A Phase 2b/3 Prospective, Randomized, Double-Blind, Sham-Controlled Trial of VTS-270 (2-hydroxypropyl-a-cyclodextrin) in Subjects with Neurologic Manifestations of Niemann-Pick Type C1 (NPC1) Disease

This Study is
No Longer Enrolling

Details
Age

Adult

Phase

II/III - A combination of phases: (2) Research Studies that gather preliminary data on whether a drug works in people who have a certain condition/disease (that is, the drug's effectiveness). For example, participants receiving the drug may be compared to similar participants receiving a different treatment, usually an inactive substance (called a placebo) or a different drug. Safety continues to be evaluated, and short-term adverse events are studied. and (3) Research Studies that gather more information about a drug's safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. These studies typically involve more participants.

Type of Study

Treatment

Locations

University of Colorado Hospital

Study ID

Protocol Number: 18-0997

More information available at ClinicalTrials.gov: NCT02534844

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