VTS-270

DRUG5 trials

Sponsors

Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company, Washington University School of Medicine, Mandos LLC, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Conditions

Niemann-Pick Disease, Type CNiemann-Pick Disease, Type C1

Phase 1

Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease

CompletedNCT01747135

Vtesse, LLC, a Mallinckrodt Pharmaceuticals CompanyNiemann-Pick Disease, Type C1

Start: 2013-01-31End: 2017-03-31Updated: 2021-07-23

Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C

CompletedNCT03471143

Washington University School of MedicineNiemann-Pick Disease, Type C

Start: 2019-02-22End: 2024-10-04Updated: 2026-01-05

Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1

TerminatedNCT03887533

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)Niemann-Pick Disease, Type C1

Start: 2020-01-06End: 2021-10-25Updated: 2022-08-30

Phase 2

Safety and Tolerability Study of VTS-270 in Pediatric Participants With Niemann-Pick Type C (NPC) Disease

WithdrawnNCT03687476

Vtesse, LLC, a Mallinckrodt Pharmaceuticals CompanyNiemann-Pick Disease, Type C

Start: 2020-05-31End: 2022-10-30Updated: 2020-07-22

Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1

TerminatedNCT03879655

Mandos LLCNiemann-Pick Disease, Type C

Start: 2019-12-02End: 2021-10-18Updated: 2023-12-26

Related Papers

Convergent Validity of the Fine Motor, Speech, and Cognitive Domains of the 5-Domain Niemann-Pick Disease Type C Clinical Severity Scale

Journal of Child Neurology2025-06-171 citations

Long-Term Neuropsychological Outcomes from an Open-Label Phase I/IIa Trial of 2-Hydroxypropyl-β-Cyclodextrins (VTS-270) in Niemann-Pick Disease, Type C1.

2019-07-0129 citations