Date: 2015-08-06
Type of
information: Interim results
phase: 1
Announcement: interim results
Company: Vtesse (USA - MD)
Product: VTS-270
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 C
Therapeutic
area: Rare diseases - Genetic diseases
Country: USA
Trial
details: (NCT01747135)
Latest
news:
- • On August 6, 2015, Vtesse announced preliminary results today from an open-label Phase 1 clinical trial with VTS-270 (a formulation of (2-hydroxypropyl)-beta-cyclodextrin) for treatment of Niemann-Pick Disease Type C (NPC) conducted by researchers at the National Institutes of Health (NIH) Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD). Preliminary analyses, conducted post-hoc, suggest that the rate of disease progression had slowed down (based on a standardized measure) in children treated with VTS-270 in the Phase 1 trial as compared to the rate in an age- and disease severity-matched cohort obtained from a separate natural history study of NPC patients. The analyses also show that children treated with VTS-270 demonstrated improvement on several disease domains.
- Researchers have matched participants in the Phase 1 study to individuals from the natural history data set according to baseline age and disease severity. The rate of disease progression of this matched cohort was compared to the rate of disease progression of children treated with VTS-270 in the Phase 1 study. In the Phase 1 trial, among the 12 children treated with direct administration of VTS-270 into the cerebrospinal fluid via an intrathecal administration for more than six months and up to 12 months, the overall NPC score showed a slowing down in the rate of decline. In these patients, when the scores for impact on hearing were removed, the NPC score showed disease stabilization and halting of progression. Domains of cognition and speech have shown improvement of the disease state for participants in the study; while ambulation, fine motor skills, cognition, swallowing, and memory demonstrate slowing down of decline. Measurements of eye movement and hearing appeared to have worsened among participants in the Phase 1 trial.
- In the Phase 1 trial to date, VTS-270 has been well tolerated other than observed worsening in the eye movement and hearing of some trial participants. Clinicians have administered more than 250 intrathecal administrations of the drug to study participants thus far with minimal administration-related side effects. Vtesse plans to submit more complete Phase 1 clinical trial results for presentation at a scientific meeting later this year.
Is
general: Yes
