NCT04970654已完成3 期
A Trial Comparing the Efficacy and Safety of Once Weekly Dosing of Somapacitan With Daily Norditropin® in Chinese Children With Growth Hormone Deficiency
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
- 110
- 试验地点
- 57
- 主要终点
- Height Velocity
研究概览
简要总结
The study compares two medicines for children with a low level of hormone to grow: somapacitan (a new medicine) given once a week and Norditropin® (a medicine doctors can already prescribe) given once a day. Researchers will test somapacitan to see how well it works, compared to the standard treatment with Norditropin®. The participants will either get Norditropin® once every day or somapacitan once every week - which treatment the participant gets is decided by chance. The participant and the study doctor will know which treatment the participant gets. The study includes a 52 week treatment period and a minimum of 30 days follow up period.
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
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Informed consent of parent or legally acceptable representative of participant and child assent, as age-appropriate must be obtained before any trial related activities
- •The parent or legally acceptable representative of the child must sign and date the Informed consent form (according to local requirements)
- •The child must sign and date child assent form or provide oral assent (if required according to local requirements)
- •Prepubertal children: a) Boys: Age more than or equal to 2 years and 26 weeks and less than or equal to 11.0 years at the time of signing informed consent.
- •Testis volume less than 4 ml. b) Girls: Age more than or equal to 2 years and 26 weeks and less than or equal to 10.0 years at the time of signing informed consent. Tanner stage 1 for breast development (no palpable glandular breast tissue)
- •Confirmed diagnosis of growth hormone deficiency determined by two different growth hormone stimulation tests performed within 12 months prior to randomisation, defined as a peak growth hormone level of less than or equal to 10.0 ng/ml using the WHO International Somatropin 98/574 standard
- •If only one growth hormone stimulation test is available before screening, then confirmation of growth hormone deficiency by second and different growth hormone stimulation test must be done
- •For children with at least 2 additional pituitary hormone deficiencies (other than growth hormone deficiency) only one growth hormone stimulation test is needed
- •Impaired height defined as at least 2.0 standard deviations below the mean height for chronological age and gender according to Chinese general population standards at screening
- •Impaired height velocity defined as annualised height velocity at screening less than 7cm/year for subjects between 2.5 and 3 years old and less than 5 cm/year for subjects from 3 years and above calculated over a time span of minimum 3 months and maximum 18 months prior to screening according to Chinese guideline and expert consensus on children with short stature and GH therapy
- •No prior exposure to growth hormone therapy or IGF-I treatment
- •Bone age less than chronological age at screening
- •Body Mass Index more than 5th and less than 95th percentile, Body Mass Index-for-age growth charts according to Chinese general population standards.
- •IGF-I < -1.0 SDS at screening, compared to age and gender normalized range measured at central laboratory
- •No intracranial tumour confirmed by magnetic resonance imaging or computer tomography scan. An image or scan taken within 9 months prior to screening can be used as screening data if the medical evaluation and conclusion is available
排除标准
- •Known or suspected hypersensitivity to trial product(s) or related products.
- •Previous participation in this trial. Participation is defined as randomisation.
- •Receipt of any investigational medicinal product within 3 months before screening or participation in another clinical trial before randomisation
- •Any known or suspected clinically significant abnormality likely to affect growth or the ability to evaluate growth with standing height measurements:
- •Turner Syndrome (including mosaicisms)
- •Chromosomal aneuploidy and significant gene mutations causing medical "syndromes" with short stature, including but not limited to Laron syndrome, Noonan syndrome, Prader-Willi Syndrome, abnormal SHOX-1 gene analysis or absence of GH receptors
- •Significant spinal abnormalities including but not limited to scoliosis, kyphosis and spina bifida variants
- •Congenital abnormalities (causing skeletal abnormalities), including but not limited to Russell-Silver Syndrome or skeletal dysplasias
- •Family history of skeletal dysplasia
- •Children born small for gestational age (birth weight 10th percentile of the recommended gender-specific birth weight for gestational age according to national standards in China5
- •Children diagnosed with diabetes mellitus or screening values from central laboratory of
- •fasting plasma glucose more than or equal to 126 mg/dl (7.0 mmol/L) or
- •HbA1c more than or equal to 6.5 %
- •Current inflammatory diseases requiring systemic corticosteroid treatment for longer than 2 consecutive weeks within the last 3 months prior to screening
- •Children requiring inhaled glucocorticoid therapy at a dose 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)
- •Diagnosis of attention deficit hyperactivity disorder
- •Prior history or presence of malignancy including intracranial tumours
- •Prior history or known presence of active Hepatitis B or Hepatitis C (exceptions to this exclusion criterion is the presence of antibodies due to vaccination against Hepatitis B)
- •Any clinically significant abnormal laboratory screening tests, as judged by the study doctor
- •Any disorder which, in the opinion of the study doctor, might jeopardise Participant's safety or compliance with the protocol
- •The participant or the parent/legally acceptable representative is likely to be non-compliant in respect to trial conduct, as judged by the study doctor
- •Children with hypothyroidism and/or adrenal insufficiency not on adequate and stable replacement therapy for at least 90 days prior to randomisation.
研究组 & 干预措施
Somapacitan weekly
Experimental
participants will receive once-weekly somapacitan for 52 weeks
干预措施: somapacitan (Drug)
Norditropin® daily
Active Comparator
Participants will receive Norditropin® daily for 52 weeks
干预措施: Norditropin® (Drug)
结局指标
主要结局
Height Velocity
时间窗: Baseline (Week 0); Week 52
Height velocity (HV) was derived from height measurements taken at baseline (week 0) and the week 52 visit as: HV = (height at 52 weeks visit - height at baseline)/(time from baseline to 52 weeks visit in years).
次要结局
- Change in Bone Age(Week -2, week 52)
- Change in Height Standard Deviation Score(Baseline (week 0), week 52)
- Change in Height Velocity Standard Deviation Score(Baseline (week 0), week 52)
- Change in Fasting Plasma Glucose(Baseline (week 0), week 52)
- Change in HbA1c(Baseline (week 0), week 52)
- Change in IGF-I Standard Deviation Score(Baseline (week 0), week 52)
- Change in IGFBP-3 Standard Deviation Score(Baseline (week 0), week 52)
研究者
研究点 (57)
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