Hunter SyndromeHOSNCT03292887

Hunter Outcome Survey (HOS)

An observational study of Hunter Syndrome, run by Shire at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Trial summary

The purpose of this study is to collect data that will increase understanding of Hunter syndrome. The data from HOS may provide guidance to healthcare professionals about disease treatment options. Read more about this study

Who is it for?: Any age
Conditions the study is for: Hunter Syndrome
How many people: 1,443 participants
Where: 1 siteUnited States
Study dates: 3 Oct 2005to 16 Feb 2023
How long will I take part?: 20 Years
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: The HOS registry is open to all participants (both alive and deceased) with Hunter syndrome who are untreated or who are receiving/received treatment with Elaprase, including participants who are alive at HOS enrollment (referred to as "Prospective Patients") and participants who are deceased at HOS enrollment (referred to as "Historical Patients").

Who may be able to join

Diagnosis of Hunter syndrome (biochemically and/or genetically)
Signed and dated written informed consent, as per either a or b below:
Prospective Participants: Signed and dated written informed consent from the participant or, for participants aged less than (<) 18 years (<16 years in Scotland), parent and/or participant's legally authorized representative (LAR), and assent of the minor where applicable.
informed consent must be obtained from LARs for cognitively impaired participants, where applicable.
OR
Historical Participants: Signed and dated informed consent from the participant's LAR (where allowed by relevant individual country or site regulations/laws). .

Who cannot join

Participants enrolled in an interventional clinical trial are not eligible. Participants may re-enroll once they have completed or withdrawn from the other clinical study.
Participants receiving treatment for Hunter syndrome with an ERT product other than Elaprase are not eligible. Participants may enroll or re-enroll once they have stopped treatment with another ERT.

Trial locations and contacts

1 site in 1 country.

United States1 site

Shire
Lexington, Massachusetts, 02421

Other trials in United States

About this study

Official title: Hunter Outcome Survey: A Global, Multi-Center, Long-Term, Observational Registry of Patients With Hunter Syndrome (Mucopolysaccharidosis Type II, MPS II)

The purpose of this study is to collect data that will increase understanding of Hunter syndrome. The data from HOS may provide guidance to healthcare professionals about disease treatment options.

Conditions: Hunter Syndrome
Keywords: Mucopolysaccharidosis II, Hunter Syndrome, Enzyme Replacement Therapy, Lysosomal, Glycosaminoglycans
Medical subject headings: Mucopolysaccharidosis II

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Study groups in NCT03292887
Study group	Role	What they receive
Elaprase Treated		Participants with Hunters Syndrome received or receiving treatment with Elaprase as prescribed by their physician following locally approved prescribing information.
Elaprase Non-Treated		Participants received no treatment for Hunters Syndrome.

What is being measured

The primary outcome is the main question the study is designed to answer.

Number of Participants With Infusion-related Reactions (IRRs)
An Infusion-related reaction (IRR) is an adverse event (AE) that occurs during or within 24 hours of an infusion and with evidence of a causal relationship with Elaprase.
Measured: Baseline to year 17
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
An AE is any noxious, pathologic, or unintended change in anatomical, physiologic, or metabolic function as indicated by physical signs, symptoms, or laboratory changes occurring in the registry, whether or not considered product-related. This includes an exacerbation of a pre-existing condition. An AE or adverse drug reaction (ADR) that meets one or more of the following criteria/outcomes is classified as serious whether considered to be related to the pharmaceutical product or not: death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalizations, a persistent or significant disability or incapacity, a congenital anomaly or birth defect and important medical events.
Measured: Baseline to year 17
Number of Participants With Positive Antibody Response
Immunogenicity is determined by time to first positive antibody response (antibody level and isotype), antibody titer, isotype, and neutralizing antibodies.
Measured: Baseline to year 17
Change in Urinary Glycosaminoglycan (GAG) Levels
Change in urinary GAG levels from the start of ERT is reported.
Measured: Baseline to year 17
Change in Height
Change in height from the start of ERT will be reported.
Measured: Baseline to year 17
Change in Weight
Change in weight from the start of ERT will be reported.
Measured: Baseline to year 17
Change in Head Circumference and Corresponding Calculated Z-scores
Change in head circumference with the corresponding Z-scores from the start of ERT will be reported.
Measured: Baseline to year 17
Change in Distance Walked in the 6-minute Walk Test
Change in distance walked in 6-minute walk test from the start of ERT is reported.
Measured: Baseline to year 17
Left Ventricular Mass Index (LVMI)
Change in LVMI will be assessed as calculated by echocardiography.
Measured: Baseline to year 17
Change in Forced Expiratory Volume in 1 Second (FEV1)
Change in pulmonary function from the start of ERT will be reported as measured by forced expiratory volume in 1 second (FEV1).
Measured: Baseline to year 17
Change in Forced Vital Capacity (FVC)
Change in pulmonary function from the start of ERT will be reported as measured by forced vital capacity (FVC).
Measured: Baseline to year 17
Change in Liver and Spleen Size
Change in liver and spleen size as estimated by palpation will be reported.
Measured: Baseline to year 17
Prevalence of Cardiac and Pulmonary-related Hospitalizations
Prevalence of cardiac and pulmonary-related hospitalizations will be reported.
Measured: Baseline to year 17
Age at the Time of Death
Age at the time of death will be reported.
Measured: Baseline to year 17
Cause of Death
Causes of death will be reported
Measured: Baseline to year 17

Secondary outcomes (7)

Natural History of Untreated Participants With Hunter Syndrome
Evaluation of signs and symptoms for the natural history of disease: hepatosplenomegaly, central nervous system involvement, skeletal involvement, ear, nose, and throat signs and symptoms, pulmonary signs and symptoms and cardiac signs and symptoms will be reported.
Measured: Baseline to year 17
Dosing Regimens of Elaprase for Prescribed Dose in Participants With Hunter Syndrome
Dosing regiments of Elaprase will be evaluated for prescribed dose.
Measured: Baseline to year 17
Dosing Regimens of Elaprase for Administered Dose in Participants With Hunter Syndrome
Dosing regiments of Elaprase will be evaluated for administered dose.
Measured: Baseline to year 17
Dosing Regimens of Elaprase for Total Infusion Time in Participants With Hunter Syndrome
Dosing regiments of Elaprase will be evaluated for total infusion time.
Measured: Baseline to year 17
Dosing Regimens of Elaprase for Missed Infusions in Participants With Hunter Syndrome
Dosing regiments of Elaprase will be evaluated for missed infusions.
Measured: Baseline to year 17
Dosing Regimens of Elaprase for Reason for Missed Infusions.
Dosing regiments of Elaprase will be evaluated for reason for missed infusions.
Measured: Baseline to year 17
Assessment of Hunter Syndrome on Health-related Quality of Life (HRQL) Using Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS)
HS-FOCUS was developed as disease-specific measure of the impact of Hunter syndrome on HRQL. The HS-FOCUS is designed to gather information on the participant's daily life and wellbeing, satisfaction with treatment, and hospitalizations, as well as on how Hunter syndrome impacts participant's general quality of life. HS-FOCUS includes 2 validated components: a parent version and a patient self-reported version for those over age 12 years. The HS-FOCUS Version 2.0 contains 6 functional status domains: Walking/Standing, Reach/Grip, Sleeping, Schooling/Work, Activities, and Breathing. Items are scored using a response scale from 0 to 4, with ="0" expressing being able to complete the activity-related functions "without any difficulty" and "4" as "unable to do so. Scores are averaged to calculate the 6 function domain scores and the Overall Function Score, with higher scores corresponding to a higher degree of incapacity.
Measured: Baseline to year 17

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

3 Oct 200516 Feb 2023

First posted26 Sep 2017
Study start3 Oct 2005
Primary completion16 Feb 2023
Study completion16 Feb 2023

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 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: Other
Time perspective: Prospective
Enrolment: 1,443 participants (actual)
Target duration: 20 Years

IDs, dates and publications

ClinicalTrials.gov ID: NCT03292887
Sponsor's protocol ID: HOS
Acronym: HOS
Status last verified: Oct 2024
First submitted: 18 Sep 2017
Last updated: 28 Oct 2024
Results posted: No
Data monitoring committee: No
FDA-regulated drug: No
FDA-regulated device: No

Publications

Muenzer J, Amartino H, Giugliani R, Harmatz P, Lin SP, Link B, Molter D, Ramaswami U, Scarpa M, Botha J, Audi J, Burton BK. Unmet needs of adults living with mucopolysaccharidosis II: data from the Hunter Outcome Survey. Orphanet J Rare Dis. 2025 Jul 1;20(1):319. doi: 10.1186/s13023-024-03464-8.
PubMed 40598289

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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
Active, not recruitingPhase 2
An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II
Mucopolysaccharidosis II
RecruitingPhase 1
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Adrenoleukodystrophy, Batten Disease

All Hunter Syndrome trials

Study at a glance

From the registry record, last updated by the sponsor on 28 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Hunter Outcome Survey: A Global, Multi-Center, Long-Term, Observational Registry of Patients With Hunter Syndrome (Mucopolysaccharidosis Type II, MPS II)
ClinicalTrials.gov ID: NCT03292887
Acronym / sponsor ID: HOS / HOS
Conditions: Hunter Syndrome
Keywords: Mucopolysaccharidosis II, Hunter Syndrome, Enzyme Replacement Therapy, Lysosomal, Glycosaminoglycans
Study type: Observational
Recruitment status: Completed
Enrolment: 1,443 participants (actual)
Lead sponsor: ShireIndustry (pharma, biotech, devices)
Study start: 3 Oct 2005 (actual)
Primary completion: 16 Feb 2023 (actual)
Study completion: 16 Feb 2023 (actual)
First posted: 26 Sep 2017 (actual)
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 collect data that will increase understanding of Hunter syndrome. The data from HOS may provide guidance to healthcare professionals about disease treatment options.

Study design

Observational model: Other
Time perspective: Prospective
Target follow-up: 20 Years
Number of arms: 2

Arm	Type	Receives
Elaprase Treated	–	–Participants with Hunters Syndrome received or receiving treatment with Elaprase as prescribed by their physician following locally approved prescribing information.
Elaprase Non-Treated	–	–Participants received no treatment for Hunters Syndrome.

Who may qualify

Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult
Study population: The HOS registry is open to all participants (both alive and deceased) with Hunter syndrome who are untreated or who are receiving/received treatment with Elaprase, including participants who are alive at HOS enrollment (referred to as "Prospective Patients") and participants who are deceased at HOS enrollment (referred to as "Historical Patients").

Key inclusion criteria (6 listed)

Diagnosis of Hunter syndrome (biochemically and/or genetically)
Signed and dated written informed consent, as per either a or b below:
informed consent must be obtained from LARs for cognitively impaired participants, where applicable.
OR

Key exclusion criteria (2 listed)

Participants enrolled in an interventional clinical trial are not eligible. Participants may re-enroll once they have completed or withdrawn from the other clinical study.
Participants receiving treatment for Hunter syndrome with an ERT product other than Elaprase are not eligible. Participants may enroll or re-enroll once they have stopped treatment with another ERT.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Primary outcome

Number of Participants With Infusion-related Reactions (IRRs)
Time frame: Baseline to year 17
An Infusion-related reaction (IRR) is an adverse event (AE) that occurs during or within 24 hours of an infusion and with evidence of a causal relationship with Elaprase.
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Baseline to year 17
An AE is any noxious, pathologic, or unintended change in anatomical, physiologic, or metabolic function as indicated by physical signs, symptoms, or laboratory changes occurring in the registry, whether or not considered product-related. This includes an exacerbation of a pre-existing condition. An AE or adverse drug reaction (ADR) that meets one or more of the following criteria/outcomes is classified as serious whether considered to be related to the pharmaceutical product or not: death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalizations, a persistent or significant disability or incapacity, a congenital anomaly or birth defect and important medical events.
Number of Participants With Positive Antibody Response
Time frame: Baseline to year 17
Immunogenicity is determined by time to first positive antibody response (antibody level and isotype), antibody titer, isotype, and neutralizing antibodies.
Change in Urinary Glycosaminoglycan (GAG) Levels
Time frame: Baseline to year 17
Change in urinary GAG levels from the start of ERT is reported.
Change in Height
Time frame: Baseline to year 17
Change in height from the start of ERT will be reported.
Change in Weight
Time frame: Baseline to year 17
Change in weight from the start of ERT will be reported.
Change in Head Circumference and Corresponding Calculated Z-scores
Time frame: Baseline to year 17
Change in head circumference with the corresponding Z-scores from the start of ERT will be reported.
Change in Distance Walked in the 6-minute Walk Test
Time frame: Baseline to year 17
Change in distance walked in 6-minute walk test from the start of ERT is reported.
Left Ventricular Mass Index (LVMI)
Time frame: Baseline to year 17
Change in LVMI will be assessed as calculated by echocardiography.
Change in Forced Expiratory Volume in 1 Second (FEV1)
Time frame: Baseline to year 17
Change in pulmonary function from the start of ERT will be reported as measured by forced expiratory volume in 1 second (FEV1).
Change in Forced Vital Capacity (FVC)
Time frame: Baseline to year 17
Change in pulmonary function from the start of ERT will be reported as measured by forced vital capacity (FVC).
Change in Liver and Spleen Size
Time frame: Baseline to year 17
Change in liver and spleen size as estimated by palpation will be reported.
Prevalence of Cardiac and Pulmonary-related Hospitalizations
Time frame: Baseline to year 17
Prevalence of cardiac and pulmonary-related hospitalizations will be reported.
Age at the Time of Death
Time frame: Baseline to year 17
Age at the time of death will be reported.
Cause of Death
Time frame: Baseline to year 17
Causes of death will be reported

7 secondary outcomes

Natural History of Untreated Participants With Hunter Syndrome
Time frame: Baseline to year 17
Dosing Regimens of Elaprase for Prescribed Dose in Participants With Hunter Syndrome
Time frame: Baseline to year 17
Dosing Regimens of Elaprase for Administered Dose in Participants With Hunter Syndrome
Time frame: Baseline to year 17
Dosing Regimens of Elaprase for Total Infusion Time in Participants With Hunter Syndrome
Time frame: Baseline to year 17
Dosing Regimens of Elaprase for Missed Infusions in Participants With Hunter Syndrome
Time frame: Baseline to year 17
Dosing Regimens of Elaprase for Reason for Missed Infusions.
Time frame: Baseline to year 17
Assessment of Hunter Syndrome on Health-related Quality of Life (HRQL) Using Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS)
Time frame: Baseline to year 17

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 28 Oct 2024.
Derived from this study: PubMed 40598289Muenzer J, Amartino H, Giugliani R, Harmatz P, Lin SP, Link B, Molter D, Ramaswami U, Scarpa M, Botha J, Audi J, Burton BK. Unmet needs of adults living with mucopolysaccharidosis II: data from the Hunter Outcome Survey. Orphanet J Rare Dis. 2025 Jul 1;20(1):319. doi: 10.1186/s13023-024-03464-8.
To obtain more information on the study, click here/on this linkLink provided by the sponsor.

Related trials for Hunter Syndrome

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
Active, not recruitingPhase 2
An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II
Mucopolysaccharidosis II
RecruitingPhase 1
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Adrenoleukodystrophy, Batten Disease

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

Shire

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 Hunter Syndrome, run by Shire at 1 site in United States.

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 6 inclusion and 2 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 28 Oct 2024. 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 United States. 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?

Number of Participants With Infusion-related Reactions (IRRs) (time frame: Baseline to year 17). The study lists 15 primary outcomes in all. 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 NCT03292887, 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 28 Oct 2024.