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

NCT06444217 · Status: RECRUITING · Phase: NA · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2024-12-13

No results posted yet for this study

Summary

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.

Conditions

  • Huntington Disease

Interventions

PROCEDURE

skin biopsy

skin biopsy

Sponsors & Collaborators

  • University Hospital, Angers

    lead OTHER_GOV

Principal Investigators

  • VERNY Christophe, MD, PhD · University Hospital, Angers

Study Design

Allocation
NA
Purpose
BASIC_SCIENCE
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
70 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-09-23
Primary Completion
2026-07-23
Completion
2028-07-23

Countries

  • France

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06444217 on ClinicalTrials.gov