NCT04621422

Brief Summary

Since 2012, NGS sequencing of long fragments or long reads has developed in various fields of research and today presents itself as a very promising alternative solution in the analysis of repeat amplifications. The Oxford Nanopore NGS automaton offers the prospect of bringing together 1st and 2nd line analyzes of all loci potentially indicated in neurogenetics at the same time. The project aims to compare the use of this new technology with methods currently used in reference laboratories. The main objective is to evaluate the ability of next-generation high-throughput Oxford Nanopore-type sequencing (NEURONGS3) to diagnose 9 neurogenetic diseases compared to reference protocols via PCR (+/- Southern blot). The secondary objective is to evaluate the repeatability of the NGS (intra-sample reproducibility) analysis in the diagnosis of 8 neurogenetic diseases.

Trial Health

35
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
60

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started Dec 2020

Typical duration for all trials

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

October 12, 2020

Completed
28 days until next milestone

First Posted

Study publicly available on registry

November 9, 2020

Completed
22 days until next milestone

Study Start

First participant enrolled

December 1, 2020

Completed
1.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2022

Completed
7 months until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2023

Completed
Last Updated

November 9, 2020

Status Verified

October 1, 2020

Enrollment Period

1.5 years

First QC Date

October 12, 2020

Last Update Submit

November 3, 2020

Conditions

Outcome Measures

Primary Outcomes (1)

  • Proportion of patients tested positive by Next Generation Sequencing among all the patients tested positive by the current algorithm based on PCRs

    For each of the 9 considered diseases, the proportion will be established as well as its 95% confidence interval.

    through study completion, an average of 2 years

Secondary Outcomes (1)

  • Difference between the number of repetition amplifications found by the Next Generation Sequencing method and the number of repetition amplifications found by the PCR diagnosis method

    through study completion, an average of 2 years

Interventions

Eligibility Criteria

Age0 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Subject carrying an amplification of nucleotide repeats in one of the following 9 genes FMR1, DMPK, ZNF9, SCA2, JPH3, HD, FXN, C9ORF72, RFC1

You may qualify if:

  • minors, adults and protected adults.
  • Subject carrying an amplification of nucleotide repeats in one of the following 9 genes FMR1, DMPK, ZNF9, SCA2, JPH3, HD, FXN, C9ORF72, RFC1
  • DNA available in sufficient quantity (5 to 10 µg)

You may not qualify if:

  • DNA degraded or of medium size \<30kb,
  • Patient objection to research - This patient objection must be reached to the center's investigator within 1 maximum period of 1 month after sending the information note.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Biospecimen

Retention: SAMPLES WITH DNA

5 to 10 µg of DNA samples per patient.

MeSH Terms

Interventions

Myotonin-Protein KinaseCNBP protein, humanLipoproteins, HDL2Frataxin

Intervention Hierarchy (Ancestors)

Protein Serine-Threonine KinasesProtein KinasesPhosphotransferases (Alcohol Group Acceptor)PhosphotransferasesTransferasesEnzymesEnzymes and CoenzymesIntracellular Signaling Peptides and ProteinsProteinsAmino Acids, Peptides, and ProteinsLipoproteins, HDLLipoproteinsLipidsIron-Sulfur ProteinsNonheme Iron ProteinsIron-Binding ProteinsCarrier ProteinsMetalloproteinsMitochondrial Proteins

Study Officials

  • Cyril Goizet

    University Hospital, Bordeaux

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Study Design

Study Type
observational
Observational Model
CASE ONLY
Time Perspective
RETROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 12, 2020

First Posted

November 9, 2020

Study Start

December 1, 2020

Primary Completion

June 1, 2022

Study Completion

January 1, 2023

Last Updated

November 9, 2020

Record last verified: 2020-10

Data Sharing

IPD Sharing
Will not share