NCT06444217 · RECRUITING
Gene Therapy Development and Validation for Huntington's Disease Fibro TG-HD
This trial is collecting skin biopsies from people with Huntington's disease to grow cell lines in a lab. Researchers want to test a gene therapy technique called trans-splicing, which aims to correct the faulty Huntingtin gene at the RNA level. No drug or therapy is given to participants — just a skin sample. This is a Phase NA study, meaning it is a laboratory validation effort, not a standard treatment trial.
You may qualify if
- 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.
You're excluded if
- 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).
The sponsor's own eligibility wording, lightly reformatted. The study team makes the final eligibility decision — worth discussing with your doctor.
Eligibility criteria as of 2024-12-13