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 range5 Years – 18 Years

SexALL

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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

What they're measuring

mean uGAG

Timeframe: Baseline, weeks 8, 10, 12, 20, 22, and 24

Trial details

NCT IDNCT06406153

SponsorCinnagen

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2023-08-12

View on ClinicalTrials.gov More Mucopolysaccharidosis Type 1 trials