Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

Global registry launches to unlock secrets of rare wolfram syndrome

NCT ID NCT02841553

Knowledge-focused Sponsor: Washington University School of Medicine Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study is building a worldwide registry for people with Wolfram syndrome, a rare genetic disorder causing diabetes, vision loss, and hearing problems. Researchers will track how the disease progresses over time and analyze participants' genetic information. The goal is to better understand the condition and lay the groundwork for future treatments.

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 successful, this registry could provide crucial insights into how Wolfram syndrome progresses, potentially guiding future treatments.
What could go wrong
This is an observational registry, not a treatment trial. It will not directly improve health and may face challenges in collecting complete data from participants worldwide.

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 5,000 people

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

Start date

Jul 2011

Expected to finish

Apr 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

Any patient worldwide with a clinical diagnosis of Wolfram syndrome and with access to the Internet can be enrolled in the Registry. Since the disease usually manifests in the first decade of life and tends to have an inevitably progressive course, participation of minors is important for establishing the natural course of the disease.

Ages

0 years 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.

Any patient worldwide with a diagnosis of Wolfram syndrome and with access to the Internet can be enrolled in the Registry. Since the disease usually manifests in the first decade of life and tends to have an inevitably progressive course, participation of minors is important for establishing the natural course of the disease. Inclusion Criteria: Major Criteria * Diabetes mellitus \<16 yrs * Optic atrophy \<16 yrs Minor Criteria * Diabetes insipidus * Diabetes mellitus \>16yrs * Optic atrophy \>16 yrs * Sensorineural deafness * Neurological signs (ataxia, epilepsy, cognitive impairment) * Renal tract abnormalities (structural or functional) * 1 loss of function mutation in WFS1/CISD2 AND/OR family history of Wolfram syndrome Minimum Required * 2 major OR * 1 major plus 2 minor criteria OR * 2 pathological WFS1 or CISD2 mutations are identified Other variable suggestive evidence * Hypogonadism (males) * An absence of type 1 diabetes auto-antibodies * Bilateral cataracts * Psychiatric disorder * Gastrointestinal Exclusion Criteria: Inability of a patient and/or a guardian to obtain help with translation and thus, inability to understand questionnaire. Parent, Sibs, and Spouses: \- Parents, sibs, and spouses that are unaffected will be recruited as controls. Inclusion criterion is having the unaffected status and exclusion criterion is if the person cannot understand the Informed Consent Document.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Ataxia are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

Ataxia cerebellar ataxia Deafness diabetes insipidus diabetes mellitus hearing loss disorder optic atrophy Wolfram syndrome

As listed by the trial registrant

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

Ataxia Deafness Diabetes insipidus Diabetes mellitus Optic nerve atrophy Wolfram syndrome

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

    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

  • Washington University School of Medicine

    RECRUITING

    St Louis, Missouri, 63110, United States

More trials for these conditions

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