Mucopolysaccharidosis I MedDRA version: 20.1 Level: PT Classification code 10056886 Term: Mucopolysaccharidosis I System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: Aldurazyme®
Product Name: Aldurazyme
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: Laronidase
CAS Number: 210589-09-6
Concentration unit: IU/ml internatio
Sponsors
Genzyme Europe B. V
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Chinese participants have a documented diagnosis of MPS I confirmed by measurable clinical signs and symptoms of MPS I and fibroblast or leukocyte IDUA activity =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Participants are excluded from the study if any of the following criteria apply: Prior tracheostomy or bone marrow transplantation or hematopoietic stem cell transplantation. Have a plan to undergo bone marrow transplantation or hematopoietic stem cell transplantation within half a year after enrollment. Received an investigational drug, or device, other than Aldurazyme, within 30 days prior to study enrollment. Received an investigational gene therapy.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: •To evaluate the safety and tolerability of Aldurazyme in Chinese MPS I participants •To evaluate the efficacy of Aldurazyme on urinary glycosaminoglycans (uGAGs) after 26 weeks of treatment in Chinese MPS I participants ;Secondary Objective: •To evaluate the effect on uGAG level in Chinese MPS I participants •To evaluate liver volume (hepatomegaly) with Aldurazyme treatment in Chinese MPS I participants;Primary end point(s): 1/ Participants with adverse events (AEs) 2/ The change during the TE period in electrocardiogram (ECG), clinical laboratory parameters (hematology and biochemistry), and vital signs 3/ The percent change of uGAGs ;Timepoint(s) of evaluation of this end point: 1/ and 2/: Baseline to Week 27 3/: Baseline to Week 26 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1/ The percent change of uGAGs 2/ The absolute change of uGAGs 3/ The percent change of liver volume (Abdominal B type ultrasound examination) ;Timepoint(s) of evaluation of this end point: 1/ and 2/: Baseline to Week 2, Week 4, Week 8, Week 12 and Week 20 3/: Baseline to Week 26 | — |
Countries
China
Contacts
Public Contact-
Genzyme Europe B. V
Outcome results
None listed