A Long-Term, Open-Label, Multicenter, Phase IV Study to Assess Longitudinal Changes on Height and Weight in Patients With MPS II Who Are Receiving Elaprase and Started Treatment With Elaprase at <6 Years of Age
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- Shire
- 入组人数
- 21
- 试验地点
- 8
- 主要终点
- Change in height
研究概览
简要总结
This long-term study will provide Elaprase treatment to children enrolled in this study and will utilize data from both enrolled patients and Hunter Outcome Survey (HOS) patient registry data to conduct the primary growth analysis to assess changes in height and weight in patients with Mucopolysaccharidosis II (Hunter syndrome) MPS II.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 5 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Group 1: Prospective Patient Group
- •The patient is male.
- •The patient is Elaprase-naïve at study entry.
- •The patient must have a documented diagnosis of MPS II. Of the 3 criteria below, the combinations (3a AND 3b) or (3a AND 3c) will be accepted as diagnostic of MPS II:
- •The patient has a deficiency in I2S enzyme activity of ≤10% of the lower limit of the normal range as measured in plasma, fibroblasts, or leukocytes (based on the reference laboratory's normal range). AND
- •The patient has a documented mutation in the I2S gene. OR
- •The patient has a normal enzyme activity level of one other sulfatase as measured in plasma, fibroblasts, or leukocytes (based on the normal range of measuring laboratory).
- •The patient will be <6 years of age at the start of Elaprase treatment.
- •The patient, patient's parent(s) or legally authorized guardian(s) 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. Consent of the patient's parent(s) or legally authorized guardian(s) and the patient's assent, as relevant, must be obtained.
- •Group 2: Retrospective Data Inclusion Criteria:
- •Retrospective Patient Group patients will be enrolled in HOS and not Study SHP-ELA-401; however, their growth data may be included in the analysis for Study SHP-ELA-401 if the following data inclusion criteria are met.
- •The patient is male.
- •The patient is enrolled in HOS.
- •The patient was <6 years of age at the start of Elaprase treatment.
- •The patient received Elaprase weekly treatment for at least 5 years.
- •The patient had a height assessment and a weight assessment documented within 3 months before or after Elaprase treatment start.
- •The patient has had annual height and weight assessments from start of Elaprase through age 10 years.
- •The patient, patient's parent(s), or legally authorized guardian(s) agree(s) to data collection.
- •The patient, patient's parent(s), or legally authorized guardian(s) must have signed an IRB/IEC-approved informed consent form after all relevant aspects of the HOS study have been explained and discussed. Consent of the patient's parent(s) or legally authorized guardian(s) and the patient's assent, as relevant, must be obtained.
排除标准
- •Group 1: Prospective Patient Group
- •The patient has received treatment with any investigational drug or device within the 30 days prior to study entry.
- •The patient has received or is receiving treatment with idursulfase-IT.
- •The patient has received growth hormones, a cord blood infusion, or a bone marrow transplant at any time.
- •The patient has received blood product transfusions within 90 days prior to Screening.
- •The patient is unable to comply with the protocol as determined by the Investigator.
- •Group 2: Retrospective Data Exclusion Criteria:
- •HOS patients that meet the following criteria are not eligible to be included into the Study SHP-ELA-401 Primary Growth Analysis:
- •Patient was treated with growth hormone or other medications or interventions intended to promote growth in the time period covered by the analysis.
研究组 & 干预措施
Enrolled Patients
Patients who are receiving treatment with Elaprase in this study (SHP-ELA-401), who are <6 years of age, and were previously treatment-naïve. Patients who are not enrolled in this study (SHP-ELA-401) but are enrolled in the Hunter Outcome Survey (HOS) patient registry and were < 6 years of age at start of Elaprase treatment. While not enrolled in the present Study SHP-ELA-401, their height and weight data from HOS will be utilized in the Primary Growth Analysis for this study.
干预措施: Elaprase for intravenous (IV) infusion (Drug)
结局指标
主要结局
Change in height
时间窗: Screening to End-of-Study (5-10 years)
Change in weight Z-scores
时间窗: Screening to End-of-Study (5-10 years)
Number of participants with adverse events as measured by type, severity, and relationship to treatment
时间窗: Screening to End-of-Study (5-10 years)
Change in neurological examination to evaluate long-term safety in patient
时间窗: Screening to End-of-Study (5-10 years)
Change in clinical laboratory testings as measured by serum chemistry to evaluate long-term safety in patients
时间窗: Screening to End-of-Study (5-10 years)
Change in clinical laboratory testings as measured by urinalysis to evaluate long-term safety in patients
时间窗: Screening to End-of-Study (5-10 years)
Change in clinical laboratory testings as measured by hematology to evaluate long-term safety in patients
时间窗: Screening to End-of-Study (5-10 years)
次要结局
- Quality of Life, as measured by the Hunter-Syndrome Functional Outcome in Clinical Understanding Scale(Screening to End-of-Study (5-10 years))
- Change in anti-idursulfase antibodies in serum(Screening to End-of-Study (5-10 years))
- Distance walked, as measured by the Six Minute Walk Test (6MWT)(Screening to End-of-Study (5-10 years))
- Urinary glycosaminoglycans (uGAG) levels normalized to urine creatinine(Screening to End-of-Study (5-10 years))
- Impact of illness on ability to function in daily life, as measured by the Childhood Health Assessment Questionnaire (CHAQ Parent Report)(Screening to End-of-Study (5-10 years))
- Normalized uGAG divided by upper limit of normal for age (uGAG/ULN) every 12 months(Baseline to End-of-Study (5-10 years))
- Joint mobility, as measured by Joint Range of Motion (JROM) scores, including global, upper-limb, and lower-limb joint scores(Screening to End-of-Study (5-10 years))
- Adaptive behavior, as measured by the Vineland Adaptive Behavior Scales (VABS II)(Screening to End-of-Study (5-10 years))