NCT00272844View on ClinicalTrials.gov

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

PHASE1/PHASE2CompletedINTERVENTIONAL
Enrollment

23

Participants

Timeline

Start Date

January 31, 1998

Primary Completion Date

June 30, 2011

Study Completion Date

July 31, 2011

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.

Trial Locations (1)

02115

Children's Hospital Boston, Boston

All Listed Sponsors
lead

Boston Children's Hospital

OTHER

NCT00272844 - Treatment of the Cholesterol Defect in Smith-Lemli-Opitz Syndrome | Biotech Hunter | Biotech Hunter