Enzyme therapy tested for rare genetic disease in china
NCT ID NCT05134571
Summary
This study tested the safety and effectiveness of Aldurazyme, an enzyme replacement therapy, in 12 Chinese patients with Mucopolysaccharidosis I (MPS I), a rare genetic disorder. Participants received weekly infusions for 26 weeks. The main goals were to check for side effects and see if the treatment reduced certain disease markers in urine and liver size.
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Contacts and locations
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Locations
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Investigational Site Number :1560001
Wuhan, 430030, 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
Guangzhou, 510623, China
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Investigational Site Number :1560006
Hangzhou, 310003, China
Conditions
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