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.
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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