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Scientists track canavan disease in 67 children to map its progression

NCT ID NCT04126005

Knowledge-focused Sponsor: Aspa Therapeutics 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study followed 67 children with Canavan disease, a rare genetic brain disorder, to learn how the condition naturally changes over time. Researchers reviewed medical records and conducted checkups to track symptoms, milestones, and disease progression. The goal was to better understand the disease, which may help design future treatments and clinical trials.

What this could mean

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

What this could lead to
If this study succeeds, it will give doctors and researchers a clearer picture of how Canavan disease progresses, which could help design future treatments and clinical trials.
What could go wrong
This is an observational study, not a treatment trial, so it does not test any therapy. The results may not apply to all patients, and the small number of participants (67) limits how much we can learn.

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

67 people

The number who actually took part.

Started

Oct 2019

Finished

Aug 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

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

Who is studied

Living and deceased patients with Canavan disease

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: 1. Meet age criteria of a specific cohort. 2. Confirmed clinical and biochemical diagnosis of Canavan disease. 3. Available medical records since birth that permit documentation of disease characteristics and developmental milestones. 4. Parent and/or legal guardian is able to read, understand, and sign the informed consent. Exclusion Criteria: 1\. Patient does not meet the Inclusion Criteria.

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Conditions

The condition(s) this trial relates to.

Canavan disease Leukoencephalopathies Rare Diseases

As listed by the trial registrant

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

Canavan disease

Contacts and locations

Locations

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

  • UCSF Benioff Children's Hospital Oakland

    Oakland, California, 94609, United States

  • University Medical Center Hamburg-Eppendorf

    Hamburg, 20246, Germany

More trials for these conditions

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