Mucopolysaccharidosis type VI. Other mucopolysaccharidoses
Conditions
Interventions
Intervention 1: Intervention group: Galsulphase (Biomarin) 1mg/kg for 12 weekly infusions, then Galsulfase (AryoGen Pharmed) 1mg/kg for another 12 weekly infusions. Intervention 2: Intervention group:
Sponsors
AryoGen Pharmed Company
Eligibility
Sex/Gender
All
Age
7 Years to No maximum
Inclusion criteria
Inclusion criteria: Age 7 years or older MPS VI diagnosis based on genetic or biochemical defects history Receiving Naglazyme continuously for at least 3 months prior to randomization informed consent
Exclusion criteria
Exclusion criteria: History of major organ or bone marrow transplantation or being a candidate for receiving bone marrow transplantation during the study period (6 months) based on investigator opinion Clinically significant spinal cord compression Having a history of severe or life-threatening hypersensitivity reaction to galsulfase or any of the components in the drug solution Pregnancy or lactation Administration of any investigational drug within 30 days before study enrollment Having a history or evidence of any condition, treatment, or laboratory data that, in the opinion of the investigator, would impair the results of the study, interfere with the patient's continued treatment during the study, or not be in the best interests of the patient's participation in the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Enzyme activity. Timepoint: Visit 12 and Vist 24 (before infusion, 120 min and 180 min after inf. start, immediately after inf.). Method of measurement: Blood sampling and laboratory test. | — |
Secondary
| Measure | Time frame |
|---|---|
| 6-minute walk test. Timepoint: Visits 0, 13, 25. Method of measurement: Measuring the distance (m) that the patient walks in 6 minutes.;12-minute walk test. Timepoint: Visits 0, 13, 25. Method of measurement: Measuring the distance (m) that the patient walks in 12 minutes.;Ejection fraction. Timepoint: Visits 0, 13, 25. Method of measurement: Echocardiography.;Valve area. Timepoint: Visits 0, 13, 25. Method of measurement: Echocardiography.;Forced Expiratory Volume in one second. Timepoint: Visits 0, 13, 25. Method of measurement: Spirometry.;Urinary Glycosaminoglycan. Timepoint: Visits 0, 7, 13, 19, 25. Method of measurement: Urine sampling and laboratory test.;Forced vital capacity. Timepoint: Visits 0, 13, 25. Method of measurement: Spirometry. | — |
Countries
Iran (Islamic Republic of)
Contacts
Public ContactHamidreza Kafi
Orchid Pharmed Company
Outcome results
None listed