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

Gene therapy for krabbe disease: did it last?

NCT ID NCT06308718

Knowledge-focused Sponsor: Forge Biologics, Inc 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
The trial has finished. Results may not be published yet.
Stopped early This study
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This status has not been confirmed recently, so it may be out of date.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 26, 2026

Summary

This study follows up on children with Krabbe disease who received a one-time gene therapy infusion (FBX-101) in earlier trials. Researchers will monitor safety and measure motor skills over time. Only 2 participants are enrolled, so results are very limited.

What this could mean

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

Active substance
FBX-101 (gene therapy)
What this could lead to
If successful, this follow-up could show whether a single gene therapy infusion provides lasting safety and motor benefits for children with Krabbe disease.
What could go wrong
This is a tiny observational follow-up (only 2 participants) of an already terminated trial. It cannot prove effectiveness and may not apply to other patients.

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

2 people

The number who actually took part.

Started

Aug 2024

Finished

Apr 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

This is an observational long-term follow-up (LTFU) study of participants from prior interventional trials involving the administration of FBX-101.

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: * Participants that have completed a prior clinical trial involving the administration of FBX-101. * Parent(s)/legal guardian(s) of participant willing and able to complete the informed consent process and comply with study procedures and visit schedule. Exclusion Criteria: •Planned or current participation in any other interventional clinical study that may confound the safety or efficacy evaluation of FBX-101 during this study.

Get updates

Get notified about this study

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

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

Krabbe disease lysosomal storage disease

As listed by the trial registrant

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

Krabbe disease

Contacts and locations

Locations

  • University of Michigan Hospitals - Michigan Medicine

    Ann Arbor, Michigan, 48109, United States

More trials for these conditions

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