Mucopolysaccharidosis Type 1
Conditions
Keywords
Urinary Glycosaminoglycans, Laronidase, Mucopolysaccharidosis type I, Aldurazyme®
Brief summary
The purpose of this phase III study is to assess the efficacy and safety of YW17 produced by CinnaGen Company compared to Aldurazyme® in mucopolysaccharidosis type I (MPS I) patients. All patients receive Aldurazyme® for 12 weeks, followed by YW17 for another 12 weeks. The primary outcome is the assessment of the maintenance of the mean uGAG levels at the end of each medication administration. The secondary outcomes are the assessment of 6-minute walking test (6MWT), predicted forced vital capacity (FVC), enzyme activity assay, and adverse events (AEs).
Detailed description
This is a phase III, single-sequence, cross-over study to assess the efficacy and safety of YW17 produced by CinnaGen Company in comparison with Aldurazyme® in MPS I patients. All patients receive 0.58 mg/kg of Aldurazyme® for 12 weeks and then receive 0.58 mg/kg of YW17 for another 12 weeks. Premedication with antipyretics and/or antihistamines is administered for all patients one hour before the infusion. The primary outcome is to compare the mean uGAG levels at weeks 8, 10, and 12 (related to Aldurazyme®) with the mean uGAG levels at weeks 20, 22, and 24 (related to YW17). The secondary outcomes, including 6MWT and FVC are assessed at the beginning and the end of each medication administration. Enzyme activity is assessed at the end of each medication administration. Safety assessments are performed during the study.
Interventions
BIOLOGICALLaronidase
Laronidase as Aldurazyme® (the first 12 weeks) or YW17 (the second 12 weeks) is administered.
DRUGAntihistamine
An antihistamine is administered one hour before the infusion.
DRUGAntipyretic
An antipyretic is administered one hour before the infusion.
Sponsors
Cinnagen
Study design
Allocation
NON_RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
5 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
* Age 5-18 * Diagnosed with MPS I * Signing informed consent form
Exclusion criteria
* Prior bone marrow transplantation or being a candidate for receiving haematopoietic stem cell transplantation (HSCT) * Prior tracheotomy * Being naïve to laronidase * Acute hydrocephalus * Abnormal renal function determined by measuring serum creatinine and blood urea nitrogen (BUN) levels * Any severe organic disease that is not associated with MPS I * Known hypersensitivity to laronidase or components of the laronidase solution * Presence of any medical condition or other circumstances that could significantly interfere with study compliance * Pregnancy and lactation * Administration of any investigational drug within 30 days before study enrollment
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| mean uGAG | Baseline, weeks 8, 10, 12, 20, 22, and 24 | urinary glycosaminoglycan |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| mean 6MWT | Baseline, week 12, week 24 | 6-minute walking test |
| mean predicted FVC | Baseline, week 12, week 24 | Forced vital capacity |
| mean enzyme activity level | Weeks 11 and 23 | Enzyme activity assay |
| Number of participants with adverse events | During the study period (screening visit up to week 24) | All reported AEs are classified based on the Medical Dictionary for Regulatory Activities (MedDRA) terms and are graded according to the Common Terminology Criteria for Adverse Events (CTCAE) v5.0. |
Countries
Iran
Outcome results
None listed