Trial Completed: Gait in Rare Diseases (NCT05161494)
WHY IT MATTERS
If successful, this research could lead to new treatment strategies for gait and movement problems in Tuberous Sclerosis Complex and STXBP1 Encephalopathy—conditions where walking difficulties significantly impact quality of life but have limited treatment options.
Researchers completed a study looking at whether treatments that help with walking problems in one rare genetic disorder (Dravet Syndrome) could also help people with two other rare genetic disorders: Tuberous Sclerosis Complex and STXBP1 Encephalopathy. The study enrolled 41 people total and aimed to see if the same approach to treating movement and walking difficulties caused by epilepsy and developmental delays could work across different genetic conditions.
NCT ID: NCT05161494 Status: COMPLETED Conditions: Tuberous Sclerosis, STXBP1 Encephalopathy With Epilepsy Enrollment: 41 Sponsor: Universiteit Antwerpen Summary: The aim of this pilot study is to explore whether the knowledge and experience gained during the T-GaiD project (Treatment of Gait Disorders in Dravet Syndrome - NCT03857451) can be transferred to other populations with similar problems, i.e. motor and gait problems as a result of a genetic disorder characterized by epilepsy and developmental delay. In this pilot study, 40 people with Tuberous Sclerosis Complex and 30 people with STXBP1 will be recruited via the Antwerp University Hospital and i