Growth Hormone DisorderPhase 2NCT02616562
Investigating Efficacy and Safety of Once-weekly NNC0195-0092 Treatment Compared to Daily Growth Hormone Treatment (Norditropin® FlexPro®) in Growth Hormone Treatment naïve Pre-pubertal Children With Growth Hormone Deficiency
A Phase 2 study testing somapacitan and Norditropin® FlexPro® pen in people with Growth Hormone Disorder and Growth Hormone Deficiency in Children, run by Novo Nordisk A/S at 89 sites in 14 countries.
Trial summary
This trial is conducted globally. The aim of the trial is to investigate efficacy and safety of once-weekly NNC0195-0092 treatment compared to daily growth hormone treatment (Norditropin® FlexPro®) in growth hormone treatment naïve pre-pubertal children with growth hormone deficiency. Read more about this study
- Who is it for?
- Ages 2 to 10 years
- Conditions the study is for
- Growth Hormone Disorder, Growth Hormone Deficiency in Children
- What is being tested?
- somapacitan and Norditropin® FlexPro® pen
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 76 participants
- Where
- 89 sitesin 14 countries
- Study dates
- 31 Mar 2016to 26 Sep 2024
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 10 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- Any clinically significant abnormality likely to affect growth or the ability to evaluate
- growth with standing 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, including but not limited to methylphenidate for treatment of attention deficit hyperactivity disorder (ADHD)
- Prior history or presence of malignancy and/or intracranial tumour
Trial locations and contacts
89 sites in 14 countries.
89 sites in 14 countries
United States27 sites
Dell Pediatric Research Institute
Austin, Texas, 78723
Children's of Alabama
Birmingham, Alabama, 35233
UBMD Peds-Div of Endo/Diabetes
Buffalo, New York, 14203
Rocky Mt Ped and Endo
Centennial, Colorado, 80112
Childrens Hospital of Chicago
Chicago, Illinois, 60611
CCHMC_Cinc
Cincinnati, Ohio, 45229
Novo Nordisk Clinical Trial Call Center
Cincinnati, Ohio, 45229
Ped Endo Assoc PC-G.V
Greenwood Village, Colorado, 80111-2803
Riley Hospital For Children
Indianapolis, Indiana, 46202
Children's Hospital Los Angeles - Endocrinology
Los Angeles, California, 90027
Mattel Children's Hospital at UCLA
Los Angeles, California, 90095
Valley Children's Hospital
Madera, California, 93636-8762
Novo Nordisk Clinical Trial Call Center
Mineola, New York, 11501
NYU Langone Hospital-LI
Mineola, New York, 11501
Novo Nordisk Clinical Trial Call Center
Minneapolis, Minnesota, 55454
University of Minnesota_Minneapolis_2
Minneapolis, Minnesota, 55454
Goryeb Children's Hospital
Morristown, New Jersey, 07962
Novo Nordisk Clinical Trial Call Center
Morristown, New Jersey, 07962
Rutgers-Rwjms
New Brunswick, New Jersey, 08901
Children's Hsptl Of The Kings
Norfolk, Virginia, 23507
The Endocrine Center
Pembroke Pines, Florida, 33028
Endocrine Associates Of Dallas
Plano, Texas, 75093
Novo Nordisk Clinical Trial Call Center
San Diego, California, 92123
The Regents of the Univ of CA
San Diego, California, 92123
MultiCare Inst for Res & Innov
Tacoma, Washington, 98405
University Of Oklahoma-Tulsa
Tulsa, Oklahoma, 74135
Nemours/AI duPont Hosp-Chld
Wilmington, Delaware, 19803
Japan11 sites
Kyushu Univ. HP, Maternity & Perinatal Care Center
Fukuoka, 812-8582
Kurume University Hospital, Pediatrics
Fukuoka, 830-0011
St. Marianna University School of Medicine Hospital_Pediatrics
Kanagawa, 216-8511
Univ.HP, Kyoto Pref Univ of Medicine, Dept. of Pediatrics
Kyoto, 602-8566
Osaka City General Hospital, Pediatric Endocrinology and Me
Osaka, 534-0021
JCHO Osaka Hospital_Pediatric
Osaka, 553-0003
Osaka Women's and Children's Hospital
Osaka, 594-1101
Institute of Science Tokyo Hospital_Pediatrics
Tokyo, 113-8519
Tokyo Medical and Dental University Hospital
Tokyo, 113-8519
National Center for Child Health and Dev, Endo and Metabo
Tokyo, 157 8535
Study site
Tokyo, 157 8535
Austria7 sites
Med. Univ. Graz -Klinische Abteilung f. Allgemeine Pädiatrie
Graz, 8036
Study site
Graz, 8036
Kepler Universitätsklinikum GmbH - Med Campus IV (vorm.LFKK)
Linz, Upper Austria, 4020
LKH Salzburg- Univ. Klinik f. Kinder- und Jugendheilkunde
Salzburg, 5020
LKH St. Poelten, Kinder-und Jugendheilkunde
Sankt Pölten, 3100
Landeskrankenhaus Villach
Villach, 9500
Salzkammergut-Klinikum Vöcklabruck
Vöcklabruck, 4840
France7 sites
Centre Hospitalier Universitaire D'Angers-2
Angers, 49033
Centre Hospitalier Universitaire de Bordeaux-Hopital Pellegrin
Bordeaux, 33076
Study site
Bordeaux, 33076
Centre Hospitalier Universitaire de Nantes-Hopital Enfant-Adolescent
Nantes, 44000
Ap-Hp-Hopital Necker
Paris, 75015
HOPITAL SUD de RENNES
Rennes, 35056
HOPITAL DES ENFANTS-HOPITAL PAULE DE VIGUIER - Pharmacie
Toulouse, 31059
Israel6 sites
Soroka MC - Pediatric Endocrinology
Beersheba, 84101
Rambam MC - Department of Pediatrics A
Haifa, 31096
Meir MC - Department of Paediatrics
Kfar Saba, 44281
Study site
Kfar Saba, 44281
Schneider MC - Endrocrinology and Diabetes
Petah Tikva, 49202
Sheba MC - Pediatric endocrinology and adolescent diabetes clinics
Tel Litwinsky, 52621
Turkey (Türkiye)6 sites
Çukurova Üniversitesi Tıp Fakültesi Balcalı Hastanesi
Adana, 01130
Hacettepe Üniversitesi Hastanesi- Endokrinoloji
Ankara, 06230
I.U Istanbul Medical Faculty
Istanbul, 34093
Study site
Istanbul, 34093
Marmara University Medical Faculty
Istanbul, 34854
Marmara Üniversitesi Pendik EAH- Başıbüyük Yerleşkesi- Kardiyoloji
Istanbul, 34854
Belgium5 sites
UZ Brussel
Brussels, 1090
Study site
Brussels, 1090
Cliniques Universitaires Saint-Luc - Serv. Pédiatrie
Brussels, 1200
UZ Leuven - Kindergeneeskunde
Leuven, 3000
CHU de Liège, site N.-D. des Bruyères
Liège, 4030
Brazil4 sites
Serviço de Endocrinologia e Metabologia do HC-UFPR
Curitiba, Paraná, 80030-110
Hospital São Lucas - PUC/RS
Porto Alegre, Rio Grande do Sul, 90610-000
CPQuali Pesquisa Clínica Ltda
São Paulo, São Paulo, 01228-000
Study site
São Paulo, São Paulo, 01228-000
India4 sites
Amrita Institute Of Medical Sciences & Research Centre
Kochi, Kerala, 682041
All India Institute of Medical Sciences
New Dehli, New Delhi, 110029
Study site
New Dehli, New Delhi, 110029
Jehangir Clinical Development Centre
Pune, Maharashtra, 411001
Germany3 sites
Study site
Frankfurt, 60596
Endokrinologikum Frankfurt
Frankfurt am Main, 60596
Uniklinik Ulm - Dt. Zentrum für Kinder- und Jugendgesundheit (DZKJ)
Ulm, 89075
Sweden3 sites
Astrid Lindgrens Barnsjukhus
Stockholm, 171 76
Study site
Stockholm, 171 76
Barn och ungdomscentrum Västerbotten
Umeå, 901 85
Ukraine3 sites
Ivano-Frankivsk Regional Clinical Children Hospital - Endocrinology dept.
Ivano-Frankivsk, 76018
Study site
Kiev, 04114
Komisarenko Institute of Endocrinology and Metabolism of NAMSU - Department of paediatric endocrine pathology
Kyiv, 04114
Slovenia2 sites
University Children's Hospital
Ljubljana, 1525
Study site
Ljubljana, 1525
About this study
Official title: A Randomised, Multinational, Active-controlled, (Open-labelled), Dose Finding, (Double-blinded), Parallel Group Trial Investigating Efficacy and Safety of Once-weekly NNC0195-0092 Treatment Compared to Daily Growth Hormone Treatment (Norditropin® FlexPro®) in Growth Hormone Treatment naïve Pre-pubertal Children With Growth Hormone Deficiency
This trial is conducted globally. The aim of the trial is to investigate efficacy and safety of once-weekly NNC0195-0092 treatment compared to daily growth hormone treatment (Norditropin® FlexPro®) in growth hormone treatment naïve pre-pubertal children with growth hormone deficiency.
The main trial period will consist of 26 weeks of treatment, followed by a 26 week extension period.
- Being tested
- somapacitan, Norditropin® FlexPro® pen
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
somapacitanDrug
Also called NNC0195-0092
Administered subcutaneously (s.c., under the skin) once-weekly.
Norditropin® FlexPro® penDrug
Administered subcutaneously (s.c., under the skin) once daily.
| Study group | Role | What they receive |
|---|---|---|
| Cohort I Norditropin/somapacitan | Experimental | Participants received Norditropin subcutaneously daily in main trial period, extension trial period and safety extension trial period. After completing the safety extension trial period (week 156), participants who received Norditropin were allocated to open-labelled Somapacitan dose 3 subcutaneously once weekly for the 208-week (up till week 364) long-term safety extension period. After week 364, participants received somapacitan dose 3 subcutaneously once weekly until somapacitan was available for prescription for children with GHD in their country or until August 2024, at the latest. Receives: Drug: somapacitan; Drug: Norditropin® FlexPro® pen |
| Cohort I somapacitan pooled | Experimental | Participants were randomized (1:1:1) to receive Somapacitan treatment (dose 1/dose 2/dose 3) subcutaneously once-weekly during the 26-week main trial period and the 26-week extension trial period. After completing the main and extension trial periods (week 52), all participants initially randomized to double-blinded Somapacitan received open-labelled Somapacitan dose 3 for the 104-week safety extension trial period. After completing the safety extension trial period (week 156), all participants in cohort I were allocated to open-labelled somapacitan dose 3 for the 208-week (up till week 364) long-term safety extension period. In extension after week 364 period participants received somapacitan dose 3 subcutaneously once weekly until somapacitan was available for prescription for children with GHD in their country or until August 2024, at the latest. Receives: Drug: somapacitan |
| Cohort II somapacitan previously treated | Experimental | Participant who was previously treated with GH (Growth hormone) prior to enrollment in the trial at week 156, received somapacitan dose 3 subcutaneously once weekly until it was available for prescription in participants' respective countries or until August 2024, at the latest. Receives: Drug: somapacitan |
| Cohort III somapacitan treatment naive | Experimental | Participants who were naive to treatment with GH prior to enrolment in the trial at week 156, received open-labelled somapacitan dose 3 subcutaneously once weekly until it was available for prescription in participants' respective countries or until August 2024, at the latest. Receives: Drug: somapacitan |
| Cohort III somapacitan previously treated | Experimental | Participants who were previously treated with GH prior to enrollment in the trial at week 156, received open-labelled somapacitan dose 3 subcutaneously once weekly until it was available for prescription in participants' respective countries or until August 2024, at the latest. Receives: Drug: somapacitan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Height Velocity (HV) (cm/Year) During the First 26 Weeks of Treatment, Measured as Standing Height With Stadiometer
Height velocity (HV) was derived from height measurements taken at baseline (week 0) and the week 26 as: HV = (height at 26 weeks visit- height at baseline) / (time from baseline to 26 weeks visit in years).
Measured: Baseline (week 0), week 26
Cohort II and Cohort III - Adverse Events Rate, Including Injection Site Reactions in Children With GHD.
This primary outcome measure was analysed by cohort using descriptive statistics. Adverse event per 100 patient years are presented in this outcome measure.
Measured: From week 156 up to week 364
Secondary outcomes (12)
Cohort I: Change in Height Standard Deviation Score (HSDS)
Change in height standard deviation score is presented from baseline (week 0) to end of the main trial period week 26 and end of extension trial period week 52. The formula to calculate HSDS is: HSDS = ((Height / M)**L-1) / (L*S). L: The gender and age-specific power in the Box-Cox transformation, M: The gender and age-specific median, S: The gender 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.
Measured: Baseline (Week 0), week 26, week 52
Cohort I: Change in Height Velocity Standard Deviation Score (HVSDS)
Change in height velocity standard deviation score is presented from baseline (week 0) to end of main trial period week 26 and end of extension trial period week 52. 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.
Measured: Baseline (Week 0), week 26, week 52
Cohort I: Adverse Events Rate, Including Injection Site Reactions
Adverse events per 100 patient years are presented. AEs with an onset after the first administration of trial product and up until 14 days after last trial drug administration for withdrawn participants, and with an onset after the first administration of trial product and up until visit 32 (week 364) or 14 days after last trial drug administration, which ever comes first, for all other participants, are analysed.
Measured: From week 0 Up to week 364
Cohort I: Occurrence of Anti-NNC0195-0092 and Anti-hGH Antibodies
Participants who developed anti-NNC0195-0092 and anti-hGH antibodies are reported in this outcome measure.
Measured: From week 0 Up to week 364
Change in Insulin-like Growth Factor I (IGF-I) Standard Deviation Score (SDS)
Change in IGF-I SDS is presented from baseline (week 0) to end of main trial period week 26 and end of extension period 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.
Measured: (Week 0), week 26, week 52
Change in Insulin-like Growth Factor Binding Protein 3 (IGFBP-3) Standard Deviation Score (SDS)
Change in IGFBP-3 SDS is presented from baseline (week 0) to end of main trial period week 26 and end of extension trial period 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.
Measured: Baseline (Week 0), week 26, week 52
Height Velocity (HV) (cm/Year) at Weeks 52 (Derived From Standing Height)
HV was derived from height measurements taken at baseline (week 0) and the week 52 as: HV = (height at 52 weeks visit-height at baseline) / (time from baseline to 52 weeks visit in years)
Measured: Baseline (week 0); week 52
Bone Age Progression vs. Chronological Age Ratio
The bone age vs. chronological age ratio is presented at week 52. X-Ray of left hand and wrist, central assessed according to Greulich \& Pyle atlas were taken.
Measured: At week 52
Serum Somapacitan Concentrations
Serum somapacitan concentrations are presented at week 52.
Measured: At week 52
Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer)
Change in Treatment Related Impact Measure from baseline (week 0) to week 26 and week 52 were assessed in children with growth hormone deficiency. This outcome measure was assessed using patient reported outcome (PRO) questionnaires with 3 domains, such as emotional well-being score, physical health score, social wellbeing core and total score. The total score was calculated by taking average of each domain. The scale range for each domain and total score was from 0-100 and a lower score indicates a better health state. TRIMCGHD-O was analysed using descriptive statistics.
Measured: Baseline (Week 0), week 26, week 52
Total Score of TB-CGHD-O (The Treatment Burden Measure - Child Growth Hormone Deficiency - Observer)
Total score of Treatment Burden Measure (observer) was assessed at week 26 and at week 52 in children with growth hormone deficiency. This outcome measure was assessed using PRO questionnaires. The scale range for total score was from 0-100 and a lower score indicates a better health state.
Measured: At week 26, at week 52
Total Score of TB-CGHD-P (The Treatment Burden Measure - Child Growth Hormone Deficiency - Parent/Guardian)
Total score of Treatment Burden Measure (parent/guardian) is reported at week 26 and at week 52 in children with growth hormone deficiency. This outcome measure was assessed using PRO questionnaires. The scale range for total score was from 0-100 and a lower score indicates a better health state.
Measured: At week 26, at week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Nov 2015 (estimated)
- Study start31 Mar 2016
- Primary completion26 Sep 2024
- Study completion26 Sep 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Growth Hormone DisorderStudies of the same condition, open and under way.
- More trials run by Novo Nordisk A/SThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 76 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novo Nordisk A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Global Clinical Registry (GCR, 1452), Study director, Novo Nordisk A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02616562
- Sponsor's protocol ID
- NN8640-4172
- Other IDs
- 2015-000531-32, U1111-1166-7062, 2015
- Status last verified
- Dec 2025
- First submitted
- 25 Nov 2015
- Last updated
- 16 Jan 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com
Publications
Savendahl L, Battelino T, Hojby Rasmussen M, Brod M, Rohrich S, Saenger P, Horikawa R. Weekly Somapacitan in GH Deficiency: 4-Year Efficacy, Safety, and Treatment/Disease Burden Results From REAL 3. J Clin Endocrinol Metab. 2023 Sep 18;108(10):2569-2578. doi: 10.1210/clinem/dgad183.
PubMed 36995872Savendahl L, Battelino T, Hojby Rasmussen M, Brod M, Saenger P, Horikawa R. Effective GH Replacement With Once-weekly Somapacitan vs Daily GH in Children with GHD: 3-year Results From REAL 3. J Clin Endocrinol Metab. 2022 Apr 19;107(5):1357-1367. doi: 10.1210/clinem/dgab928.
PubMed 34964458Savendahl L, Battelino T, Brod M, Hojby Rasmussen M, Horikawa R, Juul RV, Saenger P; REAL 3 study group. Once-Weekly Somapacitan vs Daily GH in Children With GH Deficiency: Results From a Randomized Phase 2 Trial. J Clin Endocrinol Metab. 2020 Apr 1;105(4):e1847-61. doi: 10.1210/clinem/dgz310.
PubMed 31917835