Study of New Mutations in Cone Disorders

NCT ID: NCT04658251

Last Updated: 2022-05-17

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

RECRUITING

Total Enrollment

20 participants

Study Classification

OBSERVATIONAL

Study Start Date

2021-03-03

Study Completion Date

2026-03-31

Brief Summary

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High throughput sequencing gives the opportunity to improve the genetic diagnosis for patients suffering from retinal dystrophies and specially from cone disorders. However, a large number of mutations are identified, mostly in introns of the genes, and in silico analysis are not sufficient to assign the pathogenicity of these mutations, without which the diagnosis confirmation cannot be done. For that purpose, a functional analysis of intronic variants of unknown significance detected in patients, with minigene splice assays in parallel with the analysis of the effect of the variant on splicing directly in the cells of the patient, by analyzing the RNA from leucocytes, fibroblasts, lymphoblastoïd cells or precursor of photoreceptor cells, which is the only proof of pathogenicity for variants

Detailed Description

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Conditions

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Retinal Dystrophy, Cone-Rod Cone Dystrophy Cone Rod Dystrophy Macular Degeneration

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Patients with an intronic variant unknown in a gene implicated in cone disorders.

Blood and/or skin biopsy

Intervention Type GENETIC

Blood and/or skin biopsy will be withdrawn, for RNA extraction in order to test the effect of the variant on splicing.

Interventions

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Blood and/or skin biopsy

Blood and/or skin biopsy will be withdrawn, for RNA extraction in order to test the effect of the variant on splicing.

Intervention Type GENETIC

Eligibility Criteria

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Inclusion Criteria

* clinical diagnosis of cone disorder
* identification of a variant of unknown significance
* possibility of samplings
* informed consent

Exclusion Criteria

* no variant of unknown significance identified
* no informed consent
Minimum Eligible Age

3 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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University Hospital, Lille

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Claire-Marie DHAENENS, MD

Role: PRINCIPAL_INVESTIGATOR

University Hospital, Lille

Locations

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CHU lille

Lille, , France

Site Status RECRUITING

Countries

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France

Central Contacts

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Claire-Marie DHAENENS, MD

Role: CONTACT

0320445962 ext. +33

Facility Contacts

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Role: primary

0320445962

Other Identifiers

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2020-A02559-30

Identifier Type: OTHER

Identifier Source: secondary_id

2020_66

Identifier Type: -

Identifier Source: org_study_id

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