This is a study for intrathecal treatment of NPC
Study Details
Internal Title:
VTS-270 EXPANDED ACCESS USE AGREEMENT IT - Amendment 4
PCH IRB Number:
IRB-21-218
Clinical Trials Government Identifier:
N/A
Diagnoses & Treatments:
Neimann-Pick Disease Type C1, NPC, VTS-270 Cyclodextrin
Study Type:
Clinical Trial
Eligibility:
Ages 2months to 25years inclusive, weight of 7kg or higher, Ineligible for all currently enrolling interventional clinical trials for NPC being conducted in the USA, Diagnosis of NPC confirmed using genetic testing.
Enrollment Status:
Open
Phase:
Other