study details
start date: August 1, 2005
estimated completion: September 1, 2022
last updated: June 18, 2021
size / enrollment: 2000
primary outcomes:
- 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.
- 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.
- 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.
- Baseline to year 17
Change in Urinary Glycosaminoglycan (GAG) Levels
Change in urinary GAG levels from the start of ERT is reported.
- Baseline to year 17
Change in Height
Change in height from the start of ERT will be reported.
- Baseline to year 17
Change in Weight
Change in weight from the start of ERT will be reported.
- 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.
- 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.
- Baseline to year 17
Left Ventricular Mass Index (LVMI)
Change in LVMI will be assessed as calculated by echocardiography.
- 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).
- 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).
- Baseline to year 17
Change in Liver and Spleen Size
Change in liver and spleen size as estimated by palpation will be reported.
- Baseline to year 17
Prevalence of Cardiac and Pulmonary-related Hospitalizations
Prevalence of cardiac and pulmonary-related hospitalizations will be reported.
- Baseline to year 17
Age at the Time of Death
Age at the time of death will be reported.
- Baseline to year 17
Cause of Death
Causes of death will be reported
- Baseline to year 17
secondary outcomes:
- Natural History of Untreated Participants With Hunter Syndrome
Baseline to year 17
- Dosing Regimens of Elaprase for Prescribed Dose in Participants With Hunter Syndrome
Baseline to year 17
- Dosing Regimens of Elaprase for Administered Dose in Participants With Hunter Syndrome
Baseline to year 17
- Dosing Regimens of Elaprase for Total Infusion Time in Participants With Hunter Syndrome
Baseline to year 17
- Dosing Regimens of Elaprase for Missed Infusions in Participants With Hunter Syndrome
Baseline to year 17
- Dosing Regimens of Elaprase for Reason for Missed Infusions.
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)
Baseline to year 17
inclusion criteria:
• Eligible Ages: 0 - 120
• Eligible Sexes: all
Inclusion Criteria:
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). .
exclusion criteria: Criteria:
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.
