Hunter Outcome Survey: A Global, Multi-Center, Long-Term, Observational Registry of Patients With Hunter Syndrome (Mucopolysaccharidosis Type II, MPS II)
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- Shire
- 入组人数
- 1,443
- 试验地点
- 1
- 主要终点
- Number of Participants With Infusion-related Reactions (IRRs)
研究概览
简要总结
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
- •Historical Participants: Signed and dated informed consent from the participant's LAR (where allowed by relevant individual country or site regulations/laws). .
排除标准
- •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 Participants may re-enroll once they have completed or withdrawn from the other clinical study.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.
结局指标
主要结局
Number of Participants With Infusion-related Reactions (IRRs)
时间窗: 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.
Change in Urinary Glycosaminoglycan (GAG) Levels
时间窗: Baseline to year 17
Change in urinary GAG levels from the start of ERT is reported.
Change in Height
时间窗: Baseline to year 17
Change in height from the start of ERT will be reported.
Prevalence of Cardiac and Pulmonary-related Hospitalizations
时间窗: Baseline to year 17
Prevalence of cardiac and pulmonary-related hospitalizations will be reported.
Left Ventricular Mass Index (LVMI)
时间窗: Baseline to year 17
Change in LVMI will be assessed as calculated by echocardiography.
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
时间窗: 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.
Change in Head Circumference and Corresponding Calculated Z-scores
时间窗: 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
时间窗: Baseline to year 17
Change in distance walked in 6-minute walk test from the start of ERT is reported.
Number of Participants With Positive Antibody Response
时间窗: 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 Liver and Spleen Size
时间窗: Baseline to year 17
Change in liver and spleen size as estimated by palpation will be reported.
Change in Weight
时间窗: Baseline to year 17
Change in weight from the start of ERT will be reported.
Change in Forced Expiratory Volume in 1 Second (FEV1)
时间窗: 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)
时间窗: Baseline to year 17
Change in pulmonary function from the start of ERT will be reported as measured by forced vital capacity (FVC).
Age at the Time of Death
时间窗: Baseline to year 17
Age at the time of death will be reported.
Cause of Death
时间窗: Baseline to year 17
Causes of death will be reported
次要结局
- 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)
- 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)
- 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)