Clinical trial NCT02663024: Study of Idursulfase-beta (GC1111) in Hunter Syndrome
Official title: Phase 2, Randomized, Double-blind, Active-controlled, Dose-ranging Study to Evaluate the Pharmacokinetics, Pharmacodynamics and Safety of Idursulfase-beta (GC1111) in Hunter Syndrome (Mucopolysaccharidosis II) Patients
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Mucopolysaccharidosis II
Lead sponsor: GC Biopharma Corp
Sponsor class: INDUSTRY
First posted: 2016-01-26
Enrollment: 20
Sex: MALE
Minimum age (years): 5
Maximum age (years): 35
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Percent change from baseline in urinary GAG(Glycosaminoglycans) at Week 25
timeFrame: Baseline to Week 25
Secondary outcomes:
measure: Change from baseline in urinary GAG at Week 25
timeFrame: Baseline to Week 25
measure: Change from baseline in Six Minute Walk Test at Week 25
timeFrame: Baseline to Week 25
measure: Percent change from baseline in Six Minute Walk Test at Week 25
timeFrame: Baseline to Week 25
measure: Change from baseline in Liver volume at Week 25
timeFrame: Baseline to Week 25
measure: Percent change from baseline in Liver volume at Week 25
timeFrame: Baseline to Week 25
measure: Change from baseline in Spleen volume at Week 25
timeFrame: Baseline to Week 25
measure: Percent change from baseline in Spleen volume at Week 25
timeFrame: Baseline to Week 25
measure: Incidence of Adverse Events and Serious Adverse Events
timeFrame: Baseline to Week 25
measure: Safety changes from baseline in clinical laboratory tests, physical examination and vital signs
timeFrame: Baseline to Week 25
measure: Immunogenicity
timeFrame: Baseline to Week 25
measure: Pharmacokinetic profile - Area under the serum concentration time curve (AUClast)
timeFrame: 1 and 17 week
measure: Pharmacokinetic profile - Maximum observed peak plasma concentration (Cmax)
timeFrame: 1 and 17 week
measure: Pharmacokinetic profile - Time at which Cmax is observed (Tmax)
timeFrame: 1 and 17 week
Other outcomes:
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