Attention NPC patients! There is a new opportunity being presented by Vtesse for patient involvement. The NPC Study is a Phase 2b/3 randomized, sham-controlled clinical trial designed to evaluate the efficacy and safety of VTS-270 (a proprietary form of cyclodextrin) for the treatment of children with Niemann-Pick Disease Type C (NPC). Its primary objective is to evaluate the progression of the neurologic manifestations of NPC in children treated with VTS-270 compared to those who do not receive the drug.

The NPC Study will be conducted at multiple sites in the U.S. and Europe beginning in September. It is open to children ages 4-21 who have been diagnosed with NPC and meet all the entry requirements. Two-thirds of the participants will receive VTS-270 through a lumbar puncture and the other third will undergo a sham procedure (a needle prick that does not penetrate beyond the skin), both of which will be given under either general anesthesia or conscious sedation (a minimally depressed level of consciousness that retains the patient’s ability to independently and continuously maintain an airway and respond appropriately to physical stimulation or verbal command and that is produced by a pharmacological or non-pharmacological method or a combination thereof). Use of either general anesthesia or conscious sedation will be based upon an individual study site’s standard practice. After 12 months of bi-weekly injections or sham control procedures, all participants will be eligible to receive VTS-270 in the open-label extension phase of the trial until the regulatory agencies make a decision whether or not to approve VTS-270. In addition, any participants in the sham group who shows significant disease progression will be removed from the sham group and placed in the open-label part of the trial where they are guaranteed to receive VTS-270.

Please complete the form on the Trials Enrollment page if you would like us to contact you about your interest in The NPC Study.