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New study aims to measure how CLN2 disease affects Children's development

NCT ID NCT03862274

Knowledge-focused Sponsor: Jessica Scherr 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 This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 looks at how children with CLN2 Batten disease develop over time, including their thinking, language, and motor skills. Researchers will compare children who receive treatment (cerliponase alfa) with those who do not. The goal is to find better ways to measure the disease's impact and improve care. About 30 children will take part.

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

About 30 people

The number the study aims to enrol. It can still change while the study runs.

Started

Dec 2018

Expected to finish

Dec 2025

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

Patients that have a TPP1 enzyme deficiency and/or confirmed molecular diagnosis of pathogenic variants in the TPP1 gene are eligible to participate if they are untreated or are receiving cerliponase alfa.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

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: * Patients that have a TPP1 enzyme deficiency * Patients have confirmed molecular diagnosis of pathogenic variants in the TPP1 gene * Patients that are enrolled in post-marketing studies will be allowed to enroll into the current study Exclusion Criteria: * Patients without a diagnosis of CLN2 and deficiency of TPP1 * Patients that are currently enrolled as part of a larger multi-center clinical trial

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Conditions

The condition(s) this trial relates to.

neuronal ceroid lipofuscinosis neuronal ceroid lipofuscinosis 2 neuronal ceroid lipofuscinosis 3 Neuronal Ceroid-Lipofuscinoses

As listed by the trial registrant

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

Batten disease CLN2 Neuronal ceroid-lipofuscinoses

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.