Mucopolysaccharidosis IPhase 4NCT05134571
China Post-marketing Surveillance (PMS) Study of Aldurazyme®
A Phase 4 study testing Laronidase in people with Mucopolysaccharidosis I, run by Genzyme, a Sanofi Company at 5 sites in China.
Trial summary
This is a single treatment arm study that is open-label to be conducted in Chinese participants with MPS I. Trial Objectives are to evaluate the safety and tolerability of Aldurazyme in Chinese MPS I participants, and to evaluate the efficacy of Aldurazyme on the percent change of urinary glycosaminoglycans (uGAGs)… Read more about this study
- Who is it for?
- Ages 5 years and older
- Conditions the study is for
- Mucopolysaccharidosis I
- What is being tested?
- Laronidase
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 5 sitesChina
- Study dates
- 28 Oct 2021to 26 Jul 2023
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Show all 9Show fewer
- 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.
Who cannot join
- 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.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
China5 sites
Investigational Site Number :1560003
Beijing, 100034
Investigational Site Number :1560002
Beijing, 100730
Investigational Site Number :1560004
Guangzhou, 510623
Investigational Site Number :1560006
Hangzhou, 310003
Investigational Site Number :1560001
Wuhan, 430030
About this study
Official title: A Phase 4, Single-arm, Open-label Safety and Efficacy Study of Aldurazyme® (Laronidase) as Enzyme Replacement Therapy in Participants With Mucopolysaccharidosis I (MPS I) in China
This is a single treatment arm study that is open-label to be conducted in Chinese participants with MPS I.
Trial Objectives are to evaluate the safety and tolerability of Aldurazyme in Chinese MPS I participants, and to evaluate the efficacy of Aldurazyme on the percent change of urinary glycosaminoglycans (uGAGs) from baseline to Week 26. The study will also evaluate the effect on uGAG level and liver volume (hepatomegaly) after 26 weeks, with Aldurazyme treatment in Chinese MPS I participants.
Treatment duration will include: 2 weeks of screening, 26 weeks of treatment and 1 week of follow-up period. During the treatment period, weekly visits are designed to accommodate weekly administration of Aldurazyme (laronidase).
Read the detailed description
Study duration for each participant will be a total of 29 weeks which will include 2 weeks of screening, 26 weeks of treatment period and 1 week of follow-up period.
- Conditions
- Mucopolysaccharidosis I
- Being tested
- Laronidase
- Keywords
- clinical trial, enzyme replacement therapy, mucopolysaccharidosis I, laronidase
- Medical subject headings
- Mucopolysaccharidosis I
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LaronidaseDrug
Solution for injection; Intravenous
| Study group | Role | What they receive |
|---|---|---|
| Aldurazyme (laronidase) | Experimental | Aldurazyme (laronidase) treatment at approved dose and regimen, administered every week as an IV infusion Receives: Drug: Laronidase |
What is being measured
The primary outcome is the main question the study is designed to answer.
Participants with adverse events (AEs)
Incidence of AEs, serious adverse events (SAEs) and adverse events of special interest (AESIs) including infusion associated reactions (IARs) during the treatment emergent (TE) period
Measured: Baseline to Week 27
The incidence of Potentially clinically significant abnormality (PCSA) analyses for clinical laboratory, vital signs, and ECG parameters during the TE period
Measured: Baseline to Week 27
The percent change of uGAGs
Measured: Baseline to Week 26
Secondary outcomes (3)
The percent change of uGAGs
Measured: Baseline to Week 2, Week 4, Week 8, Week 12 and Week 20
The absolute change of uGAGs
Measured: Baseline to Week 2, Week 4, Week 8, Week 12, Week 20 and Week 26
The percent change of liver volume (Abdominal B type ultrasound examination)
Measured: Baseline to Week 26
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Nov 2021
- Study start28 Oct 2021
- Primary completion26 Jul 2023
- Study completion26 Jul 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Mucopolysaccharidosis IStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genzyme, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences & Operations, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05134571
- Sponsor's protocol ID
- LPS16578
- Other IDs
- U1111-1260-3947
- Status last verified
- Sep 2025
- First submitted
- 21 Oct 2021
- Last updated
- 17 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Publications
Liang Y, Yang YL, Zou CC, Liu L, Jiao Y, Wang Y, Liu X, Luo XP. Safety and efficacy of laronidase in Chinese patients with mucopolysaccharidosis type I: a phase IV, single-arm, open-label, multicenter study. Orphanet J Rare Dis. 2025 Oct 29;20(1):547. doi: 10.1186/s13023-025-04056-w.
PubMed 41163043
Study at a glance
From the registry record, last updated by the sponsor on 17 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 4, Single-arm, Open-label Safety and Efficacy Study of Aldurazyme® (Laronidase) as Enzyme Replacement Therapy in Participants With Mucopolysaccharidosis I (MPS I) in China
- ClinicalTrials.gov ID
- NCT05134571
- Acronym / sponsor ID
- LPS16578
- Conditions
- Mucopolysaccharidosis I
- Keywords
- clinical trial, enzyme replacement therapy, mucopolysaccharidosis I, laronidase
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Study start
- 28 Oct 2021 (actual)
- Primary completion
- 26 Jul 2023 (actual)
- Study completion
- 26 Jul 2023 (actual)
- First posted
- 26 Nov 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a single treatment arm study that is open-label to be conducted in Chinese participants with MPS I.
Trial Objectives are to evaluate the safety and tolerability of Aldurazyme in Chinese MPS I participants, and to evaluate the efficacy of Aldurazyme on the percent change of urinary glycosaminoglycans (uGAGs) from baseline to Week 26. The study will also evaluate the effect on uGAG level and liver volume (hepatomegaly) after 26 weeks, with Aldurazyme treatment in Chinese MPS I participants.
Treatment duration will include: 2 weeks of screening, 26 weeks of treatment and 1 week of follow-up period. During the treatment period, weekly visits are designed to accommodate weekly administration of Aldurazyme (laronidase).
Detailed description
Study duration for each participant will be a total of 29 weeks which will include 2 weeks of screening, 26 weeks of treatment period and 1 week of follow-up period.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Aldurazyme (laronidase) | Experimental | Drug: LaronidaseAldurazyme (laronidase) treatment at approved dose and regimen, administered every week as an IV infusion |
Who may qualify
- Ages
- 5 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- 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:
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LaronidaseSolution for injection; Intravenous | Drug | Aldurazyme (laronidase) |
Primary outcome
Participants with adverse events (AEs)
Time frame: Baseline to Week 27
Incidence of AEs, serious adverse events (SAEs) and adverse events of special interest (AESIs) including infusion associated reactions (IARs) during the treatment emergent (TE) period
The incidence of Potentially clinically significant abnormality (PCSA) analyses for clinical laboratory, vital signs, and ECG parameters during the TE period
Time frame: Baseline to Week 27
The percent change of uGAGs
Time frame: Baseline to Week 26
3 secondary outcomes
The percent change of uGAGs
Time frame: Baseline to Week 2, Week 4, Week 8, Week 12 and Week 20
The absolute change of uGAGs
Time frame: Baseline to Week 2, Week 4, Week 8, Week 12, Week 20 and Week 26
The percent change of liver volume (Abdominal B type ultrasound examination)
Time frame: Baseline to Week 26
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Sep 2025.
- Derived from this study: PubMed 41163043Liang Y, Yang YL, Zou CC, Liu L, Jiao Y, Wang Y, Liu X, Luo XP. Safety and efficacy of laronidase in Chinese patients with mucopolysaccharidosis type I: a phase IV, single-arm, open-label, multicenter study. Orphanet J Rare Dis. 2025 Oct 29;20(1):547. doi: 10.1186/s13023-025-04056-w.
- LPS16578 Plain Language Results SummaryLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Sciences & Operations study director
Sanofi
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Laronidase in people with Mucopolysaccharidosis I, run by Genzyme, a Sanofi Company at 5 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 17 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Participants with adverse events (AEs) (time frame: Baseline to Week 27). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05134571, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Sep 2025.