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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).
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| YW17 (laronidase biosimilar) | Experimental | YW17 (2.9 mg/5 mL) produced by CinnaGen Company, is administered 0.58 mg/kg weekly. |
|
| Aldurazyme® | Active Comparator | Aldurazyme® (2.9 mg/5 mL), is administered 0.58 mg/kg weekly. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Laronidase | Biological | Laronidase as Aldurazyme® (the first 12 weeks) or YW17 (the second 12 weeks) is administered. |
|
| Measure | Description | Time Frame |
|---|---|---|
| mean uGAG | urinary glycosaminoglycan | Baseline, weeks 8, 10, 12, 20, 22, and 24 |
| Measure | Description | Time Frame |
|---|---|---|
| mean 6MWT | 6-minute walking test | Baseline, week 12, week 24 |
| mean predicted FVC | Forced vital capacity | Baseline, week 12, week 24 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Ali Rabani, Professor | Professor of Pediatric Endocrinology & Metabolism, Department of Pediatrics | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Clinical Research Development Unit of Akbar Hospital, Faculty of Medicine | Mashhad | Iran | ||||
| Growth and Development Research Center, Childrens Medical Center |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 40830667 | Derived | 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. |
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| ID | Term |
|---|---|
| D008059 | Mucopolysaccharidosis I |
| ID | Term |
|---|---|
| D009083 | Mucopolysaccharidoses |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
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| ID | Term |
|---|---|
| D007068 | Iduronidase |
| D006633 | Histamine Antagonists |
| D058633 | Antipyretics |
| ID | Term |
|---|---|
| D006026 | Glycoside Hydrolases |
| D006867 | Hydrolases |
| D004798 | Enzymes |
| D045762 | Enzymes and Coenzymes |
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| Antihistamine | Drug | An antihistamine is administered one hour before the infusion. |
|
| Antipyretic | Drug | An antipyretic is administered one hour before the infusion. |
|
| mean enzyme activity level | Enzyme activity assay | 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. | During the study period (screening visit up to week 24) |
| Tehran |
| Iran |
| Loghman Hospital | Tehran | Iran |
| Mofid Childrens Hospital | Tehran | Iran |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D016464 | Lysosomal Storage Diseases |
| D017520 | Mucinoses |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D018494 |
| Histamine Agents |
| D018377 | Neurotransmitter Agents |
| D045504 | Molecular Mechanisms of Pharmacological Action |
| D020228 | Pharmacologic Actions |
| D020164 | Chemical Actions and Uses |
| D045505 | Physiological Effects of Drugs |