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RECRUITING
NCT06851377
NA

Expanding NGS Data with Optical Genome Mapping (OGM)

Sponsor: IRCCS Eugenio Medea

View on ClinicalTrials.gov

Summary

Over 50% of pediatric neurological and neurodevelopmental disorders lack a molecular diagnosis after standard DNA sequencing and molecular karyotyping. This is due to technical limitations, incomplete variant interpretation, and inadequate genotype-phenotype correlations. New sequencing technologies are crucial for clinical decision-making, offering complete profiles of variants in a patient's DNA to personalize treatment. Optical Genome Mapping (OGM) can detect nearly all structural variants in one experiment. This project aims to use OGM alongside NGS to improve diagnostic yield in 60 children with severe disorders who tested negative for NGS/CMA.

Official title: Expanding NGS Data with Optical Genome Mapping (OGM): More Comprehensive Variant Detection in Children with Unexplained Rare Genetic Disorders

Key Details

Gender

All

Age Range

2 Years - Any

Study Type

INTERVENTIONAL

Enrollment

60

Start Date

2024-05-23

Completion Date

2026-12

Last Updated

2025-02-28

Healthy Volunteers

No

Interventions

GENETIC

Optical Genome Mapping (OGM) and Whole Genome Sequencing (WGS)

After identifying causal SVs via OGM, WGS will determine rearrangement breakpoints and examine nearby genes within 100 kb that may have altered expression due to positional effects.

OTHER

Trascriptome analysis

Following genomic characterization results, transcriptome analysis will be performed on patient-derived lymphoblastoid B-cell lines or fibroblasts to investigate the molecular implications of candidate SVs found in the OGM analysis and identify potential transcriptome abnormalities, such as splicing variants, in patients with atypical clinical features.

Locations (1)

Cytogenetic Unit of Medical Genetic Laboratory

Bosisio Parini, Lecco, Italy