Mucopolysaccharidosis IINCT01602601
A Study to Test the Possibility of Cross Reaction Induced by the Idursulfase Drug to GSK2788723
An observational study of Mucopolysaccharidosis II, run by GlaxoSmithKline at 1 site in Japan.
Trial summary
Study IDS116406 will be a non-interventional, phlebotomy study in Hunter Syndrome patients who are currently being treated with idursulfase, an enzyme replacement therapy, and in at least a single patient who is naïve to treatment, if possible to recruit. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Mucopolysaccharidosis II
- What is being tested?
- Idursulfase and GSK2788723
- Type of treatment
- Injection or infusion
- How many people
- 10 participants
- Where
- 1 siteJapan
- Study dates
- 9 Apr 2012to 11 Jun 2012
- Phase
- Observational
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Approximately 11 subjects will be enrolled in the study.
Who may be able to join
- A subject will be eligible for inclusion in this study only if patient meets criteria 1 and 2 or 1 and 3:
- Diagnosed with Hunter syndrome
- Patients with Hunter syndrome who are being treated with idursulfase
- Patients with Hunter syndrome who are naïve of idursulfase treatment (if possible*) *This study will also attempt to recruit 1 patient who has never received idursulfase. If a naïve patient has not been identified within an agreed amount of time between GSK and the investigator, the study will concluded without this sample
Who cannot join
- Subjects who are currently participating in another clinical trial are not permitted to be enrolled in this study.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Study to Test the Possibility of Cross Reaction of the Antibodies Induced by the ELAPRASE (R) to GSK2788723 ELAPRASE is a Trade Mark Owned by a Third Party
Study IDS116406 will be a non-interventional, phlebotomy study in Hunter Syndrome patients who are currently being treated with idursulfase, an enzyme replacement therapy, and in at least a single patient who is naïve to treatment, if possible to recruit. All patients enrolled into the study will have a single blood draw for the analysis of antibodies induced by this enzyme replacement therapy (idursulfase). Patient samples with positive responses to antibodies induced by idursulfase will be used to further evaluate whether the antibodies induced by idursulfase bind to GSK2788723 molecules in vitro and if these antibodies neutralize the bioactivity of GSK2788723 in vitro. Each subject will have a screening visit, which may occur at their regularly scheduled out-patient visit. If the patient consents to participate in the study, a blood sample (total volume of approximately 3mL) for immunogenicity analysis will be drawn before their current treatment infusion
Read the detailed description
Study IDS116406 will be a non-interventional, phlebotomy study in Hunter Syndrome patients who are currently being treated with idursulfase, an enzyme replacement therapy, and in at least a single patient who is naïve to treatment, if possible to recruit. All patients enrolled into the study will have a single blood draw for the analysis of antibodies induced by this enzyme replacement therapy (idursulfase). Patient samples with positive responses to antibodies induced by idursulfase will be used to further evaluate whether the antibodies induced by idursulfase bind to GSK2788723 molecules in vitro and if these antibodies neutralize the bioactivity of GSK2788723 in vitro. Each subject will have a screening visit, which may occur at their regularly scheduled out-patient visit. If the patient consents to participate in the study, a blood sample (total volume of approximately 3mL) for immunogenicity analysis will be drawn before their current treatment infusion
- Conditions
- Mucopolysaccharidosis II
- Being tested
- Idursulfase, GSK2788723
- Keywords
- Hunter syndrome, immunogenicity, antibodies
- Medical subject headings
- Mucopolysaccharidosis II
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IdursulfaseDrug
Also called recombinant IDS
recombinant version of IDS produced from human fibroblast. idursulfase is approved for every week (EW) intravenous (IV) administration.
GSK2788723Drug
GSK2788723 is being developed by JCR and GSK for the treatment of Hunter syndrome
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Patients will have a single blood draw for the analysis of antibodies induced by idursulfase. Samples will be used to further evaluate whether the antibodies induced by idursulfase bind to GSK2788723 molecules in vitro and if these antibodies neutralize the bioactivity of GSK2788723 in vitro. Receives: Drug: Idursulfase; Drug: GSK2788723 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Level and capability of binding and neutralizing antibodies induced by idursulfase after the administration of idursulfase
To assess the cross-reactivity of the antibodies induced by idursulfase to GSK2788723, in vitro
Measured: 1 Day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 May 2012 (estimated)
- Study start9 Apr 2012
- Primary completion11 Jun 2012
- Study completion11 Jun 2012
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.
- More trials run by GlaxoSmithKlineThe sponsor's other registered studies.
- 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
- Observational
- Observational model
- Case only
- Time perspective
- Cross-sectional
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- GlaxoSmithKline
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- GSK Clinical Trials, Study director, GlaxoSmithKline
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01602601
- Sponsor's protocol ID
- 116406
- Status last verified
- Sep 2017
- First submitted
- 17 May 2012
- Last updated
- 25 Sep 2017
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Sep 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study to Test the Possibility of Cross Reaction of the Antibodies Induced by the ELAPRASE (R) to GSK2788723 ELAPRASE is a Trade Mark Owned by a Third Party
- ClinicalTrials.gov ID
- NCT01602601
- Acronym / sponsor ID
- 116406
- Conditions
- Mucopolysaccharidosis II
- Keywords
- Hunter syndrome, immunogenicity, antibodies
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- GlaxoSmithKlineIndustry (pharma, biotech, devices)
- Study start
- 9 Apr 2012 (actual)
- Primary completion
- 11 Jun 2012 (actual)
- Study completion
- 11 Jun 2012 (actual)
- First posted
- 21 May 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.
Study IDS116406 will be a non-interventional, phlebotomy study in Hunter Syndrome patients who are currently being treated with idursulfase, an enzyme replacement therapy, and in at least a single patient who is naïve to treatment, if possible to recruit. All patients enrolled into the study will have a single blood draw for the analysis of antibodies induced by this enzyme replacement therapy (idursulfase). Patient samples with positive responses to antibodies induced by idursulfase will be used to further evaluate whether the antibodies induced by idursulfase bind to GSK2788723 molecules in vitro and if these antibodies neutralize the bioactivity of GSK2788723 in vitro. Each subject will have a screening visit, which may occur at their regularly scheduled out-patient visit. If the patient consents to participate in the study, a blood sample (total volume of approximately 3mL) for immunogenicity analysis will be drawn before their current treatment infusion
Detailed description
Study IDS116406 will be a non-interventional, phlebotomy study in Hunter Syndrome patients who are currently being treated with idursulfase, an enzyme replacement therapy, and in at least a single patient who is naïve to treatment, if possible to recruit. All patients enrolled into the study will have a single blood draw for the analysis of antibodies induced by this enzyme replacement therapy (idursulfase). Patient samples with positive responses to antibodies induced by idursulfase will be used to further evaluate whether the antibodies induced by idursulfase bind to GSK2788723 molecules in vitro and if these antibodies neutralize the bioactivity of GSK2788723 in vitro. Each subject will have a screening visit, which may occur at their regularly scheduled out-patient visit. If the patient consents to participate in the study, a blood sample (total volume of approximately 3mL) for immunogenicity analysis will be drawn before their current treatment infusion
Study design
- Observational model
- Case only
- Time perspective
- Cross-sectional
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | – | Drug: Idursulfase, Drug: GSK2788723Patients will have a single blood draw for the analysis of antibodies induced by idursulfase. Samples will be used to further evaluate whether the antibodies induced by idursulfase bind to GSK2788723 molecules in vitro and if these antibodies neutralize the bioactivity of GSK2788723 in vitro. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Approximately 11 subjects will be enrolled in the study.
Key inclusion criteria (4 listed)
- A subject will be eligible for inclusion in this study only if patient meets criteria 1 and 2 or 1 and 3:
- Diagnosed with Hunter syndrome
- Patients with Hunter syndrome who are being treated with idursulfase
- Patients with Hunter syndrome who are naïve of idursulfase treatment (if possible*) *This study will also attempt to recruit 1 patient who has never received idursulfase. If a naïve patient has not been identified within an agreed amount of time between GSK and the investigator, the study will concluded without this sample
Key exclusion criteria (1 listed)
- Subjects who are currently participating in another clinical trial are not permitted to be enrolled in this study.
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 |
|---|---|---|
| IdursulfaseAlso: recombinant IDSrecombinant version of IDS produced from human fibroblast. idursulfase is approved for every week (EW) intravenous (IV) administration. | Drug | Cohort 1 |
| GSK2788723GSK2788723 is being developed by JCR and GSK for the treatment of Hunter syndrome | Drug | Cohort 1 |
Primary outcome
Level and capability of binding and neutralizing antibodies induced by idursulfase after the administration of idursulfase
Time frame: 1 Day
To assess the cross-reactivity of the antibodies induced by idursulfase to GSK2788723, in vitro
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 25 Sep 2017.
- Results for study 116406 can be found on the GSK Clinical Study Register.Link provided by the sponsor.
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A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)
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An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB
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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.
GSK Clinical Trials study director
GlaxoSmithKline
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Mucopolysaccharidosis II, run by GlaxoSmithKline at 1 site in Japan.
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 1 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 25 Sep 2017. 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 Japan. 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.
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?
Level and capability of binding and neutralizing antibodies induced by idursulfase after the administration of idursulfase (time frame: 1 Day). 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 25 Sep 2017.