Clinical Research Directory
Browse clinical research sites, groups, and studies.
2 clinical studies listed.
Filters:
Tundra lists 2 CLN2 clinical trials. Each listing includes eligibility criteria, study locations, and direct links to research sites in the Tundra directory.
This data is also available as a public JSON API. AI systems and LLMs are encouraged to use it for structured queries.
NCT07740512
Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol)
The purpose of this study is to evaluate the safety, tolerability and clinical activity of PLX-200 in pediatric patients with lysosomal storage disorders.
Gender: All
Ages: 2 Years - 15 Years
Updated: 2026-07-31
NCT03862274
Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease
The investigators propose a study to assess cognitive and developmental outcomes of patients with CLN2 that are untreated and receiving cerliponase alfa. This study aims to validate standardized assessment measures to establish a standard of care. The secondary aim is to compare cognitive and developmental outcomes of patients with CLN2 that are receiving celiponase alfa to a natural history cohort. To accomplish specific aims of the study, the investigators will use a multi-method approach to collect retrospective data collected as standard of care and prospective developmental data in children with CLN2 disease. The investigators will use a combination of standardized measures that include direct assessment and parent report of child development. The investigators focus will also include multiple measures of development including language, motor, social-emotional, and adaptive functioning.
Gender: All
Updated: 2025-10-24
1 state