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IRCT20150303021315N30 IRCT 2022-05-02 CinnaGen company A phase III clinical trial to assess efficacy and safety of YW17 (laronidase; CinnaGen) 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) 2022-05-22 anticipated 12 Complete https://irct.ir/trial/59822 interventional Randomization: N/A, Blinding: Not blinded, Placebo: Not used, Assignment: Crossover, Purpose: Treatment, Other design features: This clinical trial consists of screening visit, visits 1 to 24, and final visit. After screening, the interval between visits is one week. 3 Patients with Mucopolysaccharidosis type I (MPS I). 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 to 24. The Dosing of laronidase is 0.58 milligram per kilogram of patient’s body weight. Laronidase is administered by intravenous infusion in 3 to 4 hours. The dosage form of laronidase (Both products) is vial consisting of a solution with a concentration of 2.9 milligrams per 5 milliliters to be diluted in normal saline. To minimize possible infusion-related reactions, all patients will receive acetaminophen and diphenhydramine one hour before each injection.. Undecided - It is not yet known if there will be a plan to make this available Justification or reason for indecision in sharing IPD is There is no more information.
public Dr. Nasim Anjidani
No 42. Attar sq, Attar st, Valiasr st, Vanak sq, Tehran, Iran
Tehran Iran (Islamic Republic of) 1994766411 +98 21 4347 3000 anjidani.n@orchidpharmed.com Orchid Pharmed Co. scientific Dr. Nasim Anjidani
No 42. Attar sq, Attar st, Valiasr st, Vanak sq, Tehran, Iran
Tehran Iran (Islamic Republic of) 1994766411 +98 21 4347 3000 anjidani.N@orchidpharmed.com Orchid Pharmed Company Iran (Islamic Republic of) Iran (Islamic Republic of) Iran (Islamic Republic of) Iran (Islamic Republic of) 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 5 years 18 years Both 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) E76.0 Mucopolysaccharidosis, type I Treatment - Drugs 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 to 24. The Dosing of laronidase is 0.58 milligram per kilogram of patient’s body weight. Laronidase is administered by intravenous infusion in 3 to 4 hours. The dosage form of laronidase (Both products) is vial consisting of a solution with a concentration of 2.9 milligrams per 5 milliliters to be diluted in normal saline. To minimize possible infusion-related reactions, all patients will receive acetaminophen and diphenhydramine one hour before each injection. 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. 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 (α-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. CinnaGen company Approved 2022-03-17 Ethics Committee of the Institute of Pharmaceutical Sciences (TIPS) The Institute of Pharmaceutical Sciences (TIPS), Faculty of Pharmacy, Tehran University of Medical Sciences, Poursina Avenue Tehran Tehran Iran (Islamic Republic of)