NCT01927861已完成3 期
A 52-week, Multi-centre, Randomised, Double-blind, Parallel-group, no Treatment Controlled (Open-label) Trial Investigating the Efficacy and Safety of Two Doses of NN-220 in Short Stature With Noonan Syndrome
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 51
- 试验地点
- 1
- 主要终点
- Change in Height SDS (Japanese National Reference Data)
研究概览
简要总结
This trial is conducted in Asia. The aim of the trial is to investigate the long-term efficacy and safety of two doses of NN-220 (somatropin) in short stature due to Noonan syndrome.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 3 Years 至 10 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Japanese children with Noonan syndrome clinically diagnosed in one of the following ways:
- •Clinically diagnosed by at least two medical experts using van der Burgt score list,
- •Clinically diagnosed by one medical expert using van der Burgt score list and diagnosed by result of genetic testing for Noonan syndrome,
- •Clinically diagnosed by one medical expert using van der Burgt score list and diagnosed by the same medical expert based on the results of centralised evaluation of facial change using van der Burgt score list
- •Height SDS (standard deviation score): -2 SDS or below (according to the Japanese reference data)
- •Age: boys 3 to below 11 years, girls 3 to below 10 years
- •Height records must be available within the period between 40 and 64 weeks prior to Visit 1 (screening)
- •Prepubertal children (definition for girls breast and pubes of Tanner stage is I, and none of menses, and for boys testicular volume below 4 mL, and pubes and penis of Tanner stage is I)
排除标准
- •Children with known or suspected hypersensitivity against human growth hormone (hGH) or related products (including any components of the trial products)
- •Children with diabetic type diagnosed with the Japanese Diabetes Society Classification
- •Children with history or presence of active malignancy
- •Children who have received GH (growth hormone) treatment
- •Children who have received systemic administration of the following medications within two years prior to Visit 1 (screening): Thyroid hormone (except replacement therapy), antithyroid hormone, androgen, oestrogen, progesterone, anabolic steroid, adrenocortical steroid treatment period for at least 13 weeks), derivative of gonadotropin releasing hormone and somatomedin C (IGF-I)
研究组 & 干预措施
0.033 mg/kg/day
Experimental
干预措施: somatropin (Drug)
0.066 mg/kg/day
Experimental
干预措施: somatropin (Drug)
结局指标
主要结局
Change in Height SDS (Japanese National Reference Data)
时间窗: Baseline, week 104
Height SDS was calculated using the formula: SDS = (height - mean)/SD, where height was the height variable measured, mean and SD of height by sex and age for the reference population. The scores were centered around zero. Positive SDS indicated greater height and negative SDS indicated lesser height than the mean of the reference population. The change from baseline (week 0) in the height SDS after 104 weeks of treatment was analysed using an analysis of covariance (ANCOVA) model with treatment as a fixed effect and baseline height SDS as a covariate. Positive value in change from baseline indicated that SDS was better than baseline SDS. Missing values were imputed using the last observation carried forward (LOCF) method.
次要结局
- Change in Clinical Laboratory Tests (Haematology: Haemoglobin)(Baseline, week 208)
- Change in Clinical Laboratory Tests (Haematology: Basophils)(Baseline, week 208)
- Change in Blood Coagulation Test (Prothrombin Time and APTT)(Baseline, week 208)
- Change in Clinical Laboratory Tests (Haematology: Lymphocytes)(Baseline, week 208)
- Change in Clinical Laboratory Tests (Haematology: Monocytes)(Baseline, week 208)
- Incidence of Treatment Emergent Adverse Events(During 104 weeks of treatment)
- Change in IGF-I (Insulin-like Growth Factor-I)(Baseline, week 104)
- Change in HbA1c (Glycosylated Haemoglobin)(Baseline, week 104)
- Change in Clinical Laboratory Tests (Haematology: Erythrocytes)(Baseline, week 208)
- Change in Clinical Laboratory Tests (Haematology: Leukocytes and Thrombocytes)(Baseline, Week 208)
- Change in Clinical Laboratory Tests (Haematology: Haematocrit)(Baseline, week 208)
- Change in Clinical Laboratory Tests (Lipids: Total Cholesterol, LDL Cholesterol and HDL Cholesterol)(Baseline, week 208)
- Change in Clinical Laboratory Tests (Biochemistry: Total Protein)(Baseline, week 208)
- Change in Bone Age(Baseline, week 208)
- Yearly Change in Bone Age/Change in Chronological Age(Week 156, week 208)
- Height Velocity SDS(Week 156 to week 208)
- Height Velocity(Week 156 to week 208)
- Change in Clinical Laboratory Tests (Haematology: Neutrophils)(Baseline, week 208)
- Change in Clinical Laboratory Tests (Biochemistry: Blood Urea Nitrogen, Sodium, Potassium, Chloride, Total Calcium and Phosphorus)(Baseline, week 208)
- Change in Vital Signs (Diastolic Blood Pressure and Systolic Blood Pressure)(Baseline, week 208)
- Change in IGF-I(Baseline, week 208)
- Change in Clinical Laboratory Tests (Haematology: Eosinophils)(Baseline, week 208)
- Change in Glucose Tolerance (AUC of Insulin) Based on the OGTT(Baseline, week 208)
- Change in Vital Signs (Pulse)(Baseline, week 208)
- Change in ECG(Baseline, week 208)
- Change in Clinical Laboratory Tests (Biochemistry: Creatinine)(Baseline, week 208)
- Change in Clinical Laboratory Tests (Biochemistry: AST, ALT, r-GTP and Alkaline Phosphatase)(Baseline, week 208)
- Change in Bone Age/Chronological Age(Baseline, week 208)
- Change in Urinalysis (Protein, Glucose and Occult Blood)(Baseline, week 208)
- Change in Height SDS (Japanese National Reference Data)(Baseline, week 208)
- Change in Height SDS (Noonan Syndrome Reference Data in Japanese)(Baseline, week 208)
- Incidence of Treatment Emergent AEs(Week 0 to week 234 (208 weeks treatment period + 26 weeks extended treatment period) + 7 days (follow-up period))
- Change in Glucose Tolerance (AUC of Glucose) Based on the OGTT(Baseline, week 208)
- Change in HbA1c(Baseline, week 208)
研究者
研究点 (1)
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