RecruitingNCT06128226

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

A Multicenter Selective Screening Study to Investigate the Frequency of Neuronal Ceroid Lipofuxinosis Type 2 (CLN2) in the Presence of Nonspecific Neurological Findings Accompanying Seizures Between the Ages of 2 and 6


Sponsor

Nadir Hastalıkları Araştırma Derneği

Enrollment

750 participants

Start Date

Sep 6, 2023

Study Type

OBSERVATIONAL

Conditions

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 Neurology and Developmental Pediatrics clinics with the symptoms or findings defined in the protocol as below for 12 months will be included in the study. Children between the ages of 2 and 6, without hypoxic ischemic encephalopathy, head trauma and developmental brain anomalies, who are admitted to the Pediatric Metabolism, Pediatric Neurology and Developmental Pediatrics clinics with non-specific neurological symptoms such as idiopathic seizures of unknown etiology, speech disorders and motor dysfunctions, will constitute the target population of the study.


Eligibility

Min Age: 2 YearsMax Age: 6 Years

Plain Language Summary

Simplified for easier understanding

This Turkish screening study is trying to find how common a rare inherited brain disease called Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) is among young children presenting with unexplained seizures, speech problems, or movement difficulties. CLN2 is caused by a deficiency of an enzyme called Tripeptidyl Peptidase 1 and leads to progressive neurological decline — early diagnosis is critical for accessing treatments that can slow the disease. Children aged 2 to 6 who have had at least one seizure of unknown cause along with speech regression, motor problems, or specific abnormal brain scan or EEG findings — and who do not have other known brain conditions — are eligible. Participation involves an enzyme blood test and, if the enzyme is low, genetic testing, with no experimental medications involved in this diagnostic screening study. This summary was prepared with AI assistance to help patients understand the study in plain language.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

GENETICBlood Sampling

For Tripeptidyl Peptidase 1 enzyme level measurement, blood will be taken from the patient into a 5 mL EDTA tube at Gazi University Faculty of Medicine, Metabolism Laboratory, and for patients with low enzyme activity, the genetic evaluation test will be performed with whole blood at the Gene2Info Laboratory.


Locations(34)

Adana City Hospital, Pediatric Metabolism

Adana, Turkey (Türkiye)

Adana City Hospital, Pediatric Neurology

Adana, Turkey (Türkiye)

Adana Medical Park Hospital, Pediatric Neurology

Adana, Turkey (Türkiye)

Adana Şehir Hastanesi, Developmental Pediatrics

Adana, Turkey (Türkiye)

Başkent University School of Medicine, petiatric Metabolism

Adana, Turkey (Türkiye)

Seyhan State Hospital, Pediatric Neurology

Adana, Turkey (Türkiye)

Çukurova University School of Medicine, Pediatric Metabolism

Adana, Turkey (Türkiye)

Çukurova University School of Medicine, Pediatric Neurology

Adana, Turkey (Türkiye)

Batman Medical Point Hospital, Pediatric Neurology

Batman, Turkey (Türkiye)

Batman Training and Research Hospital, Pediatric Neurology

Batman, Turkey (Türkiye)

Dicle University School of Medicine, Pediatric Neurology

Diyarbakır, Turkey (Türkiye)

Diyarbakır Gazi Yaşargil Training and Research Hospital, Developmental Pediatrics

Diyarbakır, Turkey (Türkiye)

Diyarbakır Gazi Yaşargil Training and Research Hospital, Pediatric Neurology

Diyarbakır, Turkey (Türkiye)

Elazığ Fethi Tekin City Hospital

Elâzığ, Turkey (Türkiye)

Fırat University School of Medicine, Pediatric Metabolism

Elâzığ, Turkey (Türkiye)

Gaziantep Cengiz Göçek Child's Hospital, Developmental Pediatrics

Gaziantep, Turkey (Türkiye)

Gaziantep Cengiz Göçek Child's Hospital, Pediatric Metabolism

Gaziantep, Turkey (Türkiye)

Gaziantep Cengiz Göçek Child's Hospital, Pediatric Neurology

Gaziantep, Turkey (Türkiye)

Gaziantep University School of Medicine, Pediatric Neurology

Gaziantep, Turkey (Türkiye)

Hatay Training and Research Hospital, Pediatric Neurology

Hatay, Turkey (Türkiye)

Necip Fazıl City Hospital, Pediatric Neurology

Kahramanmaraş, Turkey (Türkiye)

Sütçü İmam University School of Medicine, Pediatric Neurology

Kahramanmaraş, Turkey (Türkiye)

Malatya İnönü University School of Medicine, Developmental Pediatrics

Malatya, Turkey (Türkiye)

Malatya İnönü University School of Medicine, Pediatric Neurology

Malatya, Turkey (Türkiye)

Malatya Training and Reserach Hospital, Pediatric Neurology

Malatya, Turkey (Türkiye)

Mardin Artuklu University School of Medicine, Pediartic Endocrinology

Mardin, Turkey (Türkiye)

Mardin Artuklu University School of Medicine, Pediatric Neurology

Mardin, Turkey (Türkiye)

Mersin City Hospital, Pediatric Metabolism

Mersin, Turkey (Türkiye)

Mersin City Hospital, Pediatric Neurology

Mersin, Turkey (Türkiye)

Mersin City Hospital

Mersin, Turkey (Türkiye)

Mersin University School of Medicine, Pediatric eurology

Mersin, Turkey (Türkiye)

Şanluurfa Harran University School of Medicine, Pediatric Metabolism

Sanliurfa, Turkey (Türkiye)

Şanlıurfa Training and Research Hospital, Pediatric Neurology

Sanliurfa, Turkey (Türkiye)

Van Regional Training and Research Hospital

Van, Turkey (Türkiye)

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NCT06128226