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.

A Trial Comparing the Efficacy and Safety of Once Weekly Dosing of Somapacitan With Daily Norditropin® in Chinese Children With Growth Hormone Deficiency

Status

Completed

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT04970654

Enrollment

110

Registered

2021-07-21

Start date

2021-07-22

Completion date

2023-12-18

Last updated

2026-01-06

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Growth Hormone Deficiency in Children

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.

Interventions

DRUGsomapacitan

Somapacitan (0.16 mg/kg/week) will be administered subcutaneously (s.c.; under the skin) once weekly by PDS290 pen-injector. Somapacitan can be injected any time during the once weekly dosing day. The dose will be calculated based on the subject's current body weight.

DRUGNorditropin®

Norditropin® (0.034 mg/kg/day) will be administered s.c. once daily by FlexPro® pen-injector. Norditropin® should be injected daily in the evening. The dose will be calculated based on the subject's current body weight.

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Healthy volunteers

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.

Design outcomes

Primary

Measure	Time frame	Description
Height Velocity	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).

Secondary

Measure	Time frame	Description
Change in Height Standard Deviation Score	Baseline (week 0), week 52	Change in the height standard deviation score (HSDS) is presented from baseline (week 0) to week 52. HSDS was derived using Chinese general population standards. The formula to calculate HSDS is: HSDS = ((Height / M)\\L-1) / (L\*S). L: The sex and age-specific power in the Box-Cox transformation, M: The sex and age-specific median, S: The sex and age-specific generalized coefficient of variation. The range for HSDS was -10 to +10. Negative scores indicated a height below the mean height for a child with the same age and gender, whereas positive scores indicated a height above the mean height for a child with the same age and gender. This outcome measure was analysed based on the 'on treatment' observation period. On treatment observation period: It starts from first administration of trial product and up until last trial contact, visit 7 (52 weeks+5 days) or 14 days after last administration, whichever comes first.
Change in Height Velocity Standard Deviation Score	Baseline (week 0), week 52	Change in height velocity standard deviation score (HVSDS) is presented from baseline (week 0) to week 52. HVSDS was derived using Prader standards. HVSDS was calculated using the formula: HVSDS = (height velocity - mean)/standard deviation (SD), where height velocity was the height velocity variable measured, mean and SD of height velocity by gender and age for the reference population. The range for HVSDS was -10 to +10. Negative scores indicated a height velocity below the mean height velocity for a child with the same age and gender, whereas positive scores indicated a height velocity above the mean height velocity for a child with the same age and gender. This outcome measure was analysed based on the 'on treatment' observation period. On treatment observation period: It starts from first administration of trial product and up until last trial contact, visit 7 (52 weeks+5 days) or 14 days after last administration, whichever comes first.
Change in Fasting Plasma Glucose	Baseline (week 0), week 52	Change in fasting plasma glucose is presented from baseline (week 0) to week 52. This outcome measure was analysed based on the 'on treatment' observation period. On treatment observation period: It starts from first administration of trial product and up until last trial contact, visit 7 (52 weeks+5 days) or 14 days after last administration, whichever comes first.
Change in Bone Age	Week -2, week 52	Change in bone age is presented from week -2 to week 52. This outcome measure was analysed based on the 'on treatment' observation period. On treatment observation period: It starts from first administration of trial product and up until last trial contact, visit 7 (52 weeks+5 days) or 14 days after last administration, whichever comes first.
Change in IGF-I Standard Deviation Score	Baseline (week 0), week 52	Change in Insulin like growth factor-I (IGF-I) standard deviation score is presented from baseline (week 0) to week 52. The range for IGF-I SDS was from -10 to +10. Negative scores indicated a IGF-I below the mean IGF-I for a child with the same age and gender, whereas positive scores indicated a IGF-I above the mean IGF-I for a child with the same age and gender. This outcome measure was analysed based on the 'on treatment' observation period. On treatment observation period: It starts from first administration of trial product and up until last trial contact, visit 7 (52 weeks+5 days) or 14 days after last administration, whichever comes first.
Change in IGFBP-3 Standard Deviation Score	Baseline (week 0), week 52	Change in Insulin like growth factor binding protein 3 (IGFBP-3) standard deviation score is presented from baseline (week 0) to week 52. The range for IGFBP-3 SDS was from -10 to +10. Negative scores indicated a IGFBP-3 below the mean IGFBP-3 for a child with the same age and gender, whereas positive scores indicated a IGFBP-3 above the mean IGFBP-3 for a child with the same age and gender. This outcome measure was analysed based on the 'on treatment' observation period. On treatment observation period: It starts from first administration of trial product and up until last trial contact, visit 7 (52 weeks+5 days) or 14 days after last administration, whichever comes first.
Change in HbA1c	Baseline (week 0), week 52	Change in glycosylated haemoglobin (HbA1c) is presented from baseline (week 0) to week 52 is presented. This outcome measure was analysed based on the 'on treatment' observation period. On treatment observation period: It starts from first administration of trial product and up until last trial contact, visit 7 (52 weeks+5 days) or 14 days after last administration, whichever comes first.

Countries

China

Participant flow

Recruitment details

The trial was conducted at 20 sites in China.

Pre-assignment details

Participants were randomized in a 2:1 ratio to receive either somapacitan or Norditropin.

Participants by arm

Arm	Count
Norditropin Participants received Norditropin 0.034 mg/kg (milligram per kilogram) subcutaneous injection once daily using PDS290 pen injector for 52 weeks.	36
Somapacitan Participants received somapacitan 0.16 mg/kg subcutaneous injection once weekly using PDS290 pen injector for 52 weeks.	74
Total	110

Withdrawals & dropouts

Period	Reason	FG000	FG001
Overall Study	Lost to Follow-up	1	0
Overall Study	Unclassified	2	1
Overall Study	Withdrawal by parents/guardian	1	2

Baseline characteristics

Characteristic	Somapacitan	Total	Norditropin
Age, Continuous	6.6 Years STANDARD_DEVIATION 2.1	6.5 Years STANDARD_DEVIATION 2.2	6.5 Years STANDARD_DEVIATION 2.3
Race (NIH/OMB) American Indian or Alaska Native	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) Asian	74 Participants	110 Participants	36 Participants
Race (NIH/OMB) Black or African American	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) More than one race	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) White	0 Participants	0 Participants	0 Participants
Sex: Female, Male Female	9 Participants	15 Participants	6 Participants
Sex: Female, Male Male	65 Participants	95 Participants	30 Participants

Adverse events

Event type	EG000 affected / at risk	EG001 affected / at risk
deaths Total, all-cause mortality	0 / 36	0 / 74
other Total, other adverse events	29 / 36	60 / 74
serious Total, serious adverse events	1 / 36	8 / 74

Outcome results

Primary

Height Velocity

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).

Time frame: Baseline (Week 0); Week 52

Population: FAS included all randomized participants. Here, Overall number of participants analysed = participants with available data for this outcome measure.

Arm	Measure	Value (MEAN)	Dispersion
Norditropin	Height Velocity	10.5 Cm/year (centimeter per year)	Standard Deviation 2.3
Somapacitan	Height Velocity	11.0 Cm/year (centimeter per year)	Standard Deviation 2.1

Comparison: Hypothetical strategy estimand. Height velocity at 52 weeks was analyzed using a mixed model for repeated measurements, with treatment, gender, age group, growth hormone (GH) peak group and gender by age group interaction term as factors and baseline height as a covariate, all nested within week as a factor.95% CI: [-0.2, 1.3]

Secondary

Change in Bone Age

Change in bone age is presented from week -2 to week 52. This outcome measure was analysed based on the 'on treatment' observation period. On treatment observation period: It starts from first administration of trial product and up until last trial contact, visit 7 (52 weeks+5 days) or 14 days after last administration, whichever comes first.

Time frame: Week -2, week 52

Population: FAS included all randomized participants. Here, Overall number of participants analysed = participants with available data for this outcome measure.

Arm	Measure	Value (MEAN)	Dispersion
Norditropin	Change in Bone Age	5.6 Years	Standard Deviation 2.2
Somapacitan	Change in Bone Age	5.5 Years	Standard Deviation 2.1

Secondary

Change in Fasting Plasma Glucose

Change in fasting plasma glucose is presented from baseline (week 0) to week 52. This outcome measure was analysed based on the 'on treatment' observation period. On treatment observation period: It starts from first administration of trial product and up until last trial contact, visit 7 (52 weeks+5 days) or 14 days after last administration, whichever comes first.