High-Dose therapy tested for rare genetic disorder
NCT ID NCT04656600
Summary
This study tested the highest approved dose of a replacement enzyme therapy (imiglucerase) in Chinese patients with Gaucher disease type 3, a rare genetic disorder. The goal was to see if this high dose safely improves blood cell counts, reduces enlarged organs, and improves bone health and quality of life over one year of treatment. It involved 12 patients who had not received this specific enzyme therapy before.
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Contacts and locations
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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
Conditions
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