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

Launched by UNIVERSITY HOSPITAL, ANGERS · May 30, 2024

Keywords

ClinConnect Summary

This clinical trial is looking at a new type of gene therapy for Huntington's disease, which is a serious condition that affects the brain and leads to severe movement and cognitive problems. The therapy aims to fix a specific genetic issue in the Huntingtin gene that causes the disease. By using a method called trans-splicing, the researchers hope to correct the faulty gene in cells taken from patients' skin. This study is important because, currently, there are no approved treatments that can protect the brain or cure Huntington's disease.

To participate in the trial, individuals must be between 18 and 70 years old and have a specific genetic marker (CAG≥36) related to Huntington's disease. They also need to be able to give consent to join the study. Participants will have their skin cells tested to see how well this new therapy works. It's essential to note that people who have previously participated in other gene therapy studies or who have certain health conditions may not be eligible. This trial is currently recruiting participants, so if you or someone you know fits the criteria, this could be an opportunity to contribute to potentially groundbreaking research in Huntington's disease.

Gender

ALL

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).

Trial Officials