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Gene Therapy Development and Validation for Huntington's Disease Fibro TG-HD

NCT ID: NCT06444217

Last Updated: 2024-12-13

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

Clinical Phase

NA

Total Enrollment

20 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-09-23

Study Completion Date

2028-07-23

Brief Summary

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Huntington's disease is a rare and fatal monogenic neurodegenerative disorder whose molecular origin is an expansion of CAG triplets within the first exon of the Huntingtin gene. Although a growing number of emerging therapies are in clinical trials, there are no proven neuroprotective or curative treatments approved by the health authorities, as they have not yet demonstrated any real therapeutic benefit or absence of toxicity. Trans-splicing gene therapy is defined as the correction of a mutated endogenous pre-messenger RNA by a therapeutic exogenous pre-messenger RNA. Trans-splicing is a suitable alternative approach, since it is capable of allelic selectivity and replacement of mutated sequences by the wild-type one, criteria that no therapy tested to date meets. This project involves the therapeutic validation of trans-splicing of Huntingtin gene transcripts, and will evaluate its therapeutic effects in vitro, into primary fibroblast cell lines derived from skin biopsies of Huntington's disease patients.

Detailed Description

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Conditions

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Huntington Disease

Keywords

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Huntington gene therapy trans-splicing fibroblasts

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

BASIC_SCIENCE

Blinding Strategy

NONE

Study Groups

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Huntington's patient

skin biopsy

Group Type EXPERIMENTAL

skin biopsy

Intervention Type PROCEDURE

skin biopsy

Interventions

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skin biopsy

skin biopsy

Intervention Type PROCEDURE

Eligibility Criteria

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Inclusion Criteria

* 18 ≤ age ≤ 70 years.
* Signed written, free and informed consent to participate in the study.
* Patients with a CAG≥36 allele (with reduced or full penetrance). penetrance)
* People affiliated to or benefiting from a social security scheme.

Exclusion Criteria

* Individuals who have participated in a gene therapy trial using AAV, ASO, mi/si/shRNA administration, likely to disrupt expression, splicing of pre-mRNAs, mRNA splicing, mRNA expression/regulation/translation, energy or protein metabolism directly or indirectly linked to the Huntingtin gene (HTT), its transcripts and proteins.
* Clinical or paraclinical elements that may suggest a differential diagnosis.
* People unable to express their consent.
* Pregnant, breast-feeding or parturient women
* People deprived of liberty by administrative or judicial decision
* People under legal protection (curatorship, guardianship).
Minimum Eligible Age

18 Years

Maximum Eligible Age

70 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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University Hospital, Angers

OTHER_GOV

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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VERNY Christophe, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

University Hospital, Angers

Locations

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ABRIAL

Angers, Maine et Loire, France

Site Status RECRUITING

Countries

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France

Central Contacts

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Charlotte ABRIAL, PhD

Role: CONTACT

Phone: 02.41.35.56.15

Email: [email protected]

Anne-Catherine AUBE-NATHIER, PhD

Role: CONTACT

Phone: 02 41 34 54 96

Email: [email protected]

Facility Contacts

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ABRIAL Charlotte, PhD

Role: primary

Anne-Catherine AUBE-NATHIER, PhD

Role: backup

Other Identifiers

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2024-A00877-40

Identifier Type: -

Identifier Source: org_study_id