Clinical Trial Update: Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1 [Phase 1, Phase 2]

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Summary

Phase 1/2 trial investigating combined intrathecal and intravenous VTS-270 for Niemann-Pick disease type C1 was terminated with results posted in 2022.

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Diseases / indications

• Niemann-pick Disease Type C1

Drugs mentioned

• VTS-270 (2-hydroxypropyl-beta-cyclodextrin)

Snippet

Sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Conditions: Niemann-Pick Disease, Type C1 | Phase 1, Phase 2 | Status: TERMINATED | Results posted: 2022-08-30