Actively Recruiting

Phase 2
Age: 0 - 65Years
All Genders
NCT01773278

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

Led by University of Colorado, Denver · Updated on 2024-05-08

100

Participants Needed

1

Research Sites

887 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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

Official Title

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

Who Can Participate

Age: 0 - 65Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • 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
Not Eligible

You will not qualify if you...

  • Absence of detectable 7-dehydrocholesterol/8-dehydrocholesterol
  • Allergy to antioxidant medication

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

Children's Hospital Colorado

Aurora, Colorado, United States, 80045

Actively Recruiting

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Research Team

E

Ellen R Elias, MD

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NON_RANDOMIZED

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

3

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