Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type C

PHASE1UnknownINTERVENTIONAL

Enrollment

Participants

Timeline

Start Date

May 23, 2019

Primary Completion Date

March 31, 2022

Study Completion Date

March 31, 2022

Conditions

Niemann-Pick Disease, Type C1

Interventions

DRUG

Hydroxypropyl-β-cyclodextrin

HP-β-CD will be administered as Trappsol® Cyclo 25% (250mg/mL) by slow intravenous infusion over a period of 8 up to 9 hours.

Trial Locations (1)

UCSF Benioff Children's Hospital Oakland, Oakland