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Fabrazyme shows promise for fabry disease in chinese study

NCT ID NCT05054387

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 24, 2026 · Last updated Jun 26, 2026 · Updated 1 time

Summary

This study tested the safety and effectiveness of Fabrazyme (agalsidase beta) in 22 Chinese patients with Fabry disease, a rare genetic disorder. Participants received enzyme replacement therapy through IV infusions over 54 weeks. The study monitored side effects and changes in disease markers like lyso-GL3 and GL3 levels.

What this could mean

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

Active substance
Fabrazyme (agalsidase beta)
What this could lead to
If successful, this could confirm that Fabrazyme is safe and effective for Chinese patients with Fabry disease, providing a treatment option to manage the condition.
What could go wrong
This is a small, single-arm study with only 22 participants, so results may not apply to all patients. As an enzyme replacement therapy, it requires ongoing infusions and does not cure the disease.

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

Phase 4

Runs after approval, following long-term safety and how well the treatment works in everyday use.

Participants

22 people

The number who actually took part.

Started

Oct 2021

Finished

Mar 2023

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.

Ages

8 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: * Participant must be 8 years of age or older, at the time of signing the informed consent * Participants naive to agalsidase beta and agalsidase alpha * Chinese participants diagnosed with Fabry disease and with documented plasma or leukocyte αGAL activity deficient below laboratory's reference range, and/or documented diagnosis by genotyping * Participants must have one or more symptoms and signs consistent with manifestations of Fabry disease (not limited to neuropathic pain, chronic kidney disease, hypertrophic cardiomyopathy, cardiac rhythm disturbances, cerebrovascular involvement, cornea verticillata, angiokeratoma, gastrointestinal symptoms, hypo- or anhydrosis) * A female participant is eligible to participate if she is not pregnant or breastfeeding and use an acceptable contraceptive method * Participants and/or participant's legal representative capable of giving signed informed consent. Exclusion Criteria: * The participant has undergone kidney transplantation. * The participant has a clinically significant organic disease (with the exception of symptoms relating to Fabry disease) in the opinion of the Investigator, would preclude participation in the trial. * Received an investigational drug, or device, other than Fabrazyme, within 30 days of anticipated IMPs administration or 5 half-lives of the previous investigational drug, whichever is longer. * The patient has current evidence of kidney failure or renal insufficiency, as defined by eGFR \<30 mL/min/1.73 m2. * Individuals who have life threatening hypersensitivity (anaphylactic reaction) to the active substance or any of the excipients included. The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

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

Contacts and locations

Locations

  • Investigational Site Number :1560001

    Shanghai, 200025, China

  • Investigational Site Number :1560002

    Beijing, 100730, China

  • Investigational Site Number :1560003

    Beijing, 100034, China

  • Investigational Site Number :1560004

    Shanghai, 201102, China

  • Investigational Site Number :1560005

    Wuhan, 016040, China

  • Investigational Site Number :1560006

    Taiyuan, 030001, China

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