NCT06444217Completed
Gene Therapy Development and Validation for Huntington's Disease Fibro TG-HD
Source: ClinicalTrials.gov · Last updated 10 Aug 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Completed
- Study type
- Interventional
- Enrollment
- 20 (actual)
- First submitted
- 30 May 2024
- Start date
- 23 Sept 2024
- Primary completion date
- 17 Nov 2025
- Lead sponsor
- University Hospital, Angers
- Diseases
- Huntington's disease
- NCT number
- NCT06444217
What you commit to
- Study type: Interventional
- Participants enrolled: 20
- From 23 Sept 2024 to the primary completion date, 17 Nov 2025
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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.
Other
- 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).
Who the study is looking for
- 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.
Age in the registry: 18 years to 70 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| ABRIAL Coordinator of this site? Confirm its status | Angers | France | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.