Efficacy and Safety of YW17 (Laronidase-CinnaGen) Compared to Aldurazyme® in MPS I Patients (NCT06406153) | Clinical Trial Compass
CompletedPhase 3
Efficacy and Safety of YW17 (Laronidase-CinnaGen) Compared to Aldurazyme® in MPS I Patients
Iran12 participantsStarted 2022-09-10
Plain-language 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.
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).
Who can participate
Age range
5 Years – 18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Age 5-18
* Diagnosed with MPS I
* Signing informed consent form
Exclusion Criteria:
* 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
* 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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
mean uGAG
Timeframe: Baseline, weeks 8, 10, 12, 20, 22, and 24