CTRI/2020/08/027258进行中（未招募）2 期
A Dose-finding Trial Evaluating the Effect and Safety of Once-weekly Treatment of Somapacitan Compared to Daily Norditropin® in Children With Short Stature Born Small for Gestational Age With no Catch-up Growth by 2 Years of Age or Older
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 60
- 试验地点
- 3
- 主要终点
- Height velocity
研究概览
简要总结
The study compares 2 medicines used for the treatment of children who are born small and who stayed small: somapacitan given once a week (a new medicine) and Norditropin® given once a day (the medicine doctors can already prescribe).
Participants will either get somapacitan or Norditropin® - which treatment is decided by chance. Both participants and the study doctor will know which treatment the participants get. The study will last for 2 years. Participants will take either an injection once every week or once every day. Participants will have 9 clinic visits and will be in the study for 1 year. The follow-up period is at least 30 days.
研究设计
- 研究类型
- Interventional
- 分配方式
- Computer generated randomization
- 盲法
- Open Label
入排标准
- 年龄范围
- 2.00 Year(s) 至 11.00 Year(s)（—）
- 性别
- All
入选标准
- •Subjects are eligible to be included in the trial only if all of the following criteria apply:
- •Informed consent of parent or legally acceptable representative of subject and child assent, as age-appropriate must be obtained before any trial-related activities.
- •a) The parent or legally acceptable representative of the child must sign and date the Informed Consent Form (according to local requirements).
- •b) The child must sign and date the Child Assent Form or provide oral assent (if required according to local requirements).
- •Pre-pubertal children: a) Boys: o Age ≥ 2 years and 26 weeks and < 11.0 years at screening.
- •o Testes volume < 4 ml.
- •b) Girls: o Age ≥ 2 years and 26 weeks and < 10.0 years at screening.
- •o Tanner stage 1 for breast development (no palpable glandular breast tissue).
- •Born small for gestational age (birth length and/or weight < -2 SDS) (according to national standards).
- •For Israel and Japan: see Appendix 9
- •Impaired height defined as at least 2.5 standard deviations below the mean height for chronological age and gender at screening according to the standards of Centers for Disease Control and Prevention.
- •For India: see Appendix 9
- •Impaired height velocity defined as annualised height velocity below the 50th percentile for chronological age and gender according to the standards of Prader calculated over a time span of minimum 6 months and maximum 18 months prior to screening.
- •No prior exposure to growth hormone therapy or IGF-I treatment.
- •Gestational age at birth ≥ 32 weeks.
- •Body Mass Index <95th percentile according to Centers for Disease Control and Prevention, Body Mass Index-for-age growth charts.
- •For India: see Appendix 9.
排除标准
- •Subjects are excluded from the trial if any of the following criteria apply:
- •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 at time of randomisation.
- •Any known or suspected clinically significant abnormality likely to affect growth or the ability to evaluate growth with standing height measurements: a) Turner Syndrome (including mosaicisms) b) 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 c) Significant spinal abnormalities including but not limited to scoliosis, kyphosis and spina bifida variants d) Congenital abnormalities (causing skeletal abnormalities), including but not limited to Russell-Silver Syndrome or skeletal dysplasias e) Family history of skeletal dysplasia For India: see Appendix 9
- •Children with hormonal deficiencies including suspected or confirmed growth hormone deficiency according to local practise.
- •Children diagnosed with diabetes mellitus or screening values from central laboratory of a) Fasting plasma glucose ≥126 mg/dl (7.0 mmol/L) or b) HbA1c ≥ 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 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).
- •Diagnosis of attention deficit hyperactivity disorder.
- •Prior history or known 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 disorder which, in the opinion of the investigator, might jeopardise subject’s safety or compliance with the protocol.
- •The subject or the parent/legally acceptable representative is likely to be non-compliant in respect to trial conduct, as judged by the investigator.
- •Children who are small due to malnutrition defined as -2 SD according to standards: 0-5 years: weight for height on World Health Organisation Multicentre Growth Reference Study 2006 and >5 years: World Health Organisation 2007 Body Mass Index.
- •For India: see Appendix 9.
结局指标
主要结局
Height velocity
时间窗: Time Frame: From baseline (week 0) to week 26 | Unit : cm/year
次要结局
- Change in height standard deviation score (SDS)
- Change in homeostatic model assessment (HOMA)(From screening (visit 1) to week 26)
- Change in glycated haemoglobin (HbA1c)(From screening (visit 1) to week 26)
- Change in bone age(Time Frame: From baseline (week 0) to week 52)
- Change in height velocity SDS(From baseline (week 0) to week 26)
- Change in fasting plasma glucose(From screening (visit 1) to week 26)
- Change in insulin-like growth factor I (IGF-I) SDS(From screening (visit 1) to week 26)
- Change in insulin-like growth factor binding protein 3 (IGFBP-3) SDS(From screening (visit 1) to week 26)
研究者
研究点 (3)
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