Enzyme therapy shows promise for rare disease in china

NCT ID NCT05134571

Completed Disease control Sponsor: Genzyme, a Sanofi Company Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 26, 2026

Summary

This study tested a drug called Aldurazyme (laronidase) in 12 Chinese people with mucopolysaccharidosis I (MPS I), a rare genetic disorder. Participants received weekly infusions for 26 weeks to see if the treatment is safe and reduces waste products in urine. The goal is to manage the disease, not cure it.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: Aldurazyme (laronidase)
What this could lead to: If successful, this could confirm that Aldurazyme is safe and effective for Chinese patients with MPS I, providing a treatment option to manage the disease.
What could go wrong: This is a small, single-arm study with only 12 participants, so results may not apply broadly. The treatment does not cure MPS I and requires weekly infusions, which can cause infusion reactions.

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Conditions

The condition(s) this trial relates to.

Mucopolysaccharidosis I mucopolysaccharidosis type 1

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

MUCOPOLYSACCHARIDOSIS I

Contacts and locations

Locations

Investigational Site Number :1560001

Wuhan, 430030, China
Investigational Site Number :1560002

Beijing, 100730, China
Investigational Site Number :1560003

Beijing, 100034, China
Investigational Site Number :1560004

Guangzhou, 510623, China
Investigational Site Number :1560006

Hangzhou, 310003, China

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