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

NCT ID: NCT02616562

Last Updated: 2026-01-16

Study Results

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Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

76 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-03-31

Study Completion Date

2024-09-26

Brief Summary

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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.

Detailed Description

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Conditions

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Growth Hormone Disorder Growth Hormone Deficiency in Children

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

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

Study Groups

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Cohort I Norditropin/somapacitan

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.

Group Type EXPERIMENTAL

somapacitan

Intervention Type DRUG

Administered subcutaneously (s.c., under the skin) once-weekly.

Norditropin® FlexPro® pen

Intervention Type DRUG

Administered subcutaneously (s.c., under the skin) once daily.

Cohort I somapacitan pooled

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.

Group Type EXPERIMENTAL

somapacitan

Intervention Type DRUG

Administered subcutaneously (s.c., under the skin) once-weekly.

Cohort II somapacitan previously treated

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.

Group Type EXPERIMENTAL

somapacitan

Intervention Type DRUG

Administered subcutaneously (s.c., under the skin) once-weekly.

Cohort III somapacitan treatment naive

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.

Group Type EXPERIMENTAL

somapacitan

Intervention Type DRUG

Administered subcutaneously (s.c., under the skin) once-weekly.

Cohort III somapacitan previously treated

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.

Group Type EXPERIMENTAL

somapacitan

Intervention Type DRUG

Administered subcutaneously (s.c., under the skin) once-weekly.

Interventions

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somapacitan

Administered subcutaneously (s.c., under the skin) once-weekly.

Intervention Type DRUG

Norditropin® FlexPro® pen

Administered subcutaneously (s.c., under the skin) once daily.

Intervention Type DRUG

Other Intervention Names

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NNC0195-0092

Eligibility Criteria

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Inclusion Criteria

* 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

Exclusion Criteria

* 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
Minimum Eligible Age

2 Years

Maximum Eligible Age

10 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novo Nordisk A/S

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Global Clinical Registry (GCR, 1452)

Role: STUDY_DIRECTOR

Novo Nordisk A/S

Locations

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Children's of Alabama

Birmingham, Alabama, United States

Site Status

Children's Hospital Los Angeles - Endocrinology

Los Angeles, California, United States

Site Status

Mattel Children's Hospital at UCLA

Los Angeles, California, United States

Site Status

Valley Children's Hospital

Madera, California, United States

Site Status

Novo Nordisk Clinical Trial Call Center

San Diego, California, United States

Site Status

The Regents of the Univ of CA

San Diego, California, United States

Site Status

Rocky Mt Ped and Endo

Centennial, Colorado, United States

Site Status

Ped Endo Assoc PC-G.V

Greenwood Village, Colorado, United States

Site Status

Nemours/AI duPont Hosp-Chld

Wilmington, Delaware, United States

Site Status

The Endocrine Center

Pembroke Pines, Florida, United States

Site Status

Childrens Hospital of Chicago

Chicago, Illinois, United States

Site Status

Riley Hospital For Children

Indianapolis, Indiana, United States

Site Status

Novo Nordisk Clinical Trial Call Center

Minneapolis, Minnesota, United States

Site Status

University of Minnesota_Minneapolis_2

Minneapolis, Minnesota, United States

Site Status

Goryeb Children's Hospital

Morristown, New Jersey, United States

Site Status

Novo Nordisk Clinical Trial Call Center

Morristown, New Jersey, United States

Site Status

Rutgers-Rwjms

New Brunswick, New Jersey, United States

Site Status

UBMD Peds-Div of Endo/Diabetes

Buffalo, New York, United States

Site Status

Novo Nordisk Clinical Trial Call Center

Mineola, New York, United States

Site Status

NYU Langone Hospital-LI

Mineola, New York, United States

Site Status

CCHMC_Cinc

Cincinnati, Ohio, United States

Site Status

Novo Nordisk Clinical Trial Call Center

Cincinnati, Ohio, United States

Site Status

University Of Oklahoma-Tulsa

Tulsa, Oklahoma, United States

Site Status

Dell Pediatric Research Institute

Austin, Texas, United States

Site Status

Endocrine Associates Of Dallas

Plano, Texas, United States

Site Status

Children's Hsptl Of The Kings

Norfolk, Virginia, United States

Site Status

MultiCare Inst for Res & Innov

Tacoma, Washington, United States

Site Status

Kepler Universitätsklinikum GmbH - Med Campus IV (vorm.LFKK)

Linz, Upper Austria, Austria

Site Status

Med. Univ. Graz -Klinische Abteilung f. Allgemeine Pädiatrie

Graz, , Austria

Site Status

Graz, , Austria

Site Status

LKH Salzburg- Univ. Klinik f. Kinder- und Jugendheilkunde

Salzburg, , Austria

Site Status

LKH St. Poelten, Kinder-und Jugendheilkunde

Sankt Pölten, , Austria

Site Status

Landeskrankenhaus Villach

Villach, , Austria

Site Status

Salzkammergut-Klinikum Vöcklabruck

Vöcklabruck, , Austria

Site Status

UZ Brussel

Brussels, , Belgium

Site Status

Brussels, , Belgium

Site Status

Cliniques Universitaires Saint-Luc - Serv. Pédiatrie

Brussels, , Belgium

Site Status

UZ Leuven - Kindergeneeskunde

Leuven, , Belgium

Site Status

CHU de Liège, site N.-D. des Bruyères

Liège, , Belgium

Site Status

Serviço de Endocrinologia e Metabologia do HC-UFPR

Curitiba, Paraná, Brazil

Site Status

Hospital São Lucas - PUC/RS

Porto Alegre, Rio Grande do Sul, Brazil

Site Status

CPQuali Pesquisa Clínica Ltda

São Paulo, São Paulo, Brazil

Site Status

São Paulo, São Paulo, Brazil

Site Status

The Hospital for Sick Children

Toronto, Ontario, Canada

Site Status

Centre Hospitalier Universitaire D'Angers-2

Angers, , France

Site Status

Centre Hospitalier Universitaire de Bordeaux-Hopital Pellegrin

Bordeaux, , France

Site Status

Bordeaux, , France

Site Status

Centre Hospitalier Universitaire de Nantes-Hopital Enfant-Adolescent

Nantes, , France

Site Status

Ap-Hp-Hopital Necker

Paris, , France

Site Status

HOPITAL SUD de RENNES

Rennes, , France

Site Status

HOPITAL DES ENFANTS-HOPITAL PAULE DE VIGUIER - Pharmacie

Toulouse, , France

Site Status

Frankfurt, , Germany

Site Status

Endokrinologikum Frankfurt

Frankfurt am Main, , Germany

Site Status

Uniklinik Ulm - Dt. Zentrum für Kinder- und Jugendgesundheit (DZKJ)

Ulm, , Germany

Site Status

Amrita Institute Of Medical Sciences & Research Centre

Kochi, Kerala, India

Site Status

Jehangir Clinical Development Centre

Pune, Maharashtra, India

Site Status

All India Institute of Medical Sciences

New Dehli, New Delhi, India

Site Status

New Dehli, New Delhi, India

Site Status

Soroka MC - Pediatric Endocrinology

Beersheba, , Israel

Site Status

Rambam MC - Department of Pediatrics A

Haifa, , Israel

Site Status

Meir MC - Department of Paediatrics

Kfar Saba, , Israel

Site Status

Kfar Saba, , Israel

Site Status

Schneider MC - Endrocrinology and Diabetes

Petah Tikva, , Israel

Site Status

Sheba MC - Pediatric endocrinology and adolescent diabetes clinics

Tel Litwinsky, , Israel

Site Status

Kyushu Univ. HP, Maternity & Perinatal Care Center

Fukuoka, , Japan

Site Status

Kurume University Hospital, Pediatrics

Fukuoka, , Japan

Site Status

St. Marianna University School of Medicine Hospital_Pediatrics

Kanagawa, , Japan

Site Status

Univ.HP, Kyoto Pref Univ of Medicine, Dept. of Pediatrics

Kyoto, , Japan

Site Status

Osaka City General Hospital, Pediatric Endocrinology and Me

Osaka, , Japan

Site Status

JCHO Osaka Hospital_Pediatric

Osaka, , Japan

Site Status

Osaka Women's and Children's Hospital

Osaka, , Japan

Site Status

Institute of Science Tokyo Hospital_Pediatrics

Tokyo, , Japan

Site Status

Tokyo Medical and Dental University Hospital

Tokyo, , Japan

Site Status

National Center for Child Health and Dev, Endo and Metabo

Tokyo, , Japan

Site Status

Tokyo, , Japan

Site Status

University Children's Hospital

Ljubljana, , Slovenia

Site Status

Ljubljana, , Slovenia

Site Status

Astrid Lindgrens Barnsjukhus

Stockholm, , Sweden

Site Status

Stockholm, , Sweden

Site Status

Barn och ungdomscentrum Västerbotten

Umeå, , Sweden

Site Status

Çukurova Üniversitesi Tıp Fakültesi Balcalı Hastanesi

Adana, , Turkey (Türkiye)

Site Status

Hacettepe Üniversitesi Hastanesi- Endokrinoloji

Ankara, , Turkey (Türkiye)

Site Status

I.U Istanbul Medical Faculty

Istanbul, , Turkey (Türkiye)

Site Status

Istanbul, , Turkey (Türkiye)

Site Status

Marmara University Medical Faculty

Istanbul, , Turkey (Türkiye)

Site Status

Marmara Üniversitesi Pendik EAH- Başıbüyük Yerleşkesi- Kardiyoloji

Istanbul, , Turkey (Türkiye)

Site Status

Ivano-Frankivsk Regional Clinical Children Hospital - Endocrinology dept.

Ivano-Frankivsk, , Ukraine

Site Status

Kiev, , Ukraine

Site Status

Komisarenko Institute of Endocrinology and Metabolism of NAMSU - Department of paediatric endocrine pathology

Kyiv, , Ukraine

Site Status

Countries

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United States Austria Belgium Brazil Canada France Germany India Israel Japan Slovenia Sweden Turkey (Türkiye) Ukraine

References

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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.

Reference Type DERIVED
PMID: 36995872 (View on PubMed)

Savendahl 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.

Reference Type DERIVED
PMID: 34964458 (View on PubMed)

Savendahl 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.

Reference Type DERIVED
PMID: 31917835 (View on PubMed)

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Related Links

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http://novonordisk-trials.com

Clinical Trials at Novo Nordisk

Other Identifiers

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2015-000531-32

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

U1111-1166-7062

Identifier Type: OTHER

Identifier Source: secondary_id

NN8640-4172

Identifier Type: -

Identifier Source: org_study_id

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