Enzyme therapy shows promise for rare gaucher type 3 in small chinese study
NCT ID NCT04656600
First seen Jun 25, 2026 · Last updated Jun 26, 2026 · Updated 1 time
Summary
This study tested the enzyme replacement drug imiglucerase (Cerezyme) in 12 Chinese patients with Gaucher disease type 3, a rare genetic disorder that affects the brain and body. Participants received the highest approved dose (60 units per kilogram) every two weeks for one year. Researchers measured changes in blood counts, organ sizes, bone pain, and quality of life, while also tracking side effects.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Imiglucerase (Cerezyme), a replacement enzyme given by IV infusion
- What this could lead to
- If successful, this could confirm that imiglucerase at higher doses helps manage blood problems and organ enlargement in Chinese patients with Gaucher type 3.
- What could go wrong
- This is a small, single-arm study with only 12 participants, so results may not apply broadly. It is also a phase 4 trial, meaning the drug is already approved; the main goal is to confirm safety and effectiveness in this specific population.
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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12 people
The number who actually took part.
- Started
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Mar 2021
- Finished
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Oct 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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2 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: * Capable of giving signed informed consent. * Participant is diagnosed with GD type Ⅲ * Participant with neurological manifestations * Participant whose age is \> 2 years old. * Participant whose spleen and/or liver volume is \> ULN at Screening. Exclusion criteria: * Major congenital anomaly * Clinically significant intercurrent organic disease unrelated to Gaucher disease, which means the disease or condition that may have impact on the parameters chosen for primary endpoints (e.g. level of hemoglobin platelets, liver/spleen enlargement and bone pains) * Prior treatment with ERT. * Physical conditions that cannot tolerate regular treatment or follow-up visit. * Pregnant or lactating women * Participant is participating in or has participated in another clinical study using any investigational therapy in 3 months * Participant has been diagnosed with central nervous system disease unrelated to Gaucher disease, or MRI result of the participant indicates space-occupying lesion in central nervous system * The patient has a documented hemoglobinopathies, deficiency of iron, vitamin B-12, or folate that requires treatment not yet initiated or, if initiated, the patient has not been stable under treatment for at least 6 months prior to administration of the first dose of Cerezyme in this study * Participant not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures * Any specific situation during study implementation/course that may rise ethics considerations * Sensitivity to any of the study interventions, or components thereof, or drug or other allergy that, in the opinion of the Investigator, contraindicates participation in the study 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 : 101
Beijing, 100032, China
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Investigational Site Number : 102
Guangzhou, 510080, China
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Investigational Site Number : 104
Chengdu, 610041, China
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Investigational Site Number : 105
Guangzhou, 510623, China
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Investigational Site Number : 107
Beijing, 100020, China