Simvastatin Therapy in Smith-Lemli-Opitz Syndrome

PHASE2CompletedINTERVENTIONAL
Enrollment

23

Participants

Timeline

Start Date

July 31, 2003

Primary Completion Date

December 31, 2010

Study Completion Date

December 31, 2010

Conditions
Smith-Lemli-Opitz Syndrome
Interventions
DRUG

Simvastatin Susp.

During the simvastatin phase of the trial, therapy will be initiated at 0.5 mg/kg/day for six weeks and then increased to 1.0 mg/kg/day if adverse side effects are minimal or absent.

DRUG

OraPlus

During this trial and for two months prior, patients will be maintained on 150 mg/kg/day of dietary cholesterol (150 mg/ml in OraPlus) for the duration of the trial

Trial Locations (1)

20892

National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda

All Listed Sponsors
collaborator

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

NIH

lead

Forbes Porter, M.D.

NIH

NCT00064792 - Simvastatin Therapy in Smith-Lemli-Opitz Syndrome | Biotech Hunter | Biotech Hunter