical trial?

University of Colorado, DenverLead Sponsor
1,729 Previous Clinical Trials
2,143,588 Total Patients Enrolled
1 Trials studying Smith-Lemli-Opitz Syndrome
12 Patients Enrolled for Smith-Lemli-Opitz Syndrome
Ellen R Elias, MDPrincipal InvestigatorUniversity of Colorado, Denver
1 Previous Clinical Trials
12 Total Patients Enrolled
1 Trials studying Smith-Lemli-Opitz Syndrome
12 Patients Enrolled for Smith-Lemli-Opitz Syndrome

Media Library

Antioxidants (Other) Clinical Trial Eligibility Overview. Trial Name: NCT01773278 — Phase 2
Smith-Lemli-Opitz Syndrome Research Study Groups: Antioxidant effect on Oxysterols, antioxidant effects on retinal function, antioxidant effects on hearing
Smith-Lemli-Opitz Syndrome Clinical Trial 2023: Antioxidants Highlights & Side Effects. Trial Name: NCT01773278 — Phase 2
Antioxidants (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT01773278 — Phase 2

Frequently Asked Questions

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

What does the current medical research say about cholesterol levels and heart health?

"At the moment, there are 5 different clinical trials studying cholesterol. 1 of those is currently in Phase 3 testing. A large number of these trials for cholesterol are based out of one city - Indianapolis, Indiana - but there are a total of 5 locations running these studies."

Answered by AI

Are there any available openings for participants in this research?

"Yes, this is an ongoing trial that was posted on December 1st, 2008 and edited most recently on January 4th, 2022. They are recruiting 100 participants from 1 site."

Answered by AI

How many individuals are included in this research project?

"That is correct. The information available on clinicaltrials.gov suggests that this particular clinical trial has active recruitment efforts underway. This study was first posted on December 1st, 2008 and received its most recent update on January 4th, 2022. They are looking for 100 patients from a single location to participate in the trial."

Answered by AI

What are the FDA's views on cholesterol medications?

"Because a Phase 2 trial only has data supporting safety and none for efficacy, our team rates the cholesterol medication as a 2."

Answered by AI
~10 spots leftby Dec 2025