NCT05308927Enrolling By Invitation不适用
French Registry of Children Treated With Norditropin® for Short Stature Associated With Noonan Syndrome
适应症
干预措施
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 入组人数
- 221
- 试验地点
- 3
- 主要终点
- Change in height standard deviation score
研究概览
简要总结
This is a non-interventional registry of children treated with Norditropin® for short stature due to Noonan Syndrome (NS). This study aims to provide data on long-term growth evolution and safety of Norditropin® as well as Health Related Quality of Life (HRQoL) data. This registry will include the entirety of children treated with Norditropin® for short stature due to NS over the inclusion period. The decision to initiate treatment with commercially available Norditropin® is made by the patient/parents/Legally Acceptable Representative (LAR) and the treating physician before and independently from the decision to include the patient in this study.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with a clinical and/or genetic diagnosis of NS
- •Patients who are treated with Norditropin® (already treated or initiating) and who are followed in a participating center
- •The decision to initiate treatment with commercially available Norditropin® has been made by the patient/parents/Legally Acceptable Representative (LAR) and the treating physician before and independently from the decision to include the patient in this study
排除标准
- •Patients/Parents/LAR opposed to the collection and processing of their children's medical data
- •Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation
研究组 & 干预措施
Incident patients
Children initiated Norditropin® upon their inclusion in the study but independently from the decision to participate in this study
干预措施: Norditropin (Drug)
Prevalent patients - finished growth upon inclusion
Children were already treated with Norditropin® before their inclusion in the study and finished their growth upon their inclusion. Data collected retrospectively from medical records
干预措施: Norditropin (Drug)
Prevalent patients - not finished growth upon inclusion
Children were already treated with Norditropin® before their inclusion in the study and did not finish their growth upon inclusion. Data collected both retrospectively and prospectively
干预措施: Norditropin (Drug)
结局指标
主要结局
Change in height standard deviation score
时间窗: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Measured in standard deviation scores (SDS) ranging from -10 to +10
次要结局
- Change in diastolic blood pressure(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Type and number of concomitant treatments(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Number of adverse drug reactions (ADR)(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Number of serious adverse events (SAE)(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Change in Haemoglobin A1c (HbA1c) level(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Change in fasting total cholesterol(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Change in fasting triglycerides(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Number of serious adverse drug reactions (SADR)(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Number of adverse events (AE)(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Education(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- School level(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Educational measures(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Tanner stage(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Change in pulse rate(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Change in systolic blood pressure(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Change in echocardiogram parameters (cm/s)(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Change in fasting low-density lipoproteins (LDL) cholesterol(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Change in Follicle-Stimulating Hormone (FSH) (female from 8 years old and male from 9 years old)(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Frequency of injections of Norditropin® treatment per week(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Reasons for treatment modifications(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Type and number of concomitant treatments linked to an adverse event(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Change in body mass index (BMI)(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Change in CBC (G/L)(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Change in Insulin-like growth factor 1 (IGF-1) level (ng/ml)(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Change in IGF-1 level (SDS)(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Change in height velocity (SDS/year)(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Change in corrected QT-interval (Electrocardiogram)(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Change in echocardiogram parameters (mm)(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Change in echocardiogram parameters (%)(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Change in complete blood count (CBC) (g/dL)(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Change in fasting insulin level(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Change in fasting blood glucose level(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Change in fasting high-density lipoproteins (HDL) cholesterol(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Change in Luteinizing Hormone (LH) (female from 8 years old and male from 9 years old)(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Change in oestradiol (female from 8 years old and male from 9 years old)(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Change in testosterone (female from 8 years old and male from 9 years old)(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Change in weight(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Change in fasting total cholesterol/HDL ratio(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Total duration of Norditropin® treatment(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Total cumulative dose of Norditropin® treatment(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Change in bone age(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Change in height velocity (cm/year)(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Change in inhibin B(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Change in Anti-Müllerian Hormone (AMH)(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Change in dose of Norditropin® treatment(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Number of modifications (change in dosage/ temporary stop)(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Duration of temporary stop(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Type and number of NS comorbidities(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
- Change in Health-Related Quality of Life (HRQoL) score (PedsQL)(From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years))
研究者
研究点 (3)
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