Mucopolysaccharidosis Type 1Phase 3NCT06406153
Efficacy and Safety of YW17 (Laronidase-CinnaGen) Compared to Aldurazyme® in MPS I Patients
A Phase 3 study testing Laronidase, Antihistamine and Antipyretic in people with Mucopolysaccharidosis Type 1, run by Cinnagen at 4 sites in Iran.
Trial summary
The purpose of this phase III study is to assess the efficacy and safety of YW17 produced by CinnaGen Company compared to Aldurazyme® in mucopolysaccharidosis type I (MPS I) patients. All patients receive Aldurazyme® for 12 weeks, followed by YW17 for another 12 weeks. Read more about this study
- Who is it for?
- Ages 5 to 18 years
- Conditions the study is for
- Mucopolysaccharidosis Type 1
- What is being tested?
- Laronidase, Antihistamine and Antipyretic
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 4 sitesIran
- Study dates
- 10 Sep 2022to 18 Nov 2023
- Phase
- Phase 3
- 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 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age 5-18
- Diagnosed with MPS I
- Signing informed consent form
Who cannot join
- Prior bone marrow transplantation or being a candidate for receiving haematopoietic stem cell transplantation (HSCT)
- Prior tracheotomy
- Being naïve to laronidase
- Acute hydrocephalus
- Abnormal renal function determined by measuring serum creatinine and blood urea nitrogen (BUN) levels
- Any severe organic disease that is not associated with MPS I
Show all 10Show fewer
- Known hypersensitivity to laronidase or components of the laronidase solution
- Presence of any medical condition or other circumstances that could significantly interfere with study compliance
- Pregnancy and lactation
- Administration of any investigational drug within 30 days before study enrollment
Trial locations and contacts
4 sites in 1 country.
Iran4 sites
Clinical Research Development Unit of Akbar Hospital, Faculty of Medicine
Mashhad
Growth and Development Research Center, Childrens Medical Center
Tehran
Loghman Hospital
Tehran
Mofid Childrens Hospital
Tehran
About this study
Official title: A Phase III, Single Arm, Cross-over, Multicenter Clinical Trial to Compare Efficacy and Safety of YW17(Laronidase; CinnaGen) Versus Laronidase (Aldurazyme®; Genzyme, BioMarin) in Patients With Mucopolysaccharidosis Type I (MPS I)"
The purpose of this phase III study is to assess the efficacy and safety of YW17 produced by CinnaGen Company compared to Aldurazyme® in mucopolysaccharidosis type I (MPS I) patients.
All patients receive Aldurazyme® for 12 weeks, followed by YW17 for another 12 weeks.
The primary outcome is the assessment of the maintenance of the mean uGAG levels at the end of each medication administration. The secondary outcomes are the assessment of 6-minute walking test (6MWT), predicted forced vital capacity (FVC), enzyme activity assay, and adverse events (AEs).
Read the detailed description
This is a phase III, single-sequence, cross-over study to assess the efficacy and safety of YW17 produced by CinnaGen Company in comparison with Aldurazyme® in MPS I patients.
All patients receive 0.58 mg/kg of Aldurazyme® for 12 weeks and then receive 0.58 mg/kg of YW17 for another 12 weeks.
Premedication with antipyretics and/or antihistamines is administered for all patients one hour before the infusion.
The primary outcome is to compare the mean uGAG levels at weeks 8, 10, and 12 (related to Aldurazyme®) with the mean uGAG levels at weeks 20, 22, and 24 (related to YW17).
The secondary outcomes, including 6MWT and FVC are assessed at the beginning and the end of each medication administration. Enzyme activity is assessed at the end of each medication administration. Safety assessments are performed during the study.
- Conditions
- Mucopolysaccharidosis Type 1
- Being tested
- Laronidase, Antihistamine, Antipyretic
- Keywords
- Urinary Glycosaminoglycans, Laronidase, Mucopolysaccharidosis type I, Aldurazyme®
- Medical subject headings
- Mucopolysaccharidosis I
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LaronidaseBiological
Laronidase as Aldurazyme® (the first 12 weeks) or YW17 (the second 12 weeks) is administered.
AntihistamineDrug
An antihistamine is administered one hour before the infusion.
AntipyreticDrug
An antipyretic is administered one hour before the infusion.
| Study group | Role | What they receive |
|---|---|---|
| YW17 (laronidase biosimilar) | Experimental | YW17 (2.9 mg/5 mL) produced by CinnaGen Company, is administered 0.58 mg/kg weekly. Receives: Biological: Laronidase; Drug: Antihistamine; Drug: Antipyretic |
| Aldurazyme® | Active comparator | Aldurazyme® (2.9 mg/5 mL), is administered 0.58 mg/kg weekly. Receives: Biological: Laronidase; Drug: Antihistamine; Drug: Antipyretic |
What is being measured
The primary outcome is the main question the study is designed to answer.
mean uGAG
urinary glycosaminoglycan
Measured: Baseline, weeks 8, 10, 12, 20, 22, and 24
Secondary outcomes (4)
mean 6MWT
6-minute walking test
Measured: Baseline, week 12, week 24
mean predicted FVC
Forced vital capacity
Measured: Baseline, week 12, week 24
mean enzyme activity level
Enzyme activity assay
Measured: Weeks 11 and 23
Number of participants with adverse events
All reported AEs are classified based on the Medical Dictionary for Regulatory Activities (MedDRA) terms and are graded according to the Common Terminology Criteria for Adverse Events (CTCAE) v5.0.
Measured: During the study period (screening visit up to week 24)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 May 2024
- Study start10 Sep 2022
- Primary completion12 Aug 2023
- Study completion18 Nov 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 Type 1Studies 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 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cinnagen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ali Rabani, Professor, Principal investigator, Professor of Pediatric Endocrinology & Metabolism, Department of Pediatrics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06406153
- Sponsor's protocol ID
- CIN.LAR.AR.III.97
- Status last verified
- Aug 2024
- First submitted
- 27 Apr 2024
- Last updated
- 6 Aug 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Rabbani A, Alaei M, Asl SN, Setoodeh A, Shakiba M, Salehpour S, Eshraghi P, Salehpour O, Sabzvari A, Kafi H, Jarollahi A. Efficacy and safety of a biosimilar laronidase versus the reference laronidase in patients with mucopolysaccharidosis type I. Sci Rep. 2025 Aug 19;15(1):30427. doi: 10.1038/s41598-025-16351-4.
PubMed 40830667
Study at a glance
From the registry record, last updated by the sponsor on 6 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase III, Single Arm, Cross-over, Multicenter Clinical Trial to Compare Efficacy and Safety of YW17(Laronidase; CinnaGen) Versus Laronidase (Aldurazyme®; Genzyme, BioMarin) in Patients With Mucopolysaccharidosis Type I (MPS I)"
- ClinicalTrials.gov ID
- NCT06406153
- Acronym / sponsor ID
- CIN.LAR.AR.III.97
- Conditions
- Mucopolysaccharidosis Type 1
- Keywords
- Urinary Glycosaminoglycans, Laronidase, Mucopolysaccharidosis type I, Aldurazyme®
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- CinnagenIndustry (pharma, biotech, devices)
- Study start
- 10 Sep 2022 (actual)
- Primary completion
- 12 Aug 2023 (actual)
- Study completion
- 18 Nov 2023 (actual)
- First posted
- 9 May 2024 (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.
The purpose of this phase III study is to assess the efficacy and safety of YW17 produced by CinnaGen Company compared to Aldurazyme® in mucopolysaccharidosis type I (MPS I) patients.
All patients receive Aldurazyme® for 12 weeks, followed by YW17 for another 12 weeks.
The primary outcome is the assessment of the maintenance of the mean uGAG levels at the end of each medication administration. The secondary outcomes are the assessment of 6-minute walking test (6MWT), predicted forced vital capacity (FVC), enzyme activity assay, and adverse events (AEs).
Detailed description
This is a phase III, single-sequence, cross-over study to assess the efficacy and safety of YW17 produced by CinnaGen Company in comparison with Aldurazyme® in MPS I patients.
All patients receive 0.58 mg/kg of Aldurazyme® for 12 weeks and then receive 0.58 mg/kg of YW17 for another 12 weeks.
Premedication with antipyretics and/or antihistamines is administered for all patients one hour before the infusion.
The primary outcome is to compare the mean uGAG levels at weeks 8, 10, and 12 (related to Aldurazyme®) with the mean uGAG levels at weeks 20, 22, and 24 (related to YW17).
The secondary outcomes, including 6MWT and FVC are assessed at the beginning and the end of each medication administration. Enzyme activity is assessed at the end of each medication administration. Safety assessments are performed during the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| YW17 (laronidase biosimilar) | Experimental | Biological: Laronidase, Drug: Antihistamine, Drug: AntipyreticYW17 (2.9 mg/5 mL) produced by CinnaGen Company, is administered 0.58 mg/kg weekly. |
| Aldurazyme® | Active comparator | Biological: Laronidase, Drug: Antihistamine, Drug: AntipyreticAldurazyme® (2.9 mg/5 mL), is administered 0.58 mg/kg weekly. |
Who may qualify
- Ages
- 5 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Age 5-18
- Diagnosed with MPS I
- Signing informed consent form
Key exclusion criteria (10 listed)
- Prior bone marrow transplantation or being a candidate for receiving haematopoietic stem cell transplantation (HSCT)
- Prior tracheotomy
- Being naïve to laronidase
- Acute hydrocephalus
- Abnormal renal function determined by measuring serum creatinine and blood urea nitrogen (BUN) levels
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 |
|---|---|---|
| LaronidaseLaronidase as Aldurazyme® (the first 12 weeks) or YW17 (the second 12 weeks) is administered. | Biological | Aldurazyme®, YW17 (laronidase biosimilar) |
| AntihistamineAn antihistamine is administered one hour before the infusion. | Drug | Aldurazyme®, YW17 (laronidase biosimilar) |
| AntipyreticAn antipyretic is administered one hour before the infusion. | Drug | Aldurazyme®, YW17 (laronidase biosimilar) |
Primary outcome
mean uGAG
Time frame: Baseline, weeks 8, 10, 12, 20, 22, and 24
urinary glycosaminoglycan
4 secondary outcomes
mean 6MWT
Time frame: Baseline, week 12, week 24
mean predicted FVC
Time frame: Baseline, week 12, week 24
mean enzyme activity level
Time frame: Weeks 11 and 23
Number of participants with adverse events
Time frame: During the study period (screening visit up to week 24)
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 6 Aug 2024.
- Derived from this study: PubMed 40830667Rabbani A, Alaei M, Asl SN, Setoodeh A, Shakiba M, Salehpour S, Eshraghi P, Salehpour O, Sabzvari A, Kafi H, Jarollahi A. Efficacy and safety of a biosimilar laronidase versus the reference laronidase in patients with mucopolysaccharidosis type I. Sci Rep. 2025 Aug 19;15(1):30427. doi: 10.1038/s41598-025-16351-4.
- Individual participant dataSharing planned: No
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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.
Ali Rabani, Professor principal investigator
Professor of Pediatric Endocrinology & Metabolism, Department of Pediatrics
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Laronidase, Antihistamine and Antipyretic in people with Mucopolysaccharidosis Type 1, run by Cinnagen at 4 sites in Iran.
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 3 inclusion and 10 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 6 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Iran. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
mean uGAG (time frame: Baseline, weeks 8, 10, 12, 20, 22, and 24). 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 NCT06406153, 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 6 Aug 2024.