CTRI/2016/09/007301已完成2 期
A randomised, multinational, active-controlled,(open-labelled), dose finding,double-blinded),parallel group trial investigating efficacy and safety of once-weekly NNC0195-0092 treatment compared to daily growth hormone treatment (Norditropin FlexPro)in growth hormone treatment naive pre-pubertal children with growth hormone deficiency.
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 76
- 试验地点
- 3
- 主要终点
- Height velocity (HV) (cm/year) during the first 26 weeks of treatment, measured as standing height with stadiometer
研究概览
简要总结
This trial is conducted globally. The aim of the trial is to investigate efficacy and safety of once-weekly NNC0195-0092 treatment compared to daily growth hormone treatment (Norditropin® FlexPro®) in growth hormone treatment naïe pre-pubertal children with growth hormone deficiency.
The main trial period will consist of 26 weeks of treatment, followed by a 26 week extension period.
研究设计
- 研究类型
- Interventional
- 分配方式
- Computer generated randomization
- 盲法
- Participant and Investigator Blinded
入排标准
- 年龄范围
- 2.00 Year(s) 至 10.00 Year(s)（—）
- 性别
- All
入选标准
- •For an eligible subject, all inclusion criteria must be answered “yesâ€.
- •Informed consent of parent or legally acceptable representative (LAR) of subject and child assent, as age-appropriate obtained before any trial-related activities.
- •Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial.
- •The parent or LAR of the child must sign and date the Informed Consent Form (according to local requirements).
- •The child must sign and date the Child Assent Form or provide oral assent (if required according to local requirements).
- •Pre-pubertal children o Boys: Tanner stage 1 for pubic hair and testis volume < 4 mL 1, age ≥ 2 years and 26 weeks and ≤ 10.0 years.
- •o Girls: Tanner stage 1 for breast development (no palpable glandular breast tissue) and pubic hair 1, age ≥ 2 years and 26 weeks and ≤ 9.0 years.
- •Confirmed diagnosis of GHD within 12 months prior to screening as determined by two different GH stimulation tests, defined as a peak GH level of ≤ 7.0 ng/ml.
- •For children with three or more pituitary hormone deficiencies only one GH stimulation test is needed.
- •FOR JAPAN ONLY: Confirmed diagnosis of GHD within 12 months prior to screening as determined by one GH stimulation tests for patients with intracranial organic disease or symptomatic hypoglycaemia and two different GH stimulation test for other patients, defined as a peak GH level of ≤ 6 ng/ml by assay using recombinant GH standard.
- •END OF TEXT ONLY APPLICABLE FOR JAPAN.
- •No prior exposure to GH therapy and/or IGF-I treatment.
- •Height of at least 2.0 standard deviations below the mean height for chronological age (CA) and gender according to the standards of Centers for Disease Control and Prevention (CDC) 2-20 years: Girls/Boys stature-for-age and weight-for-age percentiles CDC 2 at screening.
- •Annualized height velocity (HV) at screening below the 25th percentile for CA and gender or below -0.7 SD score for CA and sex, according to the standards of Prader 3 calculated over a time span of minimum 6 months and maximum 18 months prior to screening.
- •Body Mass Index (BMI) percentile >5th and <95th percentile according to Centers for Disease Control and Prevention (CDC)2 BMI-for-age growth charts.
- •IGF-I SDS < -1.0 at screening, compared to age and sex normalized range according to central laboratory measurements.
- •Bone age (X-ray of left hand and wrist, central reviewed according to Greulich & Pyle atlas19) less than chronological age at screening.
- •An X-ray taken within 13 weeks prior to screening can be used as screening data if the image is available and meets requirements for central reading.
排除标准
- •For an eligible subject, all exclusion criteria must be answered "no".
- •Previous participation in this trial. Participation is defined as randomisation.
- •Receipt of any investigational medicinal product within 3 months before screening. FOR BRAZIL ONLY: Participation in other trials within one year (defined as 365 days) prior to screening visit (Visit 1) unless there is a direct benefit to the research subject at the investigator´s discretion. END OF TEXT ONLY APPLICABLE FOR BRAZIL
- •Any clinically significant abnormality likely to affect growth or the ability to evaluate growth with standing measurements: ï‚· Chromosomal aneuploidy and significant gene mutations causing medical “syndromes†with short stature, including but not limited to Turner syndrome, Laron syndrome, Noonan syndrome, or absence of GH receptors. ï‚· Congenital abnormalities (causing skeletal abnormalities), including but not limited to Russell-Silver Syndrome, skeletal dysplasia. ï‚· Significant spinal abnormalities including but not limited to scoliosis, kyphosis and spina bifida variants.
- •Poorly controlled or uncontrolled pituitary insufficiencies or primary hormonal deficiencies of other axes (e.g., thyroid-stimulating hormone/T4, adrenocorticotropic hormone/cortisol, and vasopressin deficiency) in children who have been on stable replacement therapy for less than 6 months for thyroid replacement therapy, and less than 3 months for other hormonal deficiencies prior to screening.
- •Children born small for gestational age (SGA.
- •birth weight and/or birth length < -2 SD for gestational age).
- •Children diagnosed with diabetes mellitus or fasting blood glucose ≥126 mg/dl (7.0 mmol/L), or HbA1c ≥ 6.5% at screening, determined by central laboratory.
- •Current inflammatory diseases (e.g. but not limited to arthritis, inflammatory bowel diseases) requiring systemic corticosteroid treatment for longer than 2 consecutive weeks within the last 3 months prior to screening.
- •Children requiring inhaled glucocorticoid therapy (e.g. asthma) at a dose of greater than 400 μg/day of inhaled budesonide or equivalents for longer than 4 consecutive weeks within the last 12 months prior to screening.
- •Concomitant administration of other treatments that may have an effect on growth, e.g. but not limited to methylphenidate for treatment of attention deficit hyperactivity disorder (ADHD).
- •Prior history or presence of malignancy and/or intracranial tumour.
- •Prior history or presence of active Hepatitis B and/or Hepatitis C (exceptions to this exclusion criterion is the presence of antibodies due to vaccination against Hepatitis B and Hepatitis C).
- •Clinically significant abnormal ECG at screening, as evaluated by investigator.
- •Any clinically significant abnormal laboratory screening tests, as judged by the investigator
- •Any disorder which, in the opinion of the investigator, might jeopardise subject’s safety or compliance with the protocol.
- •The subject and/or the parent/LAR are likely to be non-compliant in respect to trial conduct, as judged by the investigator.
结局指标
主要结局
Height velocity (HV) (cm/year) during the first 26 weeks of treatment, measured as standing height with stadiometer
时间窗: Week 0 to Week 26
次要结局
- Change in height standard deviation score (SDS)(Week 0,Week 26)
- Change in HV (height velocity) SDS(Week 0 to Week 26)
- Incidence of adverse events, including injection site reactions(At Week 26)
- Occurrence of anti-NNC0195-0092 and anti-hGH antibodies(At Week 26)
研究者
研究点 (3)
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