CLN6 is a rare, neurodegenerative disease that causes progressive loss of acquired skills with motor delay, visual loss, seizures and ataxia. The investigators propose a natural history study of this rare disorder since it is currently unknown. It is important to understand disease progression in CLN6 disease to be able to judge therapeutic efficacy as emerging therapies like gene therapy become available.
Protocol Amendment History
6 amendments
This ClinicalTrials.gov record has been amended 6 times since 2017-09-15; most recent amendment 2025-07-28.
Status change: Recruiting → Active, Not Recruiting2025-07-28