Cholesterol and Antioxidant Treatment in Patients With Smith-Lemli-Opitz Syndrome (SLOS)

Description

Patients with biochemically confirmed SLOS are being treated with cholesterol supplementation and antioxidant medication. They are carefully monitored with visits to clinic, laboratory testing including cholesterol and 7-dehydrocholesterol levels, vitamin levels, blood counts and liver and kidney function. On a serial basis, no more often than once a year, the patients undergo a series of tests under anesthesia, including electroretinogram (ERG), brainstem audiometry (ABR), and ophthalmologic exam under anesthesia to follow pigmentary retinopathy.

Conditions

Smith-Lemli-Opitz Syndrome, Cone-Rod Dystrophy, Hearing Loss

Study Overview

Study Details

Study overview

Patients with biochemically confirmed SLOS are being treated with cholesterol supplementation and antioxidant medication. They are carefully monitored with visits to clinic, laboratory testing including cholesterol and 7-dehydrocholesterol levels, vitamin levels, blood counts and liver and kidney function. On a serial basis, no more often than once a year, the patients undergo a series of tests under anesthesia, including electroretinogram (ERG), brainstem audiometry (ABR), and ophthalmologic exam under anesthesia to follow pigmentary retinopathy.

Cholesterol and Antioxidant Treatment in Patients With Smith-Lemli-Opitz Syndrome (SLOS)

Cholesterol and Antioxidant Treatment in Patients With Smith-Lemli-Opitz Syndrome (SLOS)

Condition
Smith-Lemli-Opitz Syndrome
Intervention / Treatment

-

Contacts and Locations

Aurora

Children's Hospital Colorado, Aurora, Colorado, United States, 80045

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * Clinical diagnosis of Smith-Lemli-Opitz Syndrome
  • * Elevated levels of 7-dehydrocholesterol and 8-dehydrocholesterol
  • * Must be able to travel to Children's Hospital Colorado annually
  • * Must have insurance coverage for ERG/ABR studies
  • * absence of detectable 7-dehydrocholesterol/8-dehydrocholesterol
  • * allergy to Antioxidant medication

Ages Eligible for Study

to 65 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

University of Colorado, Denver,

Ellen R Elias, MD, PRINCIPAL_INVESTIGATOR, University of Colorado, Denver

Study Record Dates

2025-12