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15-Year checkup: how did early fabry treatment hold up?

NCT ID NCT04002531

Knowledge-focused Sponsor: Baylor Research Institute 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This study checked in on 5 adults with Fabry disease who had been taking enzyme replacement therapy (ERT) since childhood for about 15 years. Researchers measured kidney function and heart health to see how well the long-term treatment worked. The goal was to gather follow-up data, not to test a new treatment.

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.

Phase

Not a phased trial

Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.

Participants

5 people

The number who actually took part.

Started

Nov 2018

Finished

Dec 2019

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.

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: 1. Patients who participated in TKT029 and who are willing and able to come to Dallas for 1 visit for standard of care testing. 2. Sign the protocol informed consent form 3. Have been on continuous commercial ERT since TKT029 has ended Exclusion Criteria: 1. Patients who are unable to understand the nature, scope, and possible consequences of the study. 2. Patient does not give his written informed consent to participate in this study 3. Patient is unable to comply with the protocol, e.g., uncooperative with protocol schedule, refusal to agree to all of the study procedures. 4. Patient has been off ERT for an extended period of time as assessed by the investigator.

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Conditions

The condition(s) this trial relates to.

cardiovascular disorder Fabry disease kidney failure

As listed by the trial registrant

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

Cardiac event Quality of life Renal insufficiency

Contacts and locations

Locations

  • Baylor University Medical Center

    Dallas, Texas, 75246, United States

More trials for these conditions

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