Clinical Trials

Date: 2015-09-08

Type of information: Initiation of patient enrollment

phase: 2b-3

Announcement: initiation of patient enrollment

Company: Sucampo Pharmaceuticals (USA - MA)

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 the National Institutes of Health, 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:

• The trial is a Phase 2b/3 prospective, randomized, double-blind, sham-controlled trial. The 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 September 28, 2015, Sucampo announced that the first three patients have been screened for inclusion in its pivotal Phase 2b/3 clinical trial with VTS-270 for treatment of Niemann-Pick Type C1 Disease (NPC). This clinical trial follows a Phase 1 study conducted by researchers at the National Institutes of Health (NIH) Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD). The trial will be conducted in patients affected by NPC disease. The trial will take place in up to 20 centers in the United States and Europe. It is a three-part, efficacy and safety trial of VTS-270, administered by the lumbar intrathecal route (IT) every two weeks, with a planned enrollment of approximately 51 patients (in Parts A and B). In Part A of the study, researchers will evaluate three different dose levels of VTS-270 versus sham-control to determine the dose level for Parts B and C. All participants in the pivotal trial will be eligible to receive treatment with VTS-270 in Part C, the open-label extension, until the time of regulatory decisions.
• Preliminary analyses of the Phase 1 trial, conducted post-hoc, suggest that the rate of disease progression had slowed down (based on a standardized measure) in children treated with VTS-270 as compared to the rate in an age- and disease severity-matched cohort obtained from a separate natural history study of NPC patients.
• The VTS-270 pivotal study addresses a pressing need to discover new treatments for the disease, according to Forbes D. Porter, M.D., Ph.D., Co-Principal Investigator of the Phase 2b/3 study, Senior Investigator and Program Head in the intramural research program of the Developmental Endocrinology and Genetics Program (PDEGEN), NICHD. He added that the trial has the potential to provide important information about the disease as well as how patients may tolerate VTS-270 and its potential effects on the symptoms of NPC.

Is general: Yes