Clinical trial NCT04970654: A Research Study in Chinese Children With a Low Level of Hormone to Grow. Treatment is Somapacitan Once a Week Compared to Norditropin® Once a Day.
Official title: A Trial Comparing the Efficacy and Safety of Once Weekly Dosing of Somapacitan With Daily Norditropin® in Chinese Children With Growth Hormone Deficiency
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Growth Hormone Deficiency in Children
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2021-07-21
Enrollment: 110
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

* 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

  1. fasting plasma glucose more than or equal to 126 mg/dl (7.0 mmol/L) or
  2. 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.

Primary outcomes:
measure: Height Velocity
timeFrame: Baseline (Week 0); Week 52
Secondary outcomes:
measure: Change in Bone Age
timeFrame: Week -2, week 52
measure: Change in Height Standard Deviation Score
timeFrame: Baseline (week 0), week 52
measure: Change in Height Velocity Standard Deviation Score
timeFrame: Baseline (week 0), week 52
measure: Change in Fasting Plasma Glucose
timeFrame: Baseline (week 0), week 52
measure: Change in HbA1c
timeFrame: Baseline (week 0), week 52
measure: Change in IGF-I Standard Deviation Score
timeFrame: Baseline (week 0), week 52
measure: Change in IGFBP-3 Standard Deviation Score
timeFrame: Baseline (week 0), week 52
Other outcomes:
Locations:
city: Hefei
country: China
facility: The Second Hospital of Anhui Medical University
state: Anhui
city: Beijing
country: China
facility: Capital Institute of Paediatrics
state: Beijing
city: Beijing
country: China
facility: Beijing Children's Hospital，Capital Medical University
state: Beijing
city: Guangzhou
country: China
facility: The First Affiliated Hospital , Sun Yat-sen University
state: Guangdong
city: Zhengzhou
country: China
facility: Henan Children's Hospital Zhengzhou Children's Hospital
state: Henan
city: Zhengzhou
country: China
facility: Henan Children's Hospital
state: Henan
city: Wuhan
country: China
facility: Tongji Hospital，Tongji Medical College of Huazhong University of Science &Technology
state: Hubei
city: Changsha
country: China
facility: Hunan Children's Hospital
state: Hunan
city: Changsha
country: China
facility: The Second Xiangya Hospital of Central South University
state: Hunan
city: Shaoyang
country: China
facility: The First Affiliated Hospital of Shaoyang University
state: Hunan
city: Suzhou
country: China
facility: Children's Hospital of Soochow University
state: Jiangsu
city: Wuxi
country: China
facility: Wuxi Children's Hospital
state: Jiangsu
city: Jiaxing
country: China
facility: The First Hospital of Jiaxing
state: Jiangxi
city: Nanchang
country: China
facility: Jiangxi Provincial Children's Hospital
state: Jiangxi
city: Pingxiang
country: China
facility: Pingxiang Maternal and Child Health Care Hospital
state: Jiangxi
city: Changchun
country: China
facility: The First Hospital of Jilin University
state: Jilin
city: Jinan
country: China
facility: Shandong Provincial Hospital
state: Shandong
city: Qingdao
country: China
facility: Qingdao Women and Children's Hospital
state: Shandong
city: Qingdao
country: China
facility: Qingdao Women and Children's Hospital
state: Shandong
city: Chengdu
country: China
facility: Chengdu Women's and Children's central hospital
state: Sichuan
city: Hangzhou
country: China
facility: The Children's Hospital, Zhejiang University school of medicine
state: Zhejiang
Study officials:
affiliation: Novo Nordisk A/S
name: Clinical Transparency (dept. 1452)
role: STUDY_DIRECTOR
References: