A phase III clinical trial to assess efficacy and safety of YW17 (laronidase; CinnaGen)

Phase 3

Conditions: Patients with Mucopolysaccharidosis type I (MPS I).
Mucopolysaccharidosis, type I
E76.0

Registration Number: IRCT20150303021315N30

Lead Sponsor: CinnaGen company

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Complete

Sex: All

Target Recruitment: 12

Inclusion Criteria

Patients Aged Between 5 to 18 years
Mucopolysaccharidosis type I confirmed
Willingness and having informed consent (patient or his/her parents/ legal guardian) to participate in this study

Exclusion Criteria

Prior bone marrow transplantation
Being a candidate for receiving hematopoietic stem cell transplantation
Prior Tracheotomy
Pregnancy
Lactation
Administration of any investigational drug within 30 days before study enrollment
Medical condition or other circumstance that could significantly interfere with study compliance
The severe organic disease that is not associated with mucopolysaccharidosis type 1
Known hypersensitivity to laronidase or components of the laronidase solution
Abnormal renal function determined by measuring serum creatinine and blood urea nitrogen (BUN) levels
Acute hydrocephalus
Prescribing laronidase for the first time for the patient (naive patient)

Study & Design

Study Type: interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Mean level of urinary Glycosaminoglycan adjusted by the level of urinary creatinine. Timepoint: Visits 9, 11, and 13 (related to the treatment with Aldurazyme®) and visits 21, 23, and final visit (related to the treatment with YW17). Method of measurement: Liquid chromatography tandem mass spectrometry.

Secondary Outcome Measures

Name	Time	Method
Distance traveled (based on meters) in 6 minutes (6-minute walk test). Timepoint: Visits 1, 13, and Final. Method of measurement: Timer and meter.;Forced Vital Capacity percentage. Timepoint: Visits 1, 13, and Final. Method of measurement: Spirometer.;Adverse events. Timepoint: At all visits. Method of measurement: All adverse events are assessed through patient reporting, physician diagnosis, and are then classified by severity (based on common terminology criteria for adverse events (CTCAE)), seriousness and relationship to the study drug.;Enzyme assay (a-L-iduronidase activity measurement). Timepoint: Immediately after, half an hour after, and 1.5 hours after the infusion of medicine at visits 12 and 24. Method of measurement: Liquid chromatography tandem mass spectrometry or High-throughput mass spectrometry.