International study being done at Dell Children’s for Niemann-Pick disease reaches full enrollment

Pam Andrews, her husband Chris Andrews, and their two children Belle, 7, and Abby, 2, color together in their home in Southwest Austin last May. RESHMA KIRPALANI / AMERICAN-STATESMAN

Dell Children’s Medical Center of Central Texas has been participating in an international study testing a treatment for Niemann-Pick Type C1 disease called VTS-270. We wrote about the trial in November, when we told the story of Belle and Abby Andrews, who had been traveling to Chicago every other week for the treatment but since after Thanksgiving have been able to have some of those treatments done locally.

Today, Vtesse, the maker of VTS-270 announced it had finished enrolling all the participants in the study. It needed at least 51 to be statistically relevant.

“Completing enrollment in the pivotal Phase 2b/3 clinical trial of VTS-270 is a significant milestone for the NPC community and Vtesse, and we are fortunate to have exceeded our goal of 51 participants,” said Ben Machielse, Vtesse’s president and chief executive officer in a press release. “We look forward to generating topline results from the study early next year.”

Niemann-Pick Type C1 disease is a rare genetic disorder sometimes called childhood Alzheimer’s. Cholesterol in brain cells builds up, damaging the cells and eventually causing them to die.

Children who have NPC1 usually experience clumsiness, shakiness of the hands and poor fine motor skills; problems with short-term memory, thinking and learning skills; hearing loss; problems swallowing; and trouble looking up with their eyes. They also might have seizures and eventually lose the ability to do school work, talk and walk.

 

The VTS-270 study is being done at 20 sites worldwide and has been fast-tracked by the FDA. Once all the participants have been in the study for at least a year, the families will be told whether or not their children received VTS-270 or the placebo, which about a third received. After a year of being in the study, though, all the kids receive the actual treatment and are guaranteed to receive the treatment through an extension of the study.

Belle Andrews, who turned 7 this month, will finish the first year of study in May, at which point, she will then be given VTS-270 if she was receiving the placebo, something they won’t know until all the participants have finished the study. Abby, 2, was too young to be part of the study, but has been receiving the treatment through expanded access, which also is called compassionate use.

The family launched its Firefly Fund nonprofit last month to accelerate finding a cure for Niemann-Pick Type C1 disease.


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