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Study of Idursulfase-beta (GC1111) in Hunter Syndrome

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ClinicalTrials.gov Identifier: NCT02663024
Recruitment Status : Not yet recruiting
First Posted : January 26, 2016
Last Update Posted : January 26, 2016
Sponsor:
Information provided by (Responsible Party):
Green Cross Corporation

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.

Condition or disease Intervention/treatment Phase
Mucopolysaccharidosis II Biological: idursulfase beta Biological: idursulfase Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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 Start Date : December 2016
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : June 2020


Arm Intervention/treatment
Experimental: Arm 1
0.5 mg/kg, iv, weekly infusion of idursulfase beta for 24 weeks
Biological: idursulfase beta
IV, weekly infusion for 24 weeks
Other Name: GC1111

Experimental: Arm 2
1.0 mg/kg, iv, weekly infusion of idursulfase beta for 24 weeks
Biological: idursulfase beta
IV, weekly infusion for 24 weeks
Other Name: GC1111

Experimental: Arm 3
1.5 mg/kg, iv, weekly infusion of idursulfase beta for 24 weeks
Biological: idursulfase beta
IV, weekly infusion for 24 weeks
Other Name: GC1111

Active Comparator: Arm 4
0.5mg/kg, iv, weekly infusion of idursulfase for 24 weeks
Biological: idursulfase
0.5 mg/kg, iv, weekly infusion for 24 weeks
Other Name: Elaprase




Primary Outcome Measures :
  1. Percent change from baseline in urinary GAG(Glycosaminoglycans) at Week 25 [ Time Frame: Baseline to Week 25 ]

Secondary Outcome Measures :
  1. Change from baseline in urinary GAG at Week 25 [ Time Frame: Baseline to Week 25 ]
  2. Change from baseline in Six Minute Walk Test at Week 25 [ Time Frame: Baseline to Week 25 ]
  3. Percent change from baseline in Six Minute Walk Test at Week 25 [ Time Frame: Baseline to Week 25 ]
  4. Change from baseline in Liver volume at Week 25 [ Time Frame: Baseline to Week 25 ]
    Liver volume measured by MRI

  5. Percent change from baseline in Liver volume at Week 25 [ Time Frame: Baseline to Week 25 ]
    Liver volume measured by MRI

  6. Change from baseline in Spleen volume at Week 25 [ Time Frame: Baseline to Week 25 ]
    Spleen volume measured by MRI

  7. Percent change from baseline in Spleen volume at Week 25 [ Time Frame: Baseline to Week 25 ]
    Spleen volume measured by MRI

  8. Incidence of Adverse Events and Serious Adverse Events [ Time Frame: Baseline to Week 25 ]
  9. Safety changes from baseline in clinical laboratory tests, physical examination and vital signs [ Time Frame: Baseline to Week 25 ]
  10. Immunogenicity [ Time Frame: Baseline to Week 25 ]
    anti-drug-antibody

  11. Pharmacokinetic profile - Area under the serum concentration time curve (AUClast) [ Time Frame: 1 and 17 week ]
  12. Pharmacokinetic profile - Maximum observed peak plasma concentration (Cmax) [ Time Frame: 1 and 17 week ]
  13. Pharmacokinetic profile - Time at which Cmax is observed (Tmax) [ Time Frame: 1 and 17 week ]


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Ages Eligible for Study:   5 Years to 35 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02663024


Contacts
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Contact: Sangmi Kang 82-31-260-1957 sm6220@greencross.com

Sponsors and Collaborators
Green Cross Corporation

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Responsible Party: Green Cross Corporation
ClinicalTrials.gov Identifier: NCT02663024     History of Changes
Other Study ID Numbers: GC1111B_P2
First Posted: January 26, 2016    Key Record Dates
Last Update Posted: January 26, 2016
Last Verified: January 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Additional relevant MeSH terms:
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Mucopolysaccharidosis II
Mucopolysaccharidoses
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Genetic Diseases, X-Linked
Heredodegenerative Disorders, Nervous System