NCT06444217 Gene Therapy Development and Validation for Huntington's Disease Fibro TG-HD
| NCT ID | NCT06444217 |
| Status | Recruiting |
| Phase | — |
| Sponsor | University Hospital, Angers |
| Condition | Huntington Disease |
| Study Type | INTERVENTIONAL |
| Enrollment | 20 participants |
| Start Date | 2024-09-23 |
| Primary Completion | 2026-07-23 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
This trial targets 20 participants in total. It began in 2024-09-23 with a primary completion date of 2026-07-23.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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.
Eligibility Criteria
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).
Contact & Investigator
VERNY Christophe, MD, PhD
PRINCIPAL INVESTIGATOR
University Hospital, Angers
Frequently Asked Questions
Who can join the NCT06444217 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, up to 70 Years, studying Huntington Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT06444217 currently recruiting?
Yes, NCT06444217 is actively recruiting participants. Contact the research team at Charlotte.abrial@chu-angers.fr for enrollment information.
Where is the NCT06444217 trial being conducted?
This trial is being conducted at Angers, France.
Who is sponsoring the NCT06444217 clinical trial?
NCT06444217 is sponsored by University Hospital, Angers. The principal investigator is VERNY Christophe, MD, PhD at University Hospital, Angers. The trial plans to enroll 20 participants.