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Clinical Trial Details — Status: Not yet recruiting

Administrative data

NCT number NCT06444217
Other study ID # 2024-A00877-40
Secondary ID
Status Not yet recruiting
Phase N/A
First received
Last updated
Start date July 1, 2024
Est. completion date July 1, 2028

Study information

Verified date May 2024
Source University Hospital, Angers
Contact Charlotte ABRIAL, PhD
Phone 02.41.35.56.15
Email Charlotte.abrial@chu-angers.fr
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


Recruitment information / eligibility

Status Not yet recruiting
Enrollment 20
Est. completion date July 1, 2028
Est. primary completion date July 1, 2026
Accepts healthy volunteers No
Gender All
Age group 18 Years to 70 Years
Eligibility 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).

Study Design


Related Conditions & MeSH terms


Intervention

Procedure:
skin biopsy
skin biopsy

Locations

Country Name City State
France ABRIAL Angers Maine Et Loire

Sponsors (1)

Lead Sponsor Collaborator
University Hospital, Angers

Country where clinical trial is conducted

France, 

Outcome

Type Measure Description Time frame Safety issue
Primary In vitro validation of a RNA trans-splicing gene therapy for the correction of supernumerary CAG repeats into fibroblasts derived from skin biopsies of patients with Huntington's disease. Correction of mutated endogenous transcripts. At the inclusion
Secondary Quantify the expression of Huntingtin protein (HTT) and its (in)usual protein partners. At the inclusion At the inclusion
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