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Treatment of the Cholesterol Defect in Smith-Lemli-Opitz Syndrome

NCT00272844 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 23

Last updated 2017-10-20

Study results available
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Summary

The purpose of this study is to determine whether supplementation with an oil-based cholesterol suspension will correct the biochemical abnormalities in cholesterol and its precursors in individuals with the Smith-Lemli-Opitz syndrome.

Conditions

  • Smith-Lemli-Opitz Syndrome

Interventions

DRUG

crystalline cholesterol oil-based suspension

200 mg/mL suspension of crystalline cholesterol in oil. Dosage (generally 75-300 mg/kg/day in divided doses) is based on initial cholesterol levels and regulated to increase, yet maintain, cholesterol levels no higher than normal ranges.

Sponsors & Collaborators

Principal Investigators

  • Mira Irons, M.D. · Boston Children's Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
1998-01-31
Primary Completion
2011-06-30
Completion
2011-07-31

Countries

  • United States

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00272844 on ClinicalTrials.gov