Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients

PHASE1/PHASE2CompletedINTERVENTIONAL

Enrollment

Participants

Timeline

Start Date

March 20, 2017

Primary Completion Date

March 3, 2021

Study Completion Date

March 3, 2021

Conditions

Niemann-Pick Disease, Type C1

Interventions

DRUG

Hydroxypropyl-beta-cyclodextrin

Used in the treatment of Niemann-Pick C1

Trial Locations (5)

Soroka Mc, Beersheba

HaeMek MC, Afula

Karolinska Trial Alliance, Solna

Salford Royal Hospital, Salford

Leonard Wolfson Experimental Neurology Centre, London