Status:

RECRUITING

A Multicenter Selective Screening Study to Investigate the Frequency of Neuronal Ceroid Lipofuxinosis Type 2 (CLN2)

Lead Sponsor:

Nadir Hastalıkları Araştırma Derneği

Conditions:

Neuronal Ceroid Lipofuxinosis Type2 (CLN2)

Eligibility:

All Genders

2-6 years

Brief Summary

This study is a multicenter, non-drug screening study. Enrollment period is 12 months. There are no IMP to be followed or used in the study. Patients who applied to Pediatric Metabolism, Pediatric Neu...

Detailed Description

Demographic data, medical history and family history of the patients included in the study will be recorded at their first admission. In addition, seizure frequency; cognitive functions, including ass...

Eligibility Criteria

Inclusion

  • Girls and boys aged 6 years old
  • Having a history of at least one seizure
  • With a history of idiopathic seizures;
  • Speech disorder or regression in acquired speaking skills,
  • Motor dysfunctions,
  • Photoparoxysmal response to EEG with low-frequency IFS,
  • Observation of at least one of the symptoms or signs of cerebral atrophy or preventive white matter hyperintensity on MRI
  • Without hypoxic ischemic encephalopathy, head trauma and developmental brain anomalies
  • Not having been previously diagnosed with CLN2
  • The patient and/or his/her legal representative must be willing to sign the written consent form.

Exclusion

  • Patients younger than 2 years and older than 6 years
  • Patients with a known or diagnosed neurodegenerative disorder
  • Patients for whom written consent form cannot be obtained from their legal representative

Key Trial Info

Start Date :

September 6 2023

Trial Type :

OBSERVATIONAL

Allocation :

ESTIMATED

End Date :

March 1 2025

Estimated Enrollment :

750 Patients enrolled

Trial Details

Trial ID

NCT06128226

Start Date

September 6 2023

End Date

March 1 2025

Last Update

November 15 2024

Active Locations (34)

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Page 1 of 9 (34 locations)

1

Adana City Hospital, Pediatric Metabolism

Adana, Turkey (Türkiye)

2

Adana City Hospital, Pediatric Neurology

Adana, Turkey (Türkiye)

3

Adana Medical Park Hospital, Pediatric Neurology

Adana, Turkey (Türkiye)

4

Adana Şehir Hastanesi, Developmental Pediatrics

Adana, Turkey (Türkiye)