An Open-label Expanded Access Treatment Protocol for VTS-270 (2-hydroxypropyl- -cyclodextrin) in Patients with Neurologic Manifestations of Niemann-Pick Type C Disease (NPC) (VTS-270-001)

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An Open-label Expanded Access Treatment Protocol for VTS-270 (2-hydroxypropyl- -cyclodextrin) in Patients with Neurologic Manifestations of Niemann-Pick Type C Disease (NPC) (VTS-270-001)

Brief description of study

This is a prospective, open-label, treatment protocol to provide expanded access to VTS-270 to eligible Niemann-Pick type C disease (NPC) patients. Eligible patients will receive VTS-270 via intrathecal (IT) lumbar puncture (LP) every 2 weeks, and assessments of safety and exploratory efficacy measures will be performed at prescribed intervals.

Clinical Study Identifier: s18-00704

ClinicalTrials.gov Identifier: NCT02534844

Clinical Research Studies

An Open-label Expanded Access Treatment Protocol for VTS-270 (2-hydroxypropyl- -cyclodextrin) in Patients with Neurologic Manifestations of Niemann-Pick Type C Disease (NPC) (VTS-270-001)

Brief description of study

Recruitment Status

212-263-4432

646-754-7432