Hunter SyndromePhase 3GC1111NCT01645189
Safety and Efficacy of Hunterase
A Phase 3 study testing Hunterase in people with Hunter Syndrome, run by Green Cross Corporation at 1 site in South Korea.
Trial summary
The objective of this study is to determine the safety and efficacy of once weekly dosing of idursulfase-beta 0.5mg/kg administered by intravenous(IV) infusion for Hunter syndrome patients < 6 years old. Read more about this study
- Who is it for?
- Ages Up to 5 years, men only
- Conditions the study is for
- Hunter Syndrome
- What is being tested?
- Hunterase
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteSouth Korea
- Study dates
- Jul 2012to Sep 2013
- Phase
- Phase 3
- 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 Up to 5 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- The patient has a diagnosis of Hunter syndrome based upon biochemical criteria:
- as measured in plasma, leukocytes, or fibroblasts,
- a deficiency in iduronate-2-sulfatase (I2S) enzyme activity of ≤ 10 % of the lower limit of the normal range
- That corresponds to one or more of the following:
- a normal enzyme activity level of one other sulfatase
- Confirmed as MPS2 by genetic test results
Show all 10Show fewer
- shows clinical symptoms/ visible signs of MPS2
- < 6 years old and male
- Patients who are able to comply with the study requirements
- The patient's parent(s), or patient's legal guardian must have given voluntary written consent to participate in the study
Who cannot join
- The patient has had a tracheostomy
- The patient has known severe hypersensitivity or shock to any of the components of idursulfase
- The patient has received treatment with another investigational therapy within 30 days prior to enrollment
- History of a stem cell transplant
- The patient has known severe hypersensitivity or shock to any of the components of test drug(excipient etc)
Trial locations and contacts
1 site in 1 country.
About this study
Official title: To Evaluate the Safety and Efficacy of Hunterase(Idursulfase-beta) in Hunter Syndrome Patients < 6 Years of Age Receiving Idursulfase Enzyme Replacement Therapy
The objective of this study is to determine the safety and efficacy of once weekly dosing of idursulfase-beta 0.5mg/kg administered by intravenous(IV) infusion for Hunter syndrome patients < 6 years old.
- Conditions
- Hunter Syndrome
- Being tested
- Hunterase
- Keywords
- Hunter syndrome, idursulfase-beta, Hunterase, GC1111
- Medical subject headings
- Mucopolysaccharidosis II
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HunteraseBiological
once weekly, 0.5mg/kg IV infusion
| Study group | Role | What they receive |
|---|---|---|
| Test drug | Experimental | Idursulfase-beta Receives: Biological: Hunterase |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse events
Measured: One year
Secondary outcomes (2)
change of anti-idursulfase-beta antibody status
Measured: baseline and one year
Percent Change of Urine GAG
Measured: baseline to 53 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jul 2012 (estimated)
- Study startJul 2012
- Primary completionSep 2013
- Study completionSep 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hunter SyndromeStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Green Cross Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Dong-Kyu Jin, Principal investigator, Samsung medical center, Seoul, Republic of Korea
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01645189
- Sponsor's protocol ID
- GC1111C
- Acronym
- GC1111
- Status last verified
- Jul 2014
- First submitted
- 16 Jul 2012
- Last updated
- 8 Jul 2014
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Jul 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- To Evaluate the Safety and Efficacy of Hunterase(Idursulfase-beta) in Hunter Syndrome Patients < 6 Years of Age Receiving Idursulfase Enzyme Replacement Therapy
- ClinicalTrials.gov ID
- NCT01645189
- Acronym / sponsor ID
- GC1111 / GC1111C
- Conditions
- Hunter Syndrome
- Keywords
- Hunter syndrome, idursulfase-beta, Hunterase, GC1111
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Green Cross CorporationIndustry (pharma, biotech, devices)
- Study start
- Jul 2012
- Primary completion
- Sep 2013 (actual)
- Study completion
- Sep 2013 (actual)
- First posted
- 20 Jul 2012 (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.
The objective of this study is to determine the safety and efficacy of once weekly dosing of idursulfase-beta 0.5mg/kg administered by intravenous(IV) infusion for Hunter syndrome patients < 6 years old.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Test drug | Experimental | Biological: HunteraseIdursulfase-beta |
Who may qualify
- Ages
- Up to 5 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (10 listed)
- The patient has a diagnosis of Hunter syndrome based upon biochemical criteria:
- < 6 years old and male
- Patients who are able to comply with the study requirements
- The patient's parent(s), or patient's legal guardian must have given voluntary written consent to participate in the study
Key exclusion criteria (5 listed)
- The patient has had a tracheostomy
- The patient has known severe hypersensitivity or shock to any of the components of idursulfase
- The patient has received treatment with another investigational therapy within 30 days prior to enrollment
- History of a stem cell transplant
- The patient has known severe hypersensitivity or shock to any of the components of test drug(excipient etc)
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 |
|---|---|---|
| Hunteraseonce weekly, 0.5mg/kg IV infusion | Biological | Test drug |
Primary outcome
Incidence of adverse events
Time frame: One year
2 secondary outcomes
change of anti-idursulfase-beta antibody status
Time frame: baseline and one year
Percent Change of Urine GAG
Time frame: baseline to 53 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jul 2014.
Related trials for Hunter Syndrome
Active, not recruitingPhase 2
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Mucopolysaccharidosis Disorders, Hurler Syndrome
Enrolling by invitationPhase 2/3
An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007
Mucopolysaccharidosis II
Active, not recruitingPhase 3
A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)
Mucopolysaccharidosis II
Active, not recruitingPhase 2/3
A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II
Mucopolysaccharidosis Type II, Metabolic Diseases
Active, not recruitingPhase 2/3
A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)
Mucopolysaccharidosis II
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.
Dong-Kyu Jin principal investigator
Samsung medical center, Seoul, Republic of Korea
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Hunterase in people with Hunter Syndrome, run by Green Cross Corporation at 1 site in South Korea.
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 10 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: finished, as of the sponsor's last update on 8 Jul 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of adverse events (time frame: One year). 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 8 Jul 2014.