Patients with Mucopolysaccharidosis type I (MPS I). Mucopolysaccharidosis, type I
Conditions
Interventions
Intervention group: Patients will receive Aldurazyme® (laronidase produced by Genzyme, BioMarin) from visits 1 to 12 (weekly) and will receive YW17 (laronidase produced by Cinnagen Co.) from visit 13
Sponsors
CinnaGen company
Eligibility
Sex/Gender
All
Age
5 Years to 18 Years
Inclusion criteria
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
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)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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
| Measure | Time frame |
|---|---|
| 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. | — |
Countries
Iran (Islamic Republic of)
Contacts
Public ContactDr. Nasim Anjidani
Orchid Pharmed Co.
Outcome results
None listed