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A prospective observational study of neurocognitive function and multimodal brain magnetic resonance imaging in patients with glycogen storage diseases

NCT ID NCT07908212

Sponsor: Peking Union Medical College Hospital Source: ClinicalTrials.gov ↗

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Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
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Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
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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 Results may not be published yet.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)

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Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
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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

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Status unknown
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First seen Nov 02, 2026 · Last updated Nov 03, 2026 · Updated 1 time

Summary

The goal of this observational study is to learn about cognitive function and brain imaging findings in people with glycogen storage disease (GSD). The main questions it aims to answer are: * Do people with GSD perform differently on cognitive tests from people without GSD? * Do brain volume, brain structure, intracranial blood vessels, and blood flow differ between the two groups, and are these findings related to cognitive test results or measures of metabolic control? Researchers will compare people with GSD with healthy volunteers of similar age and sex. No treatment will be assigned as part of the study. Participants will: * Complete cognitive tests * Have a brain magnetic resonance imaging (MRI) scan using a 3-tesla scanner * Provide relevant medical history and clinical information

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 318 people

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

Started

Mar 2025

Expected to finish

Dec 2029

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

We enroll individuals diagnosed with glycogen storage disease who are able to complete cognitive assessments and brain MRI, and healthy volunteers matched for age and sex.

Ages

3 to 40 years

Sex

Anyone

Healthy volunteers

Accepted

You do not need to have the condition being studied to take part.

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: GSD group: * Meeting the diagnostic criteria for GSD * Having relatively complete clinical data * Able to cooperate with cognitive assessments and neuroimaging examinations Control group: * Age within 5 years of GSD patients, matched for sex, and with similar educational level * No diagnosis of GSD * No chronic metabolic diseases such as hypertension, diabetes mellitus, or hyperlipidemia; no history of heavy smoking or alcohol drinking; and no history of hypoglycemia in childhood Exclusion Criteria: * Concurrent diagnosis of other neurological disorders * Presence of factors that may cause central nervous system (CNS) injury, such as premature birth or head trauma * Contraindications to magnetic resonance imaging * Inability of the patient or their family members to understand the conditions and objectives of this study

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Conditions

The condition(s) this trial relates to.

disorder of glycogen metabolism

As listed by the trial registrant

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

Glycogen storage disease

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

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

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Contacts and locations

Locations

  • Peking Union Medical College Hospital

    RECRUITING

    Beijing, Beijing Municipality, 100730, China

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