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Gene therapy for rare brain disease tracked over years

NCT ID NCT04273243

Disease control Sponsor: Emily de los Reyes 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 This study
Running, but no longer taking on new participants.
Completed
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study follows 10 people with CLN6 Batten disease who received a single dose of gene therapy (AT-GTX-501) in an earlier trial. Researchers are checking long-term safety and how the disease progresses over time. No new treatment is given in this follow-up.

What this could mean

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

Active substance
AT-GTX-501 (gene therapy)
What this could lead to
If successful, this follow-up could show that a single dose of gene therapy safely slows or stabilizes CLN6 Batten disease over years, pointing toward a long-term treatment option.
What could go wrong
This is a very small, early-stage follow-up study with only 10 participants, so results may not apply to everyone. The gene therapy was given years ago, and long-term safety or effectiveness is still uncertain.

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.

Participants

10 people

The number who actually took part.

Started

Jan 2020

Expected to finish

Dec 2027

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

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

Who is studied

The subject population in this Long-Term Follow Up study consists of subjects with CLN6 Batten disease who previously received AT-GTX-501 in the preceding study (AT-GTX-501-01).

Ages

12 months and older

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: * Subject received AT-GTX-501 (scAAV9.CB.CLN6) in the study "Phase I/IIa Gene Transfer Clinical Trial for Variant Late Infantile Neuronal Ceroid Lipofuscinosis, Delivering the CLN6 Gene by Self-Complementary AAV9." * Subject completed or prematurely discontinued from the study "Phase I/IIa Gene Transfer Clinical Trial for Variant Late Infantile Neuronal Ceroid Lipofuscinosis, Delivering the CLN6 Gene by Self-Complementary AAV9." * Subject has a legally authorized representative who has provided written informed consent and authorization for use and disclosure of personal health information or research-related health information. Exclusion Criteria: * None

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Conditions

The condition(s) this trial relates to.

ceroid lipofuscinosis, neuronal, 6A juvenile neuronal ceroid lipofuscinosis neuronal ceroid lipofuscinosis 3 neuronal ceroid lipofuscinosis, CLN6-related, domestic cat Neuronal Ceroid-Lipofuscinoses

As listed by the trial registrant

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

Batten disease CLN6

Contacts and locations

Locations

  • Nationwide Children's Hosptial

    Columbus, Ohio, 43205, United States

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