Clinical trial NCT03292887: Hunter Outcome Survey (HOS)
Official title: Hunter Outcome Survey: A Global, Multi-Center, Long-Term, Observational Registry of Patients With Hunter Syndrome (Mucopolysaccharidosis Type II, MPS II)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hunter Syndrome
Keywords: Mucopolysaccharidosis II
Hunter Syndrome
Enzyme Replacement Therapy
Lysosomal
Glycosaminoglycans
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2017-09-26
Enrollment: 1443
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
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").

Brief 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.


Eligibility criteria: Inclusion Criteria:

1. Diagnosis of Hunter syndrome (biochemically and/or genetically)
2. Signed and dated written informed consent, as per either a or b below:

   1. 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
   2. Historical Participants: Signed and dated informed consent from the participant's LAR (where allowed by relevant individual country or site regulations/laws). .

Exclusion Criteria:

1. 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 Participants may re-enroll once they have completed or withdrawn from the other clinical study.
2. 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.

Primary outcomes:
measure: Number of Participants With Infusion-related Reactions (IRRs)
timeFrame: Baseline to year 17
measure: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: Baseline to year 17
measure: Number of Participants With Positive Antibody Response
timeFrame: Baseline to year 17
measure: Change in Urinary Glycosaminoglycan (GAG) Levels
timeFrame: Baseline to year 17
measure: Change in Height
timeFrame: Baseline to year 17
measure: Change in Weight
timeFrame: Baseline to year 17
measure: Change in Head Circumference and Corresponding Calculated Z-scores
timeFrame: Baseline to year 17
measure: Change in Distance Walked in the 6-minute Walk Test
timeFrame: Baseline to year 17
measure: Left Ventricular Mass Index (LVMI)
timeFrame: Baseline to year 17
measure: Change in Forced Expiratory Volume in 1 Second (FEV1)
timeFrame: Baseline to year 17
measure: Change in Forced Vital Capacity (FVC)
timeFrame: Baseline to year 17
measure: Change in Liver and Spleen Size
timeFrame: Baseline to year 17
measure: Prevalence of Cardiac and Pulmonary-related Hospitalizations
timeFrame: Baseline to year 17
measure: Age at the Time of Death
timeFrame: Baseline to year 17
measure: Cause of Death
timeFrame: Baseline to year 17
Secondary outcomes:
measure: Natural History of Untreated Participants With Hunter Syndrome
timeFrame: Baseline to year 17
measure: Dosing Regimens of Elaprase for Prescribed Dose in Participants With Hunter Syndrome
timeFrame: Baseline to year 17
measure: Dosing Regimens of Elaprase for Administered Dose in Participants With Hunter Syndrome
timeFrame: Baseline to year 17
measure: Dosing Regimens of Elaprase for Total Infusion Time in Participants With Hunter Syndrome
timeFrame: Baseline to year 17
measure: Dosing Regimens of Elaprase for Missed Infusions in Participants With Hunter Syndrome
timeFrame: Baseline to year 17
measure: Dosing Regimens of Elaprase for Reason for Missed Infusions.
timeFrame: Baseline to year 17
measure: Assessment of Hunter Syndrome on Health-related Quality of Life (HRQL) Using Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS)
timeFrame: Baseline to year 17
Other outcomes:
Locations:
city: Lexington
country: United States
facility: Shire
state: Massachusetts
Study officials:
affiliation: Shire
name: Study Director
role: STUDY_DIRECTOR
References: