Investigating Efficacy and Safety of Once-weekly NNC0195-0092 (Somapacitan) Treatment Compared to Daily Growth Hormone Treatment (Norditropin® FlexPro®) in Growth Hormone Treatment naïve Pre-pubertal Children With Growth Hormone Deficiency

Sponsor

Novo Nordisk A/S (Industry)

Overall Status

Recruiting

CT.gov ID

NCT02616562

Collaborator

(none)

Enrollment

Locations

Arms

101.1

Anticipated Duration (Months)

1.3

Patients Per Site

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This trial is conducted globally. The aim of the trial is to investigate efficacy and safety of once-weekly NNC0195-0092 (somapacitan) treatment compared to daily growth hormone treatment (Norditropin® FlexPro®) in growth hormone treatment naïve pre-pubertal children with growth hormone deficiency.

The trial consists of a 26 week main trial period, followed by a 26 week extension trial period, a 104 week safety extension period, a 208 week longterm safety extension trial period and a 30 day follow up period. Participants receive NNC0195-0092 (somapacitan) (0.04 mg/kg/week) during the main trial and the extension period and thereafter NNC0195-0092 (somapacitan) (0.16 mg/kg/week) during the safety extension and the long-term safety extension periods. Two additional age groups, cohort II (age below 2 years and 26 weeks at screening) and cohort III (above 9 years (girls)/ above 10 years (boys) and equal to or below 17 years at screening) are included in the 208 week long-term safety extension trial period only.

Condition or Disease	Intervention/Treatment	Phase
Growth Hormone Disorder Growth Hormone Deficiency in Children	Drug: somapacitan Drug: Norditropin® FlexPro® pen	Phase 2

Study Design

Study Type:

Interventional

Anticipated Enrollment :

74 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Masking Description:

Sponsor staff involved in the clinical trial is masked according to company standard procedures.

Primary Purpose:

Treatment

Official Title:

A Trial Investigating Efficacy and Safety of Once-weekly NNC0195-0092 (Somapacitan) Treatment Compared to Daily Growth Hormone Treatment (Norditropin® FlexPro®) in Growth Hormone Treatment naïve Pre-pubertal Children With Growth Hormone Deficiency naïve Pre-pubertal Children With Growth Hormone Deficiency

Actual Study Start Date :

Mar 23, 2016

Anticipated Primary Completion Date :

Aug 26, 2024

Anticipated Study Completion Date :

Aug 26, 2024

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Blinded NNC0195-0092 (somapacitan) (0.04 mg/kg/week) Participants receive NNC0195-0092 (somapacitan) (0.04 mg/kg/week) during the main trial and the extension period and thereafter NNC0195-0092 (somapacitan) (0.16 mg/kg/week) during the safety extension and the long-term safety extension periods.	Drug: somapacitan Administered subcutaneously (s.c., under the skin) once-weekly. Other Names: NNC0195-0092
Experimental: Blinded NNC0195-0092 (somapacitan) (0.08 mg/kg/week) Participants receive NNC0195-0092 (somapacitan) (0.08 mg/kg/week) during the main trial and the extension period and thereafter NNC0195-0092 (somapacitan) (0.16 mg/kg/week) during the safety extension and the long-term safety extension periods.	Drug: somapacitan Administered subcutaneously (s.c., under the skin) once-weekly. Other Names: NNC0195-0092
Experimental: Blinded NNC0195-0092 (somapacitan) (0.16 mg/kg/week) Participants receive the same dose (0.16 mg/kg/week) of NNC0195-0092 (somapacitan) during all 4 trial periods.	Drug: somapacitan Administered subcutaneously (s.c., under the skin) once-weekly. Other Names: NNC0195-0092
Active Comparator: Open labelled daily Norditropin® (0.034 mg/kg/day) Participants receive Norditropin during the main trial, the extension period and the safety extension period and thereafter NNC0195-0092 (somapacitan) (0.16 mg/kg/week) during the long-term safety extension periods.	Drug: somapacitan Administered subcutaneously (s.c., under the skin) once-weekly. Other Names: NNC0195-0092 Drug: Norditropin® FlexPro® pen Administered subcutaneously (s.c., under the skin) once daily.

Outcome Measures

Primary Outcome Measures

Cohort I: Height velocity (HV) during the first 26 weeks of treatment, measured as standing height with stadiometer [Week 0-26]
cm/year
Cohort II and III: Incidence of adverse events, including injection site reactions, in children with GHD [During 208 weeks]
Number of events

Secondary Outcome Measures

Change in height standard deviation score (SDS) [Week 0-26]
Typically -10 to +10
Change in height standard deviation score (SDS) [Week 0-52]
Typically -10 to +10
Change in height standard deviation score (SDS) [Week 26-52]
Typically -10 to +10
Change in HV (height velocity) SDS [Week 0-26]
Typically -10 to +10. Baseline (week 0) HV SDS is derived from reported pre-trial standing height measured at minimum 6 months and maximum 18 months prior to screening visit to standing height at baseline (week 0)
Change in HV (height velocity) SDS [Week 0-52]
Baseline (week 0) HV SDS is derived from reported pre-trial standing height measured at minimum 6 months and maximum 18 months prior to screening visit to standing height at baseline (week 0)
Insulin-like growth factor 1 (IGF-I) SDS [Week 0-26]
Typically -10 to +10
Insulin-like growth factor 1 (IGF-I) SDS [Week 0-52]
Typically -10 to +10
Insulin-like growth factor 1 (IGF-I) SDS [Week 26-52]
Typically -10 to +10
Insulin-like growth factor binding protein 3 (IGFBP-3) SDS [Week 0-26]
Typically -10 to +10
Insulin-like growth factor binding protein 3 (IGFBP-3) SDS [Week 0-52]
Typically -10 to +10
Insulin-like growth factor binding protein 3 (IGFBP-3) SDS [Week 26-52]
Typically -10 to +10
Height velocity [Week 52]
cm/year, derived from standing height from baseline (week 0) to week 52
Bone age [Week 52]
X-Ray of left hand and wrist, central assessed according to Greulich & Pyle atlas progression vs. chronological age
Serum NNC0195-0092 (somapacitan) concentrations [Week 52]
ng/mL
Changes in emotional well-being score, physical health score, social well-being score and total score in Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer (TRIM-CGHD-O) [Week 0-26]
The scores range from 0-100. A lower score indicates a better health state.
Changes in emotional well-being score, physical health score, social well-being score and total score in Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer (TRIM-CGHD-O) [Week 0-52]
The scores range from 0-100. A lower score indicates a better health state.
Total score of The Treatment Burden Measure - Child Growth Hormone Deficiency - Observer (TB-CGHD-O) [Week 26]
The scores range from 0-100. A lower score indicates a better health state.
Total score of The Treatment Burden Measure - Child Growth Hormone Deficiency - Observer (TB-CGHD-O) [Week 52]
The scores range from 0-100. A lower score indicates a better health state.
Total score of The Treatment Burden Measure - Child Growth Hormone Deficiency - Parent/Guardian (TB-CGHD-P) [Week 26]
The scores range from 0-100. A lower score indicates a better health state.
Total score of The Treatment Burden Measure - Child Growth Hormone Deficiency - Parent/Guardian (TB-CGHD-P) [Week 52]
The scores range from 0-100. A lower score indicates a better health state.
Incidence of adverse events, including injection site reactions [Week 364]
Number of events
Occurrence of anti-NNC0195-0092 (somapacitan) antibodies [Week 364]
Yes/no
Occurrence of anti-hGH antibodies [Week 364]
Yes/no

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A to 17 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Cohort I:

Boys: Tanner stage 1 for pubic hair and testis volume below 4 ml , age at least 2 years and 26 weeks and below or equal to 10.0 years at screening
Girls: Tanner stage 1 for breast development (no palpable glandular breast tissue) and pubic hair, age at least 2 years and 26 weeks and below or equal to 9.0 years at screening
Confirmed diagnosis of GHD (growth hormone deficiency) within 12 months prior to screening as determined by two different GH (growth hormone) stimulation tests, defined as a peak GH level of below or equal to 7.0 ng/ml. For children with three or more pituitary hormone deficiencies only one GH stimulation test is needed
No prior exposure to GH therapy and/or IGF-I (insulin-like growth factor I) treatment
Height of at least 2.0 standard deviations below the mean height for chronological age (CA) and gender according to the standards of Centers for Disease Control and Prevention 2-20 years: Girls/Boys stature-for-age and weight-for-age percentiles CDC at screening
Annualized height velocity (HV) below the 25th percentile for CA (chronological age) and gender or below -0.7 SD (standard deviation) score for CA and sex, according to the standards of Prader calculated over a time span of minimum 6 months and maximum 18 months

Cohort II:

Below 2 years and 26 weeks and a minimum weight of 5 kg at screening.
Confirmed diagnosis of GHD, the GHD diagnosis must be confirmed by investigator according to local practice.
For GH treatment naïve subjects, no prior exposure to GH therapy and/or IGF-I treatment.
For GH treatment naïve subjects, IGF-1 SDS below -1.0 at screening, compared to age and sex normalized range according to central laboratory measurements.

Cohort III:

Age:

Girls: Above 9 years and below or equal to 17 years at screening.
Boys: Above 10 years and below or equal to 17 years at screening.
Confirmed diagnosis of GHD

for GH treatment naïve subjects, confirmed diagnosis within 12 months prior to screening as determined by two different GH stimulation tests, defined as a peak GH level of equal to or below 7.0 ng/ml. For children with three or more pituitary hormone deficiencies only one GH stimulation test is needed. FOR JAPAN ONLY: Confirmed diagnosis of GHD within 12 months prior to screening as determined by one GH stimulation tests for patients with intracranial organic disease or symptomatic hypoglycaemia and two different GH stimulation test for other patients, defined as a peak GH level of equal to or below 6 ng/ml by assay using recombinant GH standard.
for non-GH treatment naïve subjects, confirmed GHD diagnosis by investigator according to local practice

For GH treatment naïve subjects, no prior exposure to GH therapy and/or IGF-I treatment.
Open epiphyses; defined as bone age below 14 years for females and bone age below 16 years for males.

Exclusion Criteria:

Any clinically significant abnormality likely to affect growth or the ability to evaluate
growth with standing/length measurements: Chromosomal aneuploidy and significant gene mutations causing medical "syndromes" with short stature, including but not limited to Turner syndrome, Laron syndrome, Noonan syndrome, or absence of GH receptors. Congenital abnormalities (causing skeletal abnormalities), including but not limited to Russell-Silver Syndrome, skeletal dysplasias. Significant spinal abnormalities including but not limited to scoliosis, kyphosis and spina bifida variants
Children born small for gestational age (SGA - birth weight and/or birth length below-2 SD for gestational age)
Concomitant administration of other treatments that may have an effect on growth (not applicable to non-GH treatment naïve subjects in cohort II and III), including but not limited to methylphenidate for treatment of attention deficit hyperactivity disorder (ADHD)
Prior history or presence of malignancy and/or intracranial tumour

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Novo Nordisk Investigational Site	San Diego	California	United States	92123
2	Novo Nordisk Investigational Site	Centennial	Colorado	United States	80112
3	Novo Nordisk Investigational Site	Wilmington	Delaware	United States	19803
4	Novo Nordisk Investigational Site	Minneapolis	Minnesota	United States	55454
5	Novo Nordisk Investigational Site	Morristown	New Jersey	United States	07962
6	Novo Nordisk Investigational Site	New Brunswick	New Jersey	United States	08901
7	Novo Nordisk Investigational Site	Mineola	New York	United States	11501
8	Novo Nordisk Investigational Site	Cincinnati	Ohio	United States	45229
9	Novo Nordisk Investigational Site	Tacoma	Washington	United States	98405
10	Novo Nordisk Investigational Site	Graz		Austria	8036
11	Novo Nordisk Investigational Site	Linz		Austria	4020
12	Novo Nordisk Investigational Site	Salzburg		Austria	A 5020
13	Novo Nordisk Investigational Site	St. Poelten		Austria	A 3100
14	Novo Nordisk Investigational Site	Villach		Austria	9500
15	Novo Nordisk Investigational Site	Vöcklabruck		Austria	A 4840
16	Novo Nordisk Investigational Site	Brussel		Belgium	1090
17	Novo Nordisk Investigational Site	Bruxelles		Belgium	1200
18	Novo Nordisk Investigational Site	Leuven		Belgium	3000
19	Novo Nordisk Investigational Site	Liège		Belgium	4030
20	Novo Nordisk Investigational Site	Porto Alegre	Rio Grande Do Sul	Brazil	90610-000
21	Novo Nordisk Investigational Site	São Paulo	Sao Paulo	Brazil	01228-000
22	Novo Nordisk Investigational Site	Curitiba		Brazil	80030-110
23	Novo Nordisk Investigational Site	ANGERS cedex 09		France	49033
24	Novo Nordisk Investigational Site	Bordeaux		France	33076
25	Novo Nordisk Investigational Site	Nantes		France	44093
26	Novo Nordisk Investigational Site	Paris		France	75015
27	Novo Nordisk Investigational Site	Rennes		France	35056
28	Novo Nordisk Investigational Site	Toulouse cedex 9		France	31059
29	Novo Nordisk Investigational Site	Frankfurt		Germany	60596
30	Novo Nordisk Investigational Site	Ulm		Germany	89075
31	Novo Nordisk Investigational Site	Kochi	Kerala	India	682041
32	Novo Nordisk Investigational Site	Pune	Maharashtra	India	411001
33	Novo Nordisk Investigational Site	New Dehli	New Delhi	India	110029
34	Novo Nordisk Investigational Site	Beer Sheva		Israel	84101
35	Novo Nordisk Investigational Site	Haifa		Israel	31096
36	Novo Nordisk Investigational Site	Kfar Saba		Israel	44281
37	Novo Nordisk Investigational Site	Petah Tikva		Israel	49202
38	Novo Nordisk Investigational Site	Tel Hashomer		Israel	52621
39	Novo Nordisk Investigational Site	Fukuoka		Japan	812-8582
40	Novo Nordisk Investigational Site	Fukuoka		Japan	830-0011
41	Novo Nordisk Investigational Site	Kanagawa		Japan	216-8511
42	Novo Nordisk Investigational Site	Kyoto		Japan	602-8566
43	Novo Nordisk Investigational Site	Osaka		Japan	534-0021
44	Novo Nordisk Investigational Site	Osaka		Japan	553-0003
45	Novo Nordisk Investigational Site	Osaka		Japan	594-1101
46	Novo Nordisk Investigational Site	Tokyo		Japan	113-8519
47	Novo Nordisk Investigational Site	Tokyo		Japan	157 8535
48	Novo Nordisk Investigational Site	Ljubljana		Slovenia	1525
49	Novo Nordisk Investigational Site	Stockholm		Sweden	171 76
50	Novo Nordisk Investigational Site	Umeå		Sweden	901 85
51	Novo Nordisk Investigational Site	Adana		Turkey	01130
52	Novo Nordisk Investigational Site	Ankara		Turkey	06230
53	Novo Nordisk Investigational Site	Istanbul		Turkey	34093
54	Novo Nordisk Investigational Site	Istanbul		Turkey	34890
55	Novo Nordisk Investigational Site	Ivano-Frankivsk		Ukraine	76018
56	Novo Nordisk Investigational Site	Kyiv		Ukraine	04114

Sponsors and Collaborators

Novo Nordisk A/S

Investigators

Study Director: Clinical Reporting Anchor and Disclosure (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:

NCT02616562

Other Study ID Numbers:

NN8640-4172
2015-000531-32
U1111-1166-7062

First Posted:

Nov 30, 2015

Last Update Posted:

Mar 21, 2022

Last Verified:

Mar 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:

Additional relevant MeSH terms:

Dwarfism, Pituitary

Endocrine System Diseases

Study Results

No Results Posted as of Mar 21, 2022