SLC6A1-NDD Prospective Longitudinal Natural History Study

Recruiting Observational Study
SLC6A1 Neurodevelopmental Disorder (NDD) Developmental and Epileptic Encephalopathies (DEE)
No Placebo Group Every participant receives an active treatment — no one gets a placebo. No Study Drug Researchers observe your health over time — no experimental treatment is given.
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At a Glance
Age
Up to 17
Sex
Any
Study type
Observational
Participants needed
60 (estimated)
Sponsor
UCB Biopharma SRL · Industry
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About This Trial

The overall objective of this prospective longitudinal natural history study is to collect clinical data to characterize and evaluate the natural course of SLC6A1-NDD and assess the feasibility of certain assessments for the purpose of conducting future clinical studies in patients with this disease.

Trial Locations
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Eligibility Criteria
Inclusion Criteria: * Patient with a diagnosis of SLC6A1-NDD characterized by epilepsy, global developmental delay, autism spectrum disorder, or intellectual disability, with a documented history of an SLC6A1 mutation, defined as pathogenic or likely pathogenic by the Investigator. * Patients shoul…
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