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.

• Sponsor: Novo Nordisk A/S (Industry)
• Overall Status: Recruiting
• CT.gov ID: NCT04970654
• Collaborator: (none)
• Enrollment: 108
• Locations: 20
• Arms: 2
• Anticipated Duration (Months): 29.3
• Patients Per Site: 5.4
• Patients Per Site Per Month: 0.2

Study Details

Study Description

Brief Summary

The study compares 2 medicines for children who do not have enough hormone to grow:

somapacitan given once a week (a new medicine) and Norditropin® given once a day (the medicine doctors can already prescribe). Researchers will test to see how well somapacitan works.

The study will also test if somapacitan is safe. Participants will either get somapacitan or Norditropin® - which treatment participants get, is decided by chance.

The study includes a 52 week treatment period and a minimum of 30 days follow up period.

Condition or Disease	Intervention/Treatment	Phase
Growth Hormone Deficiency in Children	Drug: somapacitan; Drug: Norditropin®	Phase 3

Study Design

• Study Type: Interventional
• Anticipated Enrollment: 108 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• 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
• Actual Study Start Date: Jul 22, 2021
• Anticipated Primary Completion Date: Dec 31, 2023
• Anticipated Study Completion Date: Dec 31, 2023

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Somapacitan weekly participants will receive once-weekly somapacitan for 52 weeks	Drug: somapacitan; 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.
Active Comparator: Norditropin® daily Participants will receive Norditropin® daily for 52 weeks	Drug: Norditropin®; 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.

Outcome Measures

Primary Outcome Measures

1. Height velocity [week 0 - 52]

   Height velocity (HV) is measured in cm/year. HV = (height at 52 weeks visit - height at baseline)/(time from baseline to 52 weeks visit in years).

Secondary Outcome Measures

1. Change in height Standard Deviation Score (SDS) [from baseline (week 0) to week 52]

   Measured in scores (-10 to +10). Height SDS will be derived using Centre for Disease Control and Prevention (CDC) standards.

2. Change in height Velocity Standard Deviation Score [from baseline (week 0) to week 52]

   Measured in scores (-10 to +10). HV SDS will be derived using Prader standards as reference data.

3. Change in bone age [from Visit 1 to Week 52]

   Measured in years

4. Change in fasting plasma glucose [from baseline (week 0) to week 52]

   Measured in mmol/L.

5. Change in glycated haemoglobin (HbA1c) [from baseline (week 0) to week 52]

   Measured in percentage

6. Change in IGF-I Standard Deviation Score [From baseline (week 0) to week 52]

   Measured in scores (-10 to +10)

7. Change in IGFBP-3 Standard Deviation Score [From baseline (week 0) to week 52]

   Measured in scores (-10 to +10)

Eligibility Criteria

Criteria

• Ages Eligible for Study: 2 Years to 11 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Novo Nordisk Investigational Site	Hefei	Anhui	China	230601
2	Novo Nordisk Investigational Site	Beijing	Beijing	China	100020
3	Novo Nordisk Investigational Site	Beijing	Beijing	China	100045
4	Novo Nordisk Investigational Site	Guangzhou	Guangdong	China	510080
5	Novo Nordisk Investigational Site	Zhengzhou	Henan	China	450018
6	Novo Nordisk Investigational Site	Wuhan	Hubei	China	430030
7	Novo Nordisk Investigational Site	Changsha	Hunan	China	410007
8	Novo Nordisk Investigational Site	Changsha	Hunan	China	410011
9	Novo Nordisk Investigational Site	Shaoyang	Hunan	China	422000
10	Novo Nordisk Investigational Site	Suzhou	Jiangsu	China	215025
11	Novo Nordisk Investigational Site	Wuxi	Jiangsu	China	214023
12	Novo Nordisk Investigational Site	Nanchang	Jiangxi	China	330006
13	Novo Nordisk Investigational Site	Pingxiang	Jiangxi	China	337055
14	Novo Nordisk Investigational Site	Changchun	Jilin	China	130021
15	Novo Nordisk Investigational Site	Jinan	Shandong	China	250021
16	Novo Nordisk Investigational Site	Linyi	Shandong	China	276016
17	Novo Nordisk Investigational Site	Qingdao	Shandong	China	266043
18	Novo Nordisk Investigational Site	Chengdu	Sichuan	China	610000
19	Novo Nordisk Investigational Site	Hangzhou	Zhejiang	China	310003
20	Novo Nordisk Investigational Site	Jiaxing	Zhejiang	China	314000

Sponsors and Collaborators

• Novo Nordisk A/S

Investigators

• Study Director: Clinical Transparency (dept. 1452), Novo Nordisk A/S

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Novo Nordisk A/S
• ClinicalTrials.gov Identifier: NCT04970654
• Other Study ID Numbers: NN8640-4468; U1111-1250-7530; 2020-002974-28
• First Posted: Jul 21, 2021
• Last Update Posted: Jan 31, 2022
• Last Verified: Jan 1, 2022

• Individual Participant Data (IPD) Sharing Statement: Yes
• Plan to Share IPD: Yes
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Product Manufactured in and Exported from the U.S.: Yes
• Additional relevant MeSH terms: Dwarfism, Pituitary

Study Results

No Results Posted as of Jan 31, 2022