Mucopolysaccharidosis II
Conditions
Brief summary
This study evaluates the efficacy and safety of three doses of GC1111 in patients with Hunter Syndrome. Participants will be randomized to one of three doses of GC1111 or comparator.
Detailed description
This is a randomized, double-blind, active-controlled, dose-ranging study, where patient will receive one of the three doses of GC1111 (0.5 mg/kg, 1.0 mg/kg, and 1.5 mg/kg) or ELAPRASE 0.5 mg/kg. Approximately 20 patients will be administrated each study drug once every week as an iv infusion for 24 weeks. Efficacy of GC1111 will be evaluated in Six-Minute Walk Test (6MWT), urine Glycosaminoglycans(uGAG), liver and spleen volume, percent predicted Forced Vital Capacity(FVC), and cardiac size and function. Also immunogenicity, Pharmacokinetics(PK) and safety will be evaluated.
Interventions
BIOLOGICALidursulfase beta
IV, weekly infusion for 24 weeks
BIOLOGICALidursulfase
0.5 mg/kg, iv, weekly infusion for 24 weeks
Sponsors
Green Cross Corporation
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
MALE
Age
5 Years to 35 Years
Healthy volunteers
No
Inclusion criteria
* Male patients between 5 and 35 years of age * Informed consent form signed * Patients diagnosed with hunter syndrome * Previously untreated with an enzyme replacement therapy
Exclusion criteria
* History of tracheostomy, bone marrow transplant, or cord blood transplant * Treatment with another investigational product within 30 days prior to the start of study drug * Known hypersensitivity of any of the ingredients of study drug * Patient with severe hunter syndrome who cannot perform 6MWT * Female patients
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Percent change from baseline in urinary GAG(Glycosaminoglycans) at Week 25 | Baseline to Week 25 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from baseline in Six Minute Walk Test at Week 25 | Baseline to Week 25 | — |
| Percent change from baseline in Six Minute Walk Test at Week 25 | Baseline to Week 25 | — |
| Change from baseline in Liver volume at Week 25 | Baseline to Week 25 | Liver volume measured by MRI |
| Percent change from baseline in Liver volume at Week 25 | Baseline to Week 25 | Liver volume measured by MRI |
| Change from baseline in Spleen volume at Week 25 | Baseline to Week 25 | Spleen volume measured by MRI |
| Percent change from baseline in Spleen volume at Week 25 | Baseline to Week 25 | Spleen volume measured by MRI |
| Change from baseline in urinary GAG at Week 25 | Baseline to Week 25 | — |
| Safety changes from baseline in clinical laboratory tests, physical examination and vital signs | Baseline to Week 25 | — |
| Immunogenicity | Baseline to Week 25 | anti-drug-antibody |
| Pharmacokinetic profile - Area under the serum concentration time curve (AUClast) | 1 and 17 week | — |
| Pharmacokinetic profile - Maximum observed peak plasma concentration (Cmax) | 1 and 17 week | — |
| Pharmacokinetic profile - Time at which Cmax is observed (Tmax) | 1 and 17 week | — |
| Incidence of Adverse Events and Serious Adverse Events | Baseline to Week 25 | — |
Contacts
Primary ContactSangmi Kang
Outcome results
None listed