Growth Hormone Disorder Clinical Trial
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 (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.
Status | Recruiting |
Enrollment | 74 |
Est. completion date | September 27, 2024 |
Est. primary completion date | August 26, 2024 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 30 Months to 10 Years |
Eligibility | 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 9.0 years and below or equal to 17.0 years at screening. - Boys: Above 10.0 years and below or equal to 17.0 years at screening. - Confirmed diagnosis of GHD 1. 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. 2. 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, including but not limited to methylphenidate for treatment of attention deficit hyperactivity disorder (ADHD) - Prior history or presence of malignancy and/or intracranial tumour |
Country | Name | City | State |
---|---|---|---|
Austria | Med. Univ. Graz -Klinische Abteilung f. Allgemeine Pädiatrie | Graz | |
Austria | Kepler Universitätsklinikum GmbH - Med Campus IV (vorm.LFKK) | Linz | Upper Austria |
Austria | LKH Salzburg- Univ. Klinik f. Kinder- und Jugendheilkunde | Salzburg | |
Austria | LKH St. Poelten, Kinder-und Jugendheilkunde | St. Poelten | |
Austria | Landeskrankenhaus Villach | Villach | |
Austria | Salzkammergut-Klinikum Vöcklabruck | Vöcklabruck | |
Belgium | UZ Brussel | Brussel | |
Belgium | Cliniques Universitaires Saint-Luc - Serv. Pédiatrie | Bruxelles | |
Belgium | UZ Leuven - Kindergeneeskunde | Leuven | |
Belgium | CHU de Liège, site N.-D. des Bruyères | Liège | |
Brazil | Serviço de Endocrinologia e Metabologia do HC-UFPR | Curitiba | Paraná |
Brazil | Hospital São Lucas - PUC/RS | Porto Alegre | Rio Grande Do Sul |
Brazil | CPQuali Pesquisa Clínica Ltda | São Paulo | Sao Paulo |
France | Centre Hospitalier Régional Universitaire d' Angers | ANGERS cedex 09 | |
France | CHU Pellegrin | Bordeaux | |
France | Clinique Médicale Pédiatrique | Nantes | |
France | Hôpital Necker | Paris | |
France | HOPITAL SUD de RENNES | Rennes | |
France | Hôpital des Enfants | Toulouse cedex 9 | |
Germany | Endokrinologikum Frankfurt | Frankfurt am Main | |
Germany | Universitätsklinikum Ulm für Kinder- und Jugendmedizin | Ulm | |
India | Amrita Institute Of Medical Sciences & Research Centre | Kochi | Kerala |
India | All India Institute of Medical Sciences | New Dehli | New Delhi |
India | Jehangir Clinical Development Centre | Pune | Maharashtra |
Israel | Soroka Medical Center - Pediatric Endocrinology | Beer Sheva | |
Israel | Rambam Medical Center Children A Dept. | Haifa | |
Israel | Department of Pediatrics , Meir Medical Center | Kfar Saba | |
Israel | Endrocrinology & DM Schneider MC | Petah Tikva | |
Israel | Sheba Medical Center Pediatric Endocrinology | Tel Hashomer | |
Japan | Kurume University Hospital, Pediatrics | Fukuoka | |
Japan | Kyushu Univ. HP, Maternity & Perinatal Care Center | Fukuoka | |
Japan | St. Marianna University School of Medicine Hospital_Pediatrics | Kanagawa | |
Japan | St. Marianna University School of Medicine Hospital_Pediatrics | Kanagawa | |
Japan | Univ.HP, Kyoto Pref Univ of Medicine, Dept. of Pediatrics | Kyoto | |
Japan | JCHO Osaka HP, Pediatric | Osaka | |
Japan | Osaka City General Hospital, Pediatric Endocrinology and Me | Osaka | |
Japan | Osaka Women's and Children's Hospital | Osaka | |
Japan | National Center for Child Health and Dev, Endo and Metabo | Tokyo | |
Japan | Tokyo Medical and Dental University Hospital | Tokyo | |
Japan | Tokyo Medical and Dental University Hospital | Tokyo | |
Slovenia | PeK - Dept. of Paediatric Endocrinology, Diabetes and Metabolism | Ljubljana | |
Sweden | Astrid Lindgrens Barnsjukhus | Stockholm | |
Sweden | Barn och ungdomskliniken Västerbotten | Umeå | |
Turkey | Cukurova Universitesi Tip Fakultesi_Istanbul | Adana | |
Turkey | Hacettepe University Medical Faculty | Ankara | |
Turkey | I.U Istanbul Medical Faculty | Istanbul | |
Turkey | Marmara University Medical Faculty | Istanbul | |
Ukraine | Ivano-Frankivsk Regional Clinical Children Hospital | Ivano-Frankivsk | |
Ukraine | Institute of Endocrinology and Metabolism of AMSU | Kyiv | |
United States | Rocky Mt Ped and Endo | Centennial | Colorado |
United States | CCHMC_Cinc | Cincinnati | Ohio |
United States | NYU Langone Hospital-LI | Mineola | New York |
United States | University of Minnesota_Minneapolis_2 | Minneapolis | Minnesota |
United States | Goryeb Children's Hospital | Morristown | New Jersey |
United States | Rutgers-Rwjms | New Brunswick | New Jersey |
United States | The Regents of the Univ of CA | San Diego | California |
United States | MultiCare Inst for Res & Innov | Tacoma | Washington |
United States | Nemours/AI duPont Hosp-Chld | Wilmington | Delaware |
Lead Sponsor | Collaborator |
---|---|
Novo Nordisk A/S |
United States, Austria, Belgium, Brazil, France, Germany, India, Israel, Japan, Slovenia, Sweden, Turkey, Ukraine,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Cohort I: Height velocity (HV) during the first 26 weeks of treatment, measured as standing height with stadiometer | cm/year | Week 0-26 | |
Primary | Cohort II and III: Incidence of adverse events, including injection site reactions, in children with GHD | Number of events | During 208 weeks | |
Secondary | Change in height standard deviation score (SDS) | Typically -10 to +10 | Week 0-26 | |
Secondary | Change in height standard deviation score (SDS) | Typically -10 to +10 | Week 0-52 | |
Secondary | Change in height standard deviation score (SDS) | Typically -10 to +10 | Week 26-52 | |
Secondary | Change in HV (height velocity) SDS | 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) | Week 0-26 | |
Secondary | Change in HV (height velocity) SDS | 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) | Week 0-52 | |
Secondary | Insulin-like growth factor 1 (IGF-I) SDS | Typically -10 to +10 | Week 0-26 | |
Secondary | Insulin-like growth factor 1 (IGF-I) SDS | Typically -10 to +10 | Week 0-52 | |
Secondary | Insulin-like growth factor 1 (IGF-I) SDS | Typically -10 to +10 | Week 26-52 | |
Secondary | Insulin-like growth factor binding protein 3 (IGFBP-3) SDS | Typically -10 to +10 | Week 0-26 | |
Secondary | Insulin-like growth factor binding protein 3 (IGFBP-3) SDS | Typically -10 to +10 | Week 0-52 | |
Secondary | Insulin-like growth factor binding protein 3 (IGFBP-3) SDS | Typically -10 to +10 | Week 26-52 | |
Secondary | Height velocity | cm/year, derived from standing height from baseline (week 0) to week 52 | Week 52 | |
Secondary | Bone age | X-Ray of left hand and wrist, central assessed according to Greulich & Pyle atlas progression vs. chronological age | Week 52 | |
Secondary | Serum NNC0195-0092 (somapacitan) concentrations | ng/mL | Week 52 | |
Secondary | 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) | The scores range from 0-100. A lower score indicates a better health state. | Week 0-26 | |
Secondary | 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) | The scores range from 0-100. A lower score indicates a better health state. | Week 0-52 | |
Secondary | Total score of The Treatment Burden Measure - Child Growth Hormone Deficiency - Observer (TB-CGHD-O) | The scores range from 0-100. A lower score indicates a better health state. | Week 26 | |
Secondary | Total score of The Treatment Burden Measure - Child Growth Hormone Deficiency - Observer (TB-CGHD-O) | The scores range from 0-100. A lower score indicates a better health state. | Week 52 | |
Secondary | Total score of The Treatment Burden Measure - Child Growth Hormone Deficiency - Parent/Guardian (TB-CGHD-P) | The scores range from 0-100. A lower score indicates a better health state. | Week 26 | |
Secondary | Total score of The Treatment Burden Measure - Child Growth Hormone Deficiency - Parent/Guardian (TB-CGHD-P) | The scores range from 0-100. A lower score indicates a better health state. | Week 52 | |
Secondary | Incidence of adverse events, including injection site reactions | Number of events | Week 364 | |
Secondary | Occurrence of anti-NNC0195-0092 (somapacitan) antibodies | Yes/no | Week 364 | |
Secondary | Occurrence of anti-hGH antibodies | Yes/no | Week 364 |
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
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