Hunter SyndromeNCT00882921
An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients
An observational study of Hunter Syndrome, run by Shire at 6 sites in 3 countries.
Trial summary
The objective of this study is to evaluate the effect of anti-idursulfase antibodies on idursulfase safety (measured by infusion related adverse events) between patients who develop anti-idursulfase antibodies and patients who do not after long-term idursulfase enzyme replacement therapy (ERT). Read more about this study
- Who is it for?
- Ages 5 years and older, men only
- Conditions the study is for
- Hunter Syndrome
- What is being tested?
- Idursulfase
- How many people
- 26 participants
- Where
- 6 sitesin 3 countries
- Study dates
- 14 Oct 2008to 8 Feb 2013
- Phase
- Observational
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with Hunter syndrome
Who may be able to join
- Patients must meet all of the following criteria to be considered eligible for enrollment:
- The patient is male and enrolled in the HOS (i.e., meets the entry criteria of a documented diagnosis of Hunter syndrome)
- The patient is ≥ 5 years-old
- The patient is on idursulfase treatment or scheduled to begin idursulfase treatment within 30 days of study enrollment
- The patient, patient's parent(s), or patient's legally authorized guardian must have voluntarily signed an Institutional Review Board (IRB)/Independent Ethics Committee (IEC)-approved informed consent form after all relevant aspects of the study have been explained and discussed with the patient, patient's parent(s), or patient's legally authorized guardian.
Who cannot join
- Patients who meet any of the following criteria are not eligible for this study:
- The patient has received biologic/ERT products other than idursulfase, or other investigational product(s) for any reason within 30 days prior to study entry.
- The patient has a life expectancy of < 2 years
- The patient is unable to comply with the protocol, e.g., has a clinically relevant medical condition making implementation of the protocol difficult; has an uncooperative attitude; is unable to return for safety evaluations; or is otherwise unlikely to complete the study, as determined by the Investigator.
Trial locations and contacts
6 sites in 3 countries.
6 sites in 3 countries
United Kingdom3 sites
Birmingham Children's Hospital
Birmingham, B46NH
Great Ormond Street Hospital
London, WC1N 3JH
Central Manchester University Hospitals, St. Mary's Hospital
Manchester, M139WL
United States2 sites
Children's Hospitals and Clinics of Minnesota, Division of Genetics
Minneapolis, Minnesota, 55404
Children's Hospital & Research Center Oakland
Oakland, California, 94609
Brazil1 site
Hospital de Clinicas de Porto Alegre, Servico de Genetica Medica
Porto Alegre, Rio Grande do Sul, 90035-903
About this study
Official title: A Multi-Center Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients Enrolled in the Hunter Outcome Survey (HOS) Receiving Idursulfase Enzyme Replacement Therapy
The objective of this study is to evaluate the effect of anti-idursulfase antibodies on idursulfase safety (measured by infusion related adverse events) between patients who develop anti-idursulfase antibodies and patients who do not after long-term idursulfase enzyme replacement therapy (ERT).
Read the detailed description
This study is being conducted to satisfy post-marketing commitments to monitor anti-idursulfase antibody development in Hunter syndrome patients after long-term idursulfase enzyme replacement therapy. The study will be conducted as a sub-study within the Hunter Outcome Survey (HOS). Hunter syndrome patients in the HOS who have previously received idursulfase as well as treatment-naive patients who will begin idursulfase treatment within 30 days of study enrollment will be included.
- Conditions
- Hunter Syndrome
- Being tested
- Idursulfase
- Keywords
- MPS 2, MPSII, hunter's disease treatment, enzyme replacement therapy, hunter's disease, iduronate 2 sulfatase, mps diagnosis, mps symptoms, iduronate sulfatase, MPS II treatment, chronic ear infection, hunters syndrome, mps ii therapy, MPS2, hunter's syndrome, Hunter syndrome, elaprase, lysosomal storage disease, hunter syndrome therapy, mps society
- Medical subject headings
- Mucopolysaccharidosis II, Sudden Infant Death, Lysosomal Storage Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IdursulfaseBiological
Also called Elaprase
Patients received idursulfase as prescribed by their physician following locally approved prescribing information. Patients will not be provided idursulfase by Shire Human Genetic Therapies, Inc. or the HOS.
| Study group | Role | What they receive |
|---|---|---|
| Elaprase | Idursulfase 0.5 mg/kg Weekly Receives: Biological: Idursulfase |
What is being measured
The primary outcome is the main question the study is designed to answer.
Infusion-Related Adverse Event (IRAE) Rates Between IgG Anti-idursulfase Antibody Positive (Ab+) and Anti-idursulfase IgG Antibody Negative (Ab-) Patients
The primary analysis of how presence of antibodies affected IRAE rates was performed based on a negative binomial regression model. This was done to account for potentially differential follow-up time between antibody groups.
Measured: Baseline to 109 Weeks
Secondary outcomes (1)
Change From Baseline in uGAG Levels to 109 Weeks
Urine GAG
Measured: Baseline to 109 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Apr 2009 (estimated)
- Study start14 Oct 2008
- Primary completion8 Feb 2013
- Study completion8 Feb 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00882921
- Sponsor's protocol ID
- HGT-ELA-042
- Status last verified
- May 2021
- First submitted
- 16 Apr 2009
- Last updated
- 8 Jun 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Giugliani R, Harmatz P, Jones SA, Mendelsohn NJ, Vellodi A, Qiu Y, Hendriksz CJ, Vijayaraghavan S, Whiteman DA, Pano A. Evaluation of impact of anti-idursulfase antibodies during long-term idursulfase enzyme replacement therapy in mucopolysaccharidosis II patients. Mol Genet Metab Rep. 2017 Feb 21;12:2-7. doi: 10.1016/j.ymgmr.2017.01.014. eCollection 2017 Sep.
PubMed 28243577
Study at a glance
From the registry record, last updated by the sponsor on 8 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-Center Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients Enrolled in the Hunter Outcome Survey (HOS) Receiving Idursulfase Enzyme Replacement Therapy
- ClinicalTrials.gov ID
- NCT00882921
- Acronym / sponsor ID
- HGT-ELA-042
- Conditions
- Hunter Syndrome
- Keywords
- MPS 2, MPSII, hunter's disease treatment, enzyme replacement therapy, hunter's disease, iduronate 2 sulfatase, mps diagnosis, mps symptoms
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 26 participants (actual)
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- Study start
- 14 Oct 2008 (actual)
- Primary completion
- 8 Feb 2013 (actual)
- Study completion
- 8 Feb 2013 (actual)
- First posted
- 17 Apr 2009 (estimated)
- Results
- Posted 30 Jul 2014
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 evaluate the effect of anti-idursulfase antibodies on idursulfase safety (measured by infusion related adverse events) between patients who develop anti-idursulfase antibodies and patients who do not after long-term idursulfase enzyme replacement therapy (ERT).
Detailed description
This study is being conducted to satisfy post-marketing commitments to monitor anti-idursulfase antibody development in Hunter syndrome patients after long-term idursulfase enzyme replacement therapy. The study will be conducted as a sub-study within the Hunter Outcome Survey (HOS). Hunter syndrome patients in the HOS who have previously received idursulfase as well as treatment-naive patients who will begin idursulfase treatment within 30 days of study enrollment will be included.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Elaprase | – | Biological: IdursulfaseIdursulfase 0.5 mg/kg Weekly |
Who may qualify
- Ages
- 5 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with Hunter syndrome
Key inclusion criteria (5 listed)
- Patients must meet all of the following criteria to be considered eligible for enrollment:
- The patient is male and enrolled in the HOS (i.e., meets the entry criteria of a documented diagnosis of Hunter syndrome)
- The patient is ≥ 5 years-old
- The patient is on idursulfase treatment or scheduled to begin idursulfase treatment within 30 days of study enrollment
- The patient, patient's parent(s), or patient's legally authorized guardian must have voluntarily signed an Institutional Review Board (IRB)/Independent Ethics Committee (IEC)-approved informed consent form after all relevant aspects of the study have been explained and discussed with the patient, patient's parent(s), or patient's legally authorized guardian.
Key exclusion criteria (4 listed)
- The patient has received biologic/ERT products other than idursulfase, or other investigational product(s) for any reason within 30 days prior to study entry.
- The patient has a life expectancy of < 2 years
- The patient is unable to comply with the protocol, e.g., has a clinically relevant medical condition making implementation of the protocol difficult; has an uncooperative attitude; is unable to return for safety evaluations; or is otherwise unlikely to complete the study, as determined by the Investigator.
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: ElaprasePatients received idursulfase as prescribed by their physician following locally approved prescribing information. Patients will not be provided idursulfase by Shire Human Genetic Therapies, Inc. or the HOS. | Biological | Elaprase |
Primary outcome
Infusion-Related Adverse Event (IRAE) Rates Between IgG Anti-idursulfase Antibody Positive (Ab+) and Anti-idursulfase IgG Antibody Negative (Ab-) Patients
Time frame: Baseline to 109 Weeks
The primary analysis of how presence of antibodies affected IRAE rates was performed based on a negative binomial regression model. This was done to account for potentially differential follow-up time between antibody groups.
1 secondary outcome
Change From Baseline in uGAG Levels to 109 Weeks
Time frame: Baseline to 109 Weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jun 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 28243577Giugliani R, Harmatz P, Jones SA, Mendelsohn NJ, Vellodi A, Qiu Y, Hendriksz CJ, Vijayaraghavan S, Whiteman DA, Pano A. Evaluation of impact of anti-idursulfase antibodies during long-term idursulfase enzyme replacement therapy in mucopolysaccharidosis II patients. Mol Genet Metab Rep. 2017 Feb 21;12:2-7. doi: 10.1016/j.ymgmr.2017.01.014. eCollection 2017 Sep.
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.
Study Director study director
Takeda
6 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hunter Syndrome, run by Shire at 6 sites in 3 countries.
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 4 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 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United Kingdom, United States, Brazil. 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?
Yes. Results are posted in the registry (first posted 30 Jul 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Infusion-Related Adverse Event (IRAE) Rates Between IgG Anti-idursulfase Antibody Positive (Ab+) and Anti-idursulfase IgG Antibody Negative (Ab-) Patients (time frame: Baseline to 109 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 NCT00882921, 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 8 Jun 2021.