Date: 2017-03-15
Type of
information: Completion of patient enrollment
phase: 2-3
Announcement: completion of patient enrollment
Company: Vtesse (USA - MD)
Product: VTS-270 (2-hydroxypropyl-?-cyclodextrin)
Action
mechanism:
- VTS-270 is a well-characterized mixture of (2-hydroxypropyl)-beta-cyclodextrin that has been extensively evaluated in pre-clinical and clinical studies at NIH, as well as under individual compassionate use investigational new drug applications (iINDs) and in other academic labs.
Disease: Niemann-Pick disease type C1
Therapeutic
area: Rare diseases - Genetic diseases
Country: Australia, France, Germany, Spain, Turkey, UK, USA
Trial
details:
- This study evaluates the efficacy and safety of 2-hydroxypropyl-?-cyclodextrin (VTS-270) in patients with neurologic manifestations of Niemann-Pick Type C1 (NPC1) Disease. Approximately two-thirds of patients will receive the study drug, 2-hydroxypropyl-?-cyclodextrin (HP-?-CD), while the remaining study participants will receive sham control. (NCT02534844)
Latest
news:
- • On March 15, 2017, Vtesse announced that its registrational study of investigational drug VTS-270 in Niemann-Pick Type C1 disease (NPC) is fully enrolled. Vtesse has engaged 20 clinical trial sites across the globe, in the United States, Germany, the United Kingdom, France, Spain, Australia, and Turkey, enabling broad access for study-eligible patients worldwide. Upon completion of the pivotal phase of the trial, Vtesse will continue to provide access to VTS-270 for the clinical trial participants through an open-label extension study. Additionally, Vtesse has initiated a device development program to eliminate the need for lumbar punctures for administration of VTS-270.
Is
general: Yes
