Gene therapy for Huntington's disease using fibroblasts
Gene Therapy Development and Validation for Huntington's Disease Fibro TG-HD
This study is testing a new gene therapy that uses skin cells from people with Huntington's disease to see if it can fix the genetic problems causing the illness.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 20 (estimated) |
| Ages | 18 Years to 70 Years |
| Sex | All |
| Sponsor | University Hospital, Angers Government |
| Locations | 1 site (Angers, Maine et Loire) |
| Trial ID | NCT06444217 on ClinicalTrials.gov |
What this trial studies
This study focuses on developing and validating a novel gene therapy approach for Huntington's disease, a severe neurodegenerative disorder caused by mutations in the Huntingtin gene. The methodology involves trans-splicing gene therapy, which aims to correct mutated RNA sequences by replacing them with healthy ones, potentially offering a more targeted treatment option. The therapy will be tested in vitro using primary fibroblast cell lines derived from skin biopsies of affected individuals. The goal is to evaluate the therapeutic effects of this innovative approach on Huntington's disease pathology.
Who should consider this trial
Good fit: Ideal candidates for this study are adults aged 18 to 70 with a CAG repeat length of 36 or more in the Huntingtin gene.
Not a fit: Patients who have previously participated in gene therapy trials targeting the Huntingtin gene or those with conditions that may complicate the diagnosis of Huntington's disease may not benefit from this study.
Why it matters
Potential benefit: If successful, this therapy could provide a groundbreaking treatment option for patients with Huntington's disease, potentially slowing disease progression or offering a cure.
How similar studies have performed: While gene therapy approaches for Huntington's disease are emerging, this specific trans-splicing method is novel and has not been extensively tested in prior studies.
Eligibility criteria
Show full inclusion / exclusion 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).
Where this trial is running
Angers, Maine et Loire
- Abrial — Angers, Maine et Loire, France (Recruiting)
Study contacts
- Principal investigator: VERNY Christophe, MD, PhD — University Hospital, Angers
- Study coordinator: Charlotte ABRIAL, PhD
- Email: Charlotte.abrial@chu-angers.fr
- Phone: 02.41.35.56.15
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.