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Enzyme therapy shows promise for rare disease in china

NCT ID NCT05134571

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

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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

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

Oct 2021

Finished

Jul 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

5 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: * Chinese participants have a documented diagnosis of MPS I confirmed by measurable clinical signs and symptoms of MPS I and fibroblast or leukocyte IDUA (iduronidase) activity \<10% of normal. * Participants have to be able to stand independently and walk a minimum of 5 meters in 6 minutes. * A female participant is eligible to participate if she is not pregnant or breastfeeding, and at least one of the following conditions applies: * Is not a woman of childbearing potential (WOCBP). * OR * Is a WOCBP and agrees to use an acceptable contraceptive method during the intervention period and at a minimum until 7 days after the last dose of study intervention. * A WOCBP must have a negative highly sensitive pregnancy test (urine or serum as required by local regulations) within the screening period before the first dose of study intervention. * If a urine test cannot be confirmed as negative (eg, an ambiguous result), a serum pregnancy test is required. In such cases, the participant must be excluded from participation if the serum pregnancy result is positive. * Contraceptive/barrier method is not applicable for male participants. Exclusion Criteria: * Prior tracheostomy or bone marrow transplantation or hematopoietic stem cell transplantation. * Have a plan to undergo bone marrow transplantation or hematopoietic stem cell transplantation within half a year after enrollment. * Received an investigational drug, or device, other than Aldurazyme, within 30 days prior to study enrollment. * Received an investigational gene therapy. The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.

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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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