MRI in Pediatric Inherited Neurodegnerative Changes

NCT ID: NCT06682624

Last Updated: 2024-11-19

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

NOT_YET_RECRUITING

Total Enrollment

40 participants

Study Classification

OBSERVATIONAL

Study Start Date

2025-01-31

Study Completion Date

2026-12-31

Brief Summary

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This study aims to evaluate the diagnostic capability of brain MRI in establishing pattern recognition approach in pediatric inherited neurodegenerative disorders.

Therefore, taking into consideration specific MRI findings, we aim to highlight the potential of MRI to predict the diagnosis of pediatric inherited neurodegenerative diseases.

Detailed Description

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Pediatric inherited neurodegenerative diseases are non-homogenous group of diseases caused by inborn errors of metabolism (IEMs) that mainly impact the central nervous system. They typically affect newborns and infants . Genetic defects causing specific enzyme deficiencies that lead to deficiency or toxic accumulation of essential metabolites with specific biochemical and molecular abnormalities are the cause of these diseases .

Inborn errors of metabolism are uncommon, which leads to a lack of experience for most neuroradiologists to diagnose these disorders easily. Moreover, most of these disorders manifest in nonspecific manner, creating a diagnostic difficulty for pediatricians, neurologists, and geneticists .

Clinical presentation may be confusing and potentially lead to a delay in the diagnosis and treatment. Incidence of these disorders may vary from one region to another, being higher in communities with consanguineous marriages, ranging from 1.2 to 2 per 100,000 live births. Most exhibit an autosomal recessive mode of inheritance and fewer exhibit an X-linked mode of inheritance.

Multiple methods can be used to classify neurometabolic diseases according to their clinical presentation, biochemical features, impacted cellular organelle, or involved location of the brain.

As a non-invasive means, neuroimaging approaches play a major role in increasing diagnostic accuracy and patient follow-up in IEM by assessing the timing, degree, reversibility, and brain injury.

Imaging based classification includes leukodystrophy (primary involvement of white matter due to genetic abnormality), leukoencephalopathy (secondary involvement of white matter either due to genetic or acquired systemic disorder), poliodystrophy (predominant involvement of grey matter), and pandystrophy (mixed involvement of both white and grey matter).

White matter of the central nervous system is usually affected due to many pathological processes such as delayed myelination (myelin maturation delayed for expected age), hypomyelination (scarcity of myelin or arrest in myelination process), dysmyelination (deposition of abnormally composed fragile myelin), demyelination (secondary loss of myelin that may have been previously normal) and myelinopathy (vacuolation due to deranged brain iron and water hemostasis).

The preferred method for evaluating pediatric inherited neurodegenerative disorders is magnetic resonance imaging (MRI). Examining MRI patterns and clinical indicators aids in narrowing the differential and customizing further laboratory (focused metabolomics) or genetic research.

Magnetic resonance imaging (MRI) can be useful and even crucial for prompt treatment before the receipt of expensive and time-consuming results from genetic or biochemical tests.

Conditions

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Pediatric Neurological Disorder

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Interventions

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MRI

Magnetic Resonance Imaging in recognition of pediatric neurodegenerative disease patterns

Intervention Type DEVICE

Eligibility Criteria

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Inclusion Criteria

* Patients with clinical suspicion of neuro-developmental delay and metabolic disease consulted for or referred to pediatric neurology.
* Patients known to have inherited neurodegenerative diseases under follow-up.

Exclusion Criteria

\- Patients with prior histories of perinatal ischemia or stroke, hypoxic-ischemic encephalopathy, birth trauma or accident, or known systemic diseases (congenital heart disease, renal failure, or autoimmune disorders) will be excluded.
Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Sohag University

OTHER

Sponsor Role lead

Responsible Party

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Yasmin Asem Abulelwafa

assisstant lecturer

Responsibility Role PRINCIPAL_INVESTIGATOR

Locations

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Sohag University

Sohag, Sohag Governorate, Egypt

Site Status

Countries

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Egypt

Central Contacts

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Yasmin Asem Abu Elwafa Elsayed, Assistant lecturer

Role: CONTACT

+02 01007680891

Hisham Amein Yousef, Assistant professor

Role: CONTACT

+0201151152564

Facility Contacts

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Yasmin Asem Abu Elwafa Elsaid, Assistant Lecturer

Role: primary

+0201007680891

Hisham Amein Yousef, Assistant professor

Role: backup

+0201151152564

Nahla Mohamed Ali Hasan, professor

Role: backup

Ahmed Adel Hasan El Beheiry, Assistant professor

Role: backup

References

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van der Knaap MS, Schiffmann R, Mochel F, Wolf NI. Diagnosis, prognosis, and treatment of leukodystrophies. Lancet Neurol. 2019 Oct;18(10):962-972. doi: 10.1016/S1474-4422(19)30143-7. Epub 2019 Jul 12.

Reference Type BACKGROUND
PMID: 31307818 (View on PubMed)

Datar R, Prasad AN, Tay KY, Rupar CA, Ohorodnyk P, Miller M, Prasad C. Magnetic resonance imaging in the diagnosis of white matter signal abnormalities. Neuroradiol J. 2018 Aug;31(4):362-371. doi: 10.1177/1971400918764016. Epub 2018 Mar 8.

Reference Type BACKGROUND
PMID: 29517408 (View on PubMed)

Loftus JR, Puri S, Meyers SP. Multimodality imaging of neurodegenerative disorders with a focus on multiparametric magnetic resonance and molecular imaging. Insights Imaging. 2023 Jan 16;14(1):8. doi: 10.1186/s13244-022-01358-6.

Reference Type BACKGROUND
PMID: 36645560 (View on PubMed)

Lim YT, Mankad K, Kinali M, Tan AP. Neuroimaging Spectrum of Inherited Neurotransmitter Disorders. Neuropediatrics. 2020 Feb;51(1):6-21. doi: 10.1055/s-0039-1698422. Epub 2019 Oct 21.

Reference Type BACKGROUND
PMID: 31634934 (View on PubMed)

Lai LM, Gropman AL, Whitehead MT. MR Neuroimaging in Pediatric Inborn Errors of Metabolism. Diagnostics (Basel). 2022 Mar 30;12(4):861. doi: 10.3390/diagnostics12040861.

Reference Type BACKGROUND
PMID: 35453911 (View on PubMed)

Alamri A, Aljadhai YI, Alrashed A, Alfheed B, Abdelmoaty R, Alenazi S, Alhashim A, Benini R. Identifying Clinical Clues in Children With Global Developmental Delay / Intellectual Disability With Abnormal Brain Magnetic Resonance Imaging (MRI). J Child Neurol. 2021 May;36(6):432-439. doi: 10.1177/0883073820977330. Epub 2020 Dec 9.

Reference Type BACKGROUND
PMID: 33295251 (View on PubMed)

Soni N, Ora M, Bathla G, Nagaraj C, Boles Ponto LL, Graham MM, Saini J, Menda Y. Multiparametric magnetic resonance imaging and positron emission tomography findings in neurodegenerative diseases: Current status and future directions. Neuroradiol J. 2021 Aug;34(4):263-288. doi: 10.1177/1971400921998968. Epub 2021 Mar 5.

Reference Type BACKGROUND
PMID: 33666110 (View on PubMed)

Wenger KJ, Koldijk CE, Hattingen E, Porto L, Kurre W. Characterization of MRI White Matter Signal Abnormalities in the Pediatric Population. Children (Basel). 2023 Jan 24;10(2):206. doi: 10.3390/children10020206.

Reference Type BACKGROUND
PMID: 36832335 (View on PubMed)

Al Orf A, Waheed KB, Ali EM, Muhammad AK, Al Zahrani FM, Seth SA, Al Jubair NN, Harisi HS, Arulanantham ZJ. Inherited paediatric neurometabolic disorders, can brain magnetic resonance imaging predict? Neurosciences (Riyadh). 2020 Oct;25(5):392-398. doi: 10.17712/nsj.2020.5.20200072.

Reference Type BACKGROUND
PMID: 33459289 (View on PubMed)

Aksoy DO, Alkan A. Neurometabolic Diseases in Children: Magnetic Resonance Imaging and Magnetic Resonance Spectroscopy Features. Curr Med Imaging Rev. 2019;15(3):255-268. doi: 10.2174/1573405613666171123152451.

Reference Type BACKGROUND
PMID: 31989877 (View on PubMed)

Other Identifiers

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MRI in neurodegenerative

Identifier Type: -

Identifier Source: org_study_id

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