Fabrazyme shows promise for fabry disease in chinese study
NCT ID NCT05054387
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
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Phase 4
Runs after approval, following long-term safety and how well the treatment works in everyday use.
- Participants
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22 people
The number who actually took part.
- Started
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Oct 2021
- Finished
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Mar 2023
- Lead sponsor
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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
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8 years and older
- Sex
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Anyone
- Healthy volunteers
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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 Hide 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
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Investigational Site Number :1560001
Shanghai, 200025, China
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Investigational Site Number :1560002
Beijing, 100730, China
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Investigational Site Number :1560003
Beijing, 100034, China
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Investigational Site Number :1560004
Shanghai, 201102, China
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Investigational Site Number :1560005
Wuhan, 016040, China
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Investigational Site Number :1560006
Taiyuan, 030001, China
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