CompletedNot Applicable

Trehalose in Subjects With Neuronal Ceroid Lipofuscinoses

Italy8 participantsStarted 2020-08-01

Plain-language summary

Neuronal Ceroid Lipofuscinoses (NCL) or Batten's disease are the most common juvenile neurodegenerative disease, characterized by early blindness, movement disorders, cognitive and behavioral impairment, epilepsy, and retinopathy. This study aims to collect clinical and laboratory data of patients with NCL taking Trehalose.

Who can participate

Age range7 Years – 45 Years

SexALL

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Inclusion Criteria: * NCL genetic diagnosis (mutation in genes CLN3 or CLN6) * Signed informed consent Exclusion Criteria: * Other concomitant neurodegenerative diseases. * Therapeutic and eating changes in the last four months prior to the study * Unstable clinical conditions (myoclonus worsening, instability in sleep, parenteral nutrition) * Refusal to sign the informed consent

What they're measuring

Change from Baseline clinical status assessed using Annex VII of the Unified Batten Disease Rating Scale (UBDRS) (score 0-5 where 5 is the worst outcome) at 12 and 24 months

Timeframe: month 0, month 12, month 24

Trial details

NCT IDNCT04808297

SponsorIRCCS Fondazione Stella Maris

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2021-07-01

View on ClinicalTrials.gov More Neuronal Ceroid-Lipofuscinoses trials