Mucopolysaccharidosis IIPhase 2NCT02663024
Study of Idursulfase-beta (GC1111) in Hunter Syndrome
A Phase 2 study testing idursulfase beta and idursulfase in people with Mucopolysaccharidosis II, run by Green Cross Corporation.
Trial 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. Read more about this study
- Who is it for?
- Ages 5 to 35 years, men only
- Conditions the study is for
- Mucopolysaccharidosis II
- What is being tested?
- idursulfase beta and idursulfase
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- No sites listed yet
- Study dates
- Dec 2016to Jun 2020
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 35 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
No sites are listed yet.
About this study
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
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.
Read the 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.
- Conditions
- Mucopolysaccharidosis II
- Being tested
- idursulfase beta, idursulfase
- Medical subject headings
- Mucopolysaccharidosis II
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
idursulfase betaBiological
Also called GC1111
IV, weekly infusion for 24 weeks
idursulfaseBiological
Also called Elaprase
0.5 mg/kg, iv, weekly infusion for 24 weeks
| Study group | Role | What they receive |
|---|---|---|
| Arm 1 | Experimental | 0.5 mg/kg, iv, weekly infusion of idursulfase beta for 24 weeks Receives: Biological: idursulfase beta |
| Arm 2 | Experimental | 1.0 mg/kg, iv, weekly infusion of idursulfase beta for 24 weeks Receives: Biological: idursulfase beta |
| Arm 3 | Experimental | 1.5 mg/kg, iv, weekly infusion of idursulfase beta for 24 weeks Receives: Biological: idursulfase beta |
| Arm 4 | Active comparator | 0.5mg/kg, iv, weekly infusion of idursulfase for 24 weeks Receives: Biological: idursulfase |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent change from baseline in urinary GAG(Glycosaminoglycans) at Week 25
Measured: Baseline to Week 25
Secondary outcomes (13)
Change from baseline in urinary GAG at Week 25
Measured: Baseline to Week 25
Change from baseline in Six Minute Walk Test at Week 25
Measured: Baseline to Week 25
Percent change from baseline in Six Minute Walk Test at Week 25
Measured: Baseline to Week 25
Change from baseline in Liver volume at Week 25
Liver volume measured by MRI
Measured: Baseline to Week 25
Percent change from baseline in Liver volume at Week 25
Liver volume measured by MRI
Measured: Baseline to Week 25
Change from baseline in Spleen volume at Week 25
Spleen volume measured by MRI
Measured: Baseline to Week 25
Percent change from baseline in Spleen volume at Week 25
Spleen volume measured by MRI
Measured: Baseline to Week 25
Incidence of Adverse Events and Serious Adverse Events
Measured: Baseline to Week 25
Safety changes from baseline in clinical laboratory tests, physical examination and vital signs
Measured: Baseline to Week 25
Immunogenicity
anti-drug-antibody
Measured: Baseline to Week 25
Pharmacokinetic profile - Area under the serum concentration time curve (AUClast)
Measured: 1 and 17 week
Pharmacokinetic profile - Maximum observed peak plasma concentration (Cmax)
Measured: 1 and 17 week
Pharmacokinetic profile - Time at which Cmax is observed (Tmax)
Measured: 1 and 17 week
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jan 2016 (estimated)
- Study startDec 2016
- Primary completionDec 2019 (estimated)
- Study completionJun 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mucopolysaccharidosis IIStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Green Cross Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02663024
- Sponsor's protocol ID
- GC1111B_P2
- Status last verified
- Jan 2016
- First submitted
- 17 Jan 2016
- Last updated
- 26 Jan 2016
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Jan 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02663024
- Acronym / sponsor ID
- GC1111B_P2
- Conditions
- Mucopolysaccharidosis II
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Green Cross CorporationIndustry (pharma, biotech, devices)
- Study start
- Dec 2016
- Primary completion
- Dec 2019 (estimated)
- Study completion
- Jun 2020 (estimated)
- First posted
- 26 Jan 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Arm 1 | Experimental | Biological: idursulfase beta0.5 mg/kg, iv, weekly infusion of idursulfase beta for 24 weeks |
| Arm 2 | Experimental | Biological: idursulfase beta1.0 mg/kg, iv, weekly infusion of idursulfase beta for 24 weeks |
| Arm 3 | Experimental | Biological: idursulfase beta1.5 mg/kg, iv, weekly infusion of idursulfase beta for 24 weeks |
| Arm 4 | Active comparator | Biological: idursulfase0.5mg/kg, iv, weekly infusion of idursulfase for 24 weeks |
Who may qualify
- Ages
- 5 to 35 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| idursulfase betaAlso: GC1111IV, weekly infusion for 24 weeks | Biological | Arm 1, Arm 2, Arm 3 |
| idursulfaseAlso: Elaprase0.5 mg/kg, iv, weekly infusion for 24 weeks | Biological | Arm 4 |
Primary outcome
Percent change from baseline in urinary GAG(Glycosaminoglycans) at Week 25
Time frame: Baseline to Week 25
13 secondary outcomes
Change from baseline in urinary GAG at Week 25
Time frame: Baseline to Week 25
Change from baseline in Six Minute Walk Test at Week 25
Time frame: Baseline to Week 25
Percent change from baseline in Six Minute Walk Test at Week 25
Time frame: Baseline to Week 25
Change from baseline in Liver volume at Week 25
Time frame: Baseline to Week 25
Percent change from baseline in Liver volume at Week 25
Time frame: Baseline to Week 25
Change from baseline in Spleen volume at Week 25
Time frame: Baseline to Week 25
Percent change from baseline in Spleen volume at Week 25
Time frame: Baseline to Week 25
Incidence of Adverse Events and Serious Adverse Events
Time frame: Baseline to Week 25
Safety changes from baseline in clinical laboratory tests, physical examination and vital signs
Time frame: Baseline to Week 25
Immunogenicity
Time frame: Baseline to Week 25
Pharmacokinetic profile - Area under the serum concentration time curve (AUClast)
Time frame: 1 and 17 week
Pharmacokinetic profile - Maximum observed peak plasma concentration (Cmax)
Time frame: 1 and 17 week
Pharmacokinetic profile - Time at which Cmax is observed (Tmax)
Time frame: 1 and 17 week
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jan 2016.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing idursulfase beta and idursulfase in people with Mucopolysaccharidosis II, run by Green Cross Corporation.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 26 Jan 2016. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Sangmi Kang (82-31-260-1957, sm6220@greencross.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percent change from baseline in urinary GAG(Glycosaminoglycans) at Week 25 (time frame: Baseline to Week 25). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Jan 2016.