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)"

This clinical trial is focused on evaluating a new treatment for individuals with Mucopolysaccharidosis Type I (MPS I), a rare genetic disorder that affects the body's ability to break down certain substances. The study compares the effectiveness and safety of a new drug called YW17, developed by CinnaGen, to an existing treatment called Aldurazyme®. The participants in the study are people diagnosed with MPS I, and the researchers aim to determine if YW17 can offer similar or improved benefits in managing the condition. This study is important as it could provide an alternative treatment option for MPS I, potentially improving quality of life for those affected by this condition. Participants in the study first receive Aldurazyme® at a dose of 0.58 mg/kg for 12 weeks, followed by the same dosage of YW17 for another 12 weeks. Before each treatment, participants are given medications to prevent fever or allergic reactions. The study measures the effectiveness of the treatments by comparing levels of certain substances in urine at specified weeks, as well as evaluating walking ability and lung function at the start and end of each treatment period. Safety is monitored throughout the study to ensure participant well-being. This approach helps researchers understand how the new drug performs in real-world conditions, offering insights into its potential benefits and any risks involved.