Mucopolysaccharidosis IIPhase 1/2NCT01301898
To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients
A Phase 1/2 study testing GC1111_0.5mg/kg, GC1111_1.0mg/kg and Elaprase_0.5mg/kg in people with Mucopolysaccharidosis II, run by Green Cross Corporation at 1 site in South Korea.
Trial summary
The purpose of this study is to evaluate the safety and efficacy of GC1111 (recombinant human iduronate-w-sulfatase) in Hunter Syndrome (Mucopolysaccharidosis II) patients Read more about this study
- Who is it for?
- Ages 6 to 35 years, men only
- Conditions the study is for
- Mucopolysaccharidosis II
- What is being tested?
- GC1111_0.5mg/kg, GC1111_1.0mg/kg and Elaprase_0.5mg/kg
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 31 participants
- Where
- 1 siteSouth Korea
- Study dates
- May 2010to Mar 2011
- Phase
- Phase 1/2
- 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 6 to 35 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients with diagnosis of MPS II based on both clinical and biochemical criteria
- Male, ages 6 to 35 years old
- Patients who are able to comply with the study requirements
- Patients who have given voluntary written consent to participate in the study
- Patients who is acceptable for using an appropriate method of contraception
Who cannot join
- History of a tracheostomy or a bone marrow transplant
- Known hypersensitivity to idursulfase
- Known shock to idursulfase
- History of receiving treatment with another investigational therapy within the past 30 days
- History of a stem cell transplant
- Known hypersensitivity to any of the components of idursulfase
- Female
Trial locations and contacts
1 site in 1 country.
South Korea1 site
Samsung Medical Center
Seoul, Irwon-dong, Gangnam-Gu, 135-710
About this study
Official title: Randomized, Single-blind, Active-controlled, Phase 1/2 Study to Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome (Mucopolysaccharidosis II) Patients
The purpose of this study is to evaluate the safety and efficacy of GC1111 (recombinant human iduronate-w-sulfatase) in Hunter Syndrome (Mucopolysaccharidosis II) patients
- Conditions
- Mucopolysaccharidosis II
- Being tested
- GC1111_0.5mg/kg, GC1111_1.0mg/kg, Elaprase_0.5mg/kg
- Keywords
- Hunter syndrome, MPS II, Idursulfase
- Medical subject headings
- Mucopolysaccharidosis II, Sudden Infant Death
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GC1111_0.5mg/kgDrug
GC1111_1.0mg/kgDrug
Elaprase_0.5mg/kgDrug
| Study group | Role | What they receive |
|---|---|---|
| GC1111_0.5mg/kg | Experimental | Not described Receives: Drug: GC1111_0.5mg/kg |
| GC1111_1.0mg/kg | Experimental | Not described Receives: Drug: GC1111_1.0mg/kg |
| Elaprase_0.5mg/kg | Active comparator | Not described Receives: Drug: Elaprase_0.5mg/kg |
What is being measured
The primary outcome is the main question the study is designed to answer.
Primary Outcome
measurement of % change of Urine GAG
Measured: baseline, every 4 weeks
Secondary outcomes (1)
Secondary Outcome
- Measurements of the six-minute-walk test (6-MWT)
- Measurements of liver volume
- Measurements of heart size and heart function
- Measurements of joint range of motion
- Measurements of urine GAG levels
- Measurements of pulmonary function
- Evaluate the safety: vital signs, physical examination, laboratory tests, adverse events, immunogenecity
Measured: baseline, every 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Feb 2011 (estimated)
- Study startMay 2010
- Primary completionMar 2011
- Study completionMar 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 31 participants (actual)
Sponsor and investigators
- Lead sponsor
- Green Cross Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01301898
- Sponsor's protocol ID
- GC1111_P1/2
- Status last verified
- Feb 2012
- First submitted
- 22 Feb 2011
- Last updated
- 14 Feb 2012
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Sohn YB, Cho SY, Park SW, Kim SJ, Ko AR, Kwon EK, Han SJ, Jin DK. Phase I/II clinical trial of enzyme replacement therapy with idursulfase beta in patients with mucopolysaccharidosis II (Hunter syndrome). Orphanet J Rare Dis. 2013 Mar 18;8:42. doi: 10.1186/1750-1172-8-42.
PubMed 23497636
Study at a glance
From the registry record, last updated by the sponsor on 14 Feb 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized, Single-blind, Active-controlled, Phase 1/2 Study to Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome (Mucopolysaccharidosis II) Patients
- ClinicalTrials.gov ID
- NCT01301898
- Acronym / sponsor ID
- GC1111_P1/2
- Conditions
- Mucopolysaccharidosis II
- Keywords
- Hunter syndrome, MPS II, Idursulfase
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 31 participants (actual)
- Lead sponsor
- Green Cross CorporationIndustry (pharma, biotech, devices)
- Study start
- May 2010
- Primary completion
- Mar 2011 (actual)
- Study completion
- Mar 2011 (actual)
- First posted
- 23 Feb 2011 (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 purpose of this study is to evaluate the safety and efficacy of GC1111 (recombinant human iduronate-w-sulfatase) in Hunter Syndrome (Mucopolysaccharidosis II) patients
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| GC1111_0.5mg/kg | Experimental | Drug: GC1111_0.5mg/kg |
| GC1111_1.0mg/kg | Experimental | Drug: GC1111_1.0mg/kg |
| Elaprase_0.5mg/kg | Active comparator | Drug: Elaprase_0.5mg/kg |
Who may qualify
- Ages
- 6 to 35 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Patients with diagnosis of MPS II based on both clinical and biochemical criteria
- Male, ages 6 to 35 years old
- Patients who are able to comply with the study requirements
- Patients who have given voluntary written consent to participate in the study
- Patients who is acceptable for using an appropriate method of contraception
Key exclusion criteria (7 listed)
- History of a tracheostomy or a bone marrow transplant
- Known hypersensitivity to idursulfase
- Known shock to idursulfase
- History of receiving treatment with another investigational therapy within the past 30 days
- History of a stem cell transplant
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 |
|---|---|---|
| GC1111_0.5mg/kg | Drug | GC1111_0.5mg/kg |
| GC1111_1.0mg/kg | Drug | GC1111_1.0mg/kg |
| Elaprase_0.5mg/kg | Drug | Elaprase_0.5mg/kg |
Primary outcome
Primary Outcome
Time frame: baseline, every 4 weeks
measurement of % change of Urine GAG
1 secondary outcome
Secondary Outcome
Time frame: baseline, every 12 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 14 Feb 2012.
- Derived from this study: PubMed 23497636Sohn YB, Cho SY, Park SW, Kim SJ, Ko AR, Kwon EK, Han SJ, Jin DK. Phase I/II clinical trial of enzyme replacement therapy with idursulfase beta in patients with mucopolysaccharidosis II (Hunter syndrome). Orphanet J Rare Dis. 2013 Mar 18;8:42. doi: 10.1186/1750-1172-8-42.
Related trials for Mucopolysaccharidosis II
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 1/2
An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB
Mucopolysaccharidosis III-B
RecruitingPhase 2/3
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
MPS IIIA, Sanfilippo Syndrome
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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing GC1111_0.5mg/kg, GC1111_1.0mg/kg and Elaprase_0.5mg/kg in people with Mucopolysaccharidosis II, 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 5 inclusion and 7 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 14 Feb 2012. 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
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?
Primary Outcome (time frame: baseline, every 4 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01301898, which also lists 1 linked publication.
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 14 Feb 2012.