Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C

PHASE1/PHASE2CompletedINTERVENTIONAL
Enrollment

4

Participants

Timeline

Start Date

February 22, 2019

Primary Completion Date

October 4, 2024

Study Completion Date

October 4, 2024

Conditions
Niemann-Pick Disease, Type C
Interventions
DRUG

adrabetadex (2-Hydroxypropyl-Beta-Cyclodextrin)

"VTS-270 (2-Hydroxypropyl-Beta-Cyclodextrin) will be administered intravenously to specifically target liver disease. In the first phase of the study, dosing frequency will be twice a week with IV adrabetadex (VTS-270) for six weeks for a total of 12 administrations. Subjects will be evaluated at each study visit for evidence of adverse effects.~Doses to be studied are 500, and 1000 mg/kg. Six subjects will be studied at each dose level. Cohort 1: Subjects 1-6, 500 mg/kg Cohort 2: Subjects 7-12, 1000 mg/kg"

Trial Locations (1)

63110

St. Louis Children's Hospital, St Louis

All Listed Sponsors
collaborator

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

NIH

lead

Washington University School of Medicine

OTHER

NCT03471143 - Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C | Biotech Hunter | Biotech Hunter