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Enzyme therapy shows promise for rare gaucher type 3 in small chinese study

NCT ID NCT04656600

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 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

Phase 4

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

Participants

12 people

The number who actually took part.

Started

Mar 2021

Finished

Oct 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

2 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: * 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

  • Investigational Site Number : 101

    Beijing, 100032, China

  • Investigational Site Number : 102

    Guangzhou, 510080, China

  • Investigational Site Number : 104

    Chengdu, 610041, China

  • Investigational Site Number : 105

    Guangzhou, 510623, China

  • Investigational Site Number : 107

    Beijing, 100020, China