Skip to main content
OpenTrials
Completed

NCT Number: NCT04808297

Trehalose in Subjects With Neuronal Ceroid Lipofuscinoses

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Neuronal Ceroid-Lipofuscinoses Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipidoses Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Neurodegenerative Diseases Nutritional and Metabolic Diseases

Age range

7 year–45 year

Sex eligibility

All sexes

Study type

Observational

Primary location

IRCCS Fondazione Stella Maris

Pisa, PI, 56128, Italy

About this study

This study aims to collect clinical and laboratory data of patients with NCL taking Trehalose.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Primary outcomes

  1. 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

    Time frame: month 0, month 12, month 24

    Blood samples

Secondary outcomes

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

    Time frame: month 0, month 12, month 24

    Blood samples

Sponsors and collaborators

Lead sponsor

IRCCS Fondazione Stella Maris

Other

Registry information

Official study title

A Study in Subjects With Neuronal Ceroid Lipofuscinoses Taking Trehalose

Acronym: 3AL-CLN36

Important dates

Study start
2020
Primary completion
2021
Study completion
2021
First posted
Mar 22, 2021
Registry last updated
Apr 11, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.