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Intrathecal 2-Hydroxypropyl-ß-cyclodextrin for Neurological Decline in Patients with Niemann-Pick Disease Type C1 Open


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


Coordinator Contact: HETVI MEHTA
Contact Email:


Principal Investigator