A Research Study in Children Born Small and Who Stayed Small. Treatment is Somapacitan Once a Week Compared to Norditropin® Once a Day
- Conditions
- Health Condition 1: E20-E35- Disorders of other endocrine glands
- Registration Number
- CTRI/2020/08/027258
- Lead Sponsor
- ovo Nordisk AS
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Closed to Recruitment of Participants
- Sex
- Not specified
- Target Recruitment
- 0
Subjects are eligible to be included in the trial only if all of the following criteria apply:
1. 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).
2. 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).
3. Born small for gestational age (birth length and/or weight < -2 SDS) (according to national
standards).
For Israel and Japan: see Appendix 9
4. 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
5. 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.
6. No prior exposure to growth hormone therapy or IGF-I treatment.
7. Gestational age at birth = 32 weeks.
8. 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
Subjects are excluded from the trial if any of the following criteria apply:
1. Known or suspected hypersensitivity to trial product(s) or related products.
2. Previous participation in this trial. Participation is defined as randomisation.
3. Receipt of any investigational medicinal product within 3 months before screening or
participation in another clinical trial at time of randomisation.
4. 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
5. Children with hormonal deficiencies including suspected or confirmed growth hormone
deficiency according to local practise.
6. 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 %
7. Current inflammatory diseases requiring systemic corticosteroid treatment for longer than 2
consecutive weeks within the last 3 months prior to screening.
8. 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.
9. 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).
10. Diagnosis of attention deficit hyperactivity disorder.
11. Prior history or known presence of malignancy including intracranial tumours.
12. 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).
13. Any disorder which, in the opinion of the investigator, might jeopardise subject’s safety or
compliance with the protocol.
For France, Spain, and UK: see Appendix 9
14. The subject or the parent/legally acceptable representative is likely to be non-compliant in
respect to trial conduct, as judged by the investigator.
15. 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
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Height velocityTimepoint: Time Frame: From baseline (week 0) to week 26 <br/ ><br>Unit : cm/year
- Secondary Outcome Measures
Name Time Method Change in bone ageTimepoint: Time Frame: From baseline (week 0) to week 52 <br/ ><br>unit: years;Change in fasting plasma glucoseTimepoint: From screening (visit 1) to week 26 <br/ ><br>unit :mmol/l;Change in glycated haemoglobin (HbA1c)Timepoint: From screening (visit 1) to week 26 <br/ ><br>unit:Percentage points;Change in height standard deviation score (SDS) <br/ ><br>Timepoint: From baseline (week 0) to week 26 <br/ ><br>unit : minus 10 to Plus 10;Change in height velocity SDSTimepoint: From baseline (week 0) to week 26 <br/ ><br>unit : minus 10 to plus 10;Change in homeostatic model assessment (HOMA)Timepoint: From screening (visit 1) to week 26 <br/ ><br>unit:percent;Change in insulin-like growth factor binding protein 3 (IGFBP-3) SDSTimepoint: From screening (visit 1) to week 26 <br/ ><br>unit: minus 10 to plus 10;Change in insulin-like growth factor I (IGF-I) SDSTimepoint: From screening (visit 1) to week 26 <br/ ><br>unit : minus 10 to plus 10