Actively Recruiting

Age: 2Years - 6Years
All Genders
ID06128226

A Multicenter Selective Screening Study to Investigate the Frequency of Neuronal Ceroid Lipofusinosis Type 2 (CLN2) in Children Aged 2 to 6 With Nonspecific Neurological Symptoms and Seizures

Led by Nadir Hastalıkları Araştırma Derneği · Updated on 2024-11-15

750

Participants Needed

34

Research Sites

8 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

Researchers are investigating the frequency of Neuronal Ceroid Lipofusinosis Type 2 (CLN2) in children aged 2 to 6 years who have nonspecific neurological symptoms such as idiopathic seizures, speech disorders, and motor dysfunctions. This multicenter, non-drug screening study focuses on children without hypoxic ischemic encephalopathy, head trauma, or developmental brain anomalies, aiming to better understand the demographic and clinical features of those with possible CLN2 disease. Children first undergo assessments including recording demographic and medical history, seizure frequency, cognitive and language development evaluations, physical exams assessing muscle strength, gait, and coordination, as well as neurological evaluations using EEG and MRI scans. Those showing specific signs like speech disorder with seizures, movement problems, particular EEG responses, or MRI findings will have blood samples taken to measure Tripeptidyl Peptidase 1 enzyme levels. If enzyme activity is low, genetic testing is performed to investigate CLN2 disease. Participants are involved for up to one year during which various clinical, neurological, and imaging evaluations are done. Blood samples are collected for enzyme and genetic analyses. The main outcome measured is the frequency of CLN2 disease in this group. The study tracks each child's symptoms, neurological findings, and imaging results to better identify and understand CLN2 in children with these symptoms.

CONDITIONS

Brief Title

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

Who Can Participate

Age: 2Years - 6Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Children aged 2 to 6 years
  • History of at least one seizure
  • Idiopathic seizures accompanied by speech disorder or regression in speaking skills, motor dysfunctions, photoparoxysmal response to EEG with low-frequency IFS, or signs of cerebral atrophy or white matter hyperintensity on MRI
  • No history of hypoxic ischemic encephalopathy, head trauma, or developmental brain anomalies
  • Not previously diagnosed with CLN2
  • Willingness of patient or legal representative to sign written consent
Not Eligible

You will not qualify if you...

  • Younger than 2 years or older than 6 years
  • Known or diagnosed neurodegenerative disorder
  • Inability to obtain written consent from legal representative

AI-Screening

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Diagnostic Evaluation

Duration - Up to 1 year

Participants undergo neurological and visual assessments including EEG and MRI, along with blood sampling for enzyme level measurement and genetic testing if needed to investigate CLN2 disease.

1 to 2 visits depending on test results

Long-term Monitoring

Duration - Up to 1 year

Participants with neurological symptoms are observed to determine the frequency of CLN2 disease and to document clinical characteristics over time.

Follow-up visits as scheduled by the study

Trial Site Locations

Total: 34 locations

1

Adana City Hospital, Pediatric Metabolism

Adana, Turkey (Türkiye)

Actively Recruiting

2

Adana City Hospital, Pediatric Neurology

Adana, Turkey (Türkiye)

Actively Recruiting

3

Adana Medical Park Hospital, Pediatric Neurology

Adana, Turkey (Türkiye)

Actively Recruiting

4

Adana Şehir Hastanesi, Developmental Pediatrics

Adana, Turkey (Türkiye)

Actively Recruiting

5

Başkent University School of Medicine, petiatric Metabolism

Adana, Turkey (Türkiye)

Actively Recruiting

6

Seyhan State Hospital, Pediatric Neurology

Adana, Turkey (Türkiye)

Actively Recruiting

7

Çukurova University School of Medicine, Pediatric Metabolism

Adana, Turkey (Türkiye)

Actively Recruiting

8

Çukurova University School of Medicine, Pediatric Neurology

Adana, Turkey (Türkiye)

Actively Recruiting

9

Batman Medical Point Hospital, Pediatric Neurology

Batman, Turkey (Türkiye)

Actively Recruiting

10

Batman Training and Research Hospital, Pediatric Neurology

Batman, Turkey (Türkiye)

Actively Recruiting

11

Dicle University School of Medicine, Pediatric Neurology

Diyarbakır, Turkey (Türkiye)

Actively Recruiting

12

Diyarbakır Gazi Yaşargil Training and Research Hospital, Developmental Pediatrics

Diyarbakır, Turkey (Türkiye)

Actively Recruiting

13

Diyarbakır Gazi Yaşargil Training and Research Hospital, Pediatric Neurology

Diyarbakır, Turkey (Türkiye)

Actively Recruiting

14

Elazığ Fethi Tekin City Hospital

Elâzığ, Turkey (Türkiye)

Actively Recruiting

15

Fırat University School of Medicine, Pediatric Metabolism

Elâzığ, Turkey (Türkiye)

Actively Recruiting

16

Gaziantep Cengiz Göçek Child's Hospital, Developmental Pediatrics

Gaziantep, Turkey (Türkiye)

Actively Recruiting

17

Gaziantep Cengiz Göçek Child's Hospital, Pediatric Metabolism

Gaziantep, Turkey (Türkiye)

Actively Recruiting

18

Gaziantep Cengiz Göçek Child's Hospital, Pediatric Neurology

Gaziantep, Turkey (Türkiye)

Actively Recruiting

19

Gaziantep University School of Medicine, Pediatric Neurology

Gaziantep, Turkey (Türkiye)

Actively Recruiting

20

Hatay Training and Research Hospital, Pediatric Neurology

Hatay, Turkey (Türkiye)

Actively Recruiting

21

Necip Fazıl City Hospital, Pediatric Neurology

Kahramanmaraş, Turkey (Türkiye)

Actively Recruiting

22

Sütçü İmam University School of Medicine, Pediatric Neurology

Kahramanmaraş, Turkey (Türkiye)

Actively Recruiting

23

Malatya İnönü University School of Medicine, Developmental Pediatrics

Malatya, Turkey (Türkiye)

Actively Recruiting

24

Malatya İnönü University School of Medicine, Pediatric Neurology

Malatya, Turkey (Türkiye)

Actively Recruiting

25

Malatya Training and Reserach Hospital, Pediatric Neurology

Malatya, Turkey (Türkiye)

Actively Recruiting

26

Mardin Artuklu University School of Medicine, Pediartic Endocrinology

Mardin, Turkey (Türkiye)

Actively Recruiting

27

Mardin Artuklu University School of Medicine, Pediatric Neurology

Mardin, Turkey (Türkiye)

Actively Recruiting

28

Mersin City Hospital, Pediatric Metabolism

Mersin, Turkey (Türkiye)

Actively Recruiting

29

Mersin City Hospital, Pediatric Neurology

Mersin, Turkey (Türkiye)

Actively Recruiting

30

Mersin City Hospital

Mersin, Turkey (Türkiye)

Actively Recruiting

31

Mersin University School of Medicine, Pediatric eurology

Mersin, Turkey (Türkiye)

Actively Recruiting

32

Şanluurfa Harran University School of Medicine, Pediatric Metabolism

Sanliurfa, Turkey (Türkiye)

Actively Recruiting

33

Şanlıurfa Training and Research Hospital, Pediatric Neurology

Sanliurfa, Turkey (Türkiye)

Actively Recruiting

34

Van Regional Training and Research Hospital

Van, Turkey (Türkiye)

Actively Recruiting

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Research Team

G

Güneş Oymak

How is the study designed?

Study Type

OBSERVATIONAL

Masking

N/A

Allocation

N/A

Model

N/A

Primary Purpose

N/A

Number of Arms

0

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