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Can a genetic 'Patch' fix Huntington's disease? lab test aims to find out

NCT ID NCT06444217

Knowledge-focused Sponsor: University Hospital, Angers Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Aug 10, 2026 · Last updated Aug 11, 2026 · Updated 1 time

Summary

This study tests a gene therapy technique in the lab using skin cells from people with Huntington's disease. The goal is to see if the therapy can correct the genetic mistake that causes the disease. If it works in these cells, it could be a step toward a future treatment.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
RNA trans-splicing gene therapy (tested in the lab on patient-derived skin cells)
What this could lead to
If this lab test succeeds, it could pave the way for a gene therapy that corrects the genetic error causing Huntington's disease.
What could go wrong
This is a very early lab study on cells, not people. The approach may not work in the lab or may not translate to a safe or effective treatment for patients.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Not a phased trial

Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.

Participants

20 people

The number who actually took part.

Started

Sep 2024

Finished

Nov 2025

Lead sponsor

A government agency

The lead sponsor is a government body.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 70 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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).

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Conditions

The condition(s) this trial relates to.

Huntington disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Huntington disease

Contacts and locations

Locations

  • ABRIAL

    Angers, Maine et Loire, 49933, France

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