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Gene Therapy's lasting promise: can one infusion safely control fabry disease for years?

NCT ID NCT05039866

Disease control Sponsor: Sangamo 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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Aug 20, 2026 · Last updated Aug 21, 2026 · Updated 1 time

Summary

This study follows people with Fabry disease who previously received an experimental gene therapy called ST-920 in an earlier trial. The goal is to monitor their health for up to five years after the infusion to see if the treatment remains safe over the long term. Participants do not receive any new treatment in this study—they are simply observed and evaluated for any side effects or medical issues that may arise.

What this could mean

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

Active substance
ST-920, an experimental gene therapy for Fabry disease
What this could lead to
If successful, this could confirm that a single gene therapy infusion provides lasting, safe control of Fabry disease, potentially reducing the need for lifelong enzyme replacement.
What could go wrong
This is a long-term follow-up study, not a new treatment test. It may reveal delayed side effects or show that the therapy's benefits fade over time. Results depend on a small group of 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

32 people

The number who actually took part.

Started

Aug 2021

Expected to finish

Mar 2039

An estimate. End dates often move.

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

All subjects who have received ST-920 in separate parent trial and who have consented to participate in this Long Term Follow-up study.

Ages

18 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.

Inclusion Criteria: * Subjects who received ST-920 therapy in a separate parent trial * Subjects who have consented to participate in this LTFU study. Exclusion Criteria: -This study has no exclusion criteria

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Conditions

The condition(s) this trial relates to.

Fabry disease Fabry Disease, Cardiac Variant

As listed by the trial registrant

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

Fabry disease Fabry disease, cardiac variant

Contacts and locations

Locations

  • Addenbrooke's Hospital

    Cambridge, CB2 0QQ, United Kingdom

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Emory University School of Medicine

    Atlanta, Georgia, 30322, United States

  • Lysosomal & Rare Disorders Research & Treatment Center (LDRTC)

    Fairfax, Virginia, 22030, United States

  • M.A.G.I.C. Clinic Ltd.

    Calgary, Alberta, T2E 7Z4, Canada

  • Mt. Sinai Hospital

    New York, New York, 10029, United States

  • Royal Free Hospital

    London, United Kingdom

  • The Royal Melbourne Hospital

    Parkville, Victoria, 3050, Australia

  • University Hospital of Würzburg

    Würzburg, Germany

  • University of California, Irvine

    Irvine, California, 92697, United States

  • University of Iowa Hospital and Clinics

    Iowa City, Iowa, 52242, United States

  • University of Minnesota Medical Center

    Minneapolis, Minnesota, 55455, United States

  • University of South Florida

    Tampa, Florida, 33620, United States

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