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
- Conditions
- Growth Hormone DisorderGrowth Hormone Deficiency in Children
- Interventions
- Registration Number
- NCT02616562
- Lead Sponsor
- Novo Nordisk A/S
- Brief 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.
The main trial period will consist of 26 weeks of treatment, followed by a 26 week extension period.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 74
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Blinded NNC0195-0092 (0.04 mg/kg/week) somapacitan 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. Blinded NNC0195-0092 (0.04 mg/kg/week) NNC0195-0092 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. Blinded NNC0195-0092 (0.08 mg/kg/week) somapacitan 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. Blinded NNC0195-0092 (0.08 mg/kg/week) NNC0195-0092 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. Blinded NNC0195-0092 (0.16 mg/kg/week) somapacitan Participants receive the same dose (0.16 mg/kg/week) of NNC0195-0092 (somapacitan) during all 4 trial periods. Blinded NNC0195-0092 (0.16 mg/kg/week) NNC0195-0092 Participants receive the same dose (0.16 mg/kg/week) of NNC0195-0092 (somapacitan) during all 4 trial periods. Open labelled daily Norditropin® (0.034 mg/kg/day) somapacitan 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. Open labelled daily Norditropin® (0.034 mg/kg/day) Norditropin® FlexPro® pen 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.
- Primary Outcome Measures
Name Time Method Cohort I: Height velocity (HV) during the first 26 weeks of treatment, measured as standing height with stadiometer Week 0-26 cm/year
Height velocity (HV) (cm/year) during the first 26 weeks of treatment, measured as standing height with stadiometer Week 0-26 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
Name Time Method Insulin-like growth factor binding protein 3 (IGFBP-3) SDS Week 26-52 Typically -10 to +10
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 0-52 The scores range from 0-100. A lower score indicates a better health state.
Change in height standard deviation score (SDS) Week 26-52 Typically -10 to +10
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 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
Incidence of adverse events, including injection site reactions At week 53 Occurrence of anti-hGH antibodies Week 364 Yes/no
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 52 The scores range from 0-100. A lower score indicates a better health state.
Occurrence of anti-NNC0195-0092 (somapacitan) antibodies Week 364 Yes/no
Occurrence of anti-NNC0195-0092 and anti-hGH antibodies At week 53
Trial Locations
- Locations (38)
Novo Nordisk Clinical Trial Call Center
🇺🇸Cincinnati, Ohio, United States
Nemours/AI duPont Hosp-Chld
🇺🇸Wilmington, Delaware, United States
Goryeb Children's Hospital
🇺🇸Morristown, New Jersey, United States
Rutgers-Rwjms
🇺🇸New Brunswick, New Jersey, United States
NYU Langone Hospital-LI
🇺🇸Mineola, New York, United States
CCHMC_Cinc
🇺🇸Cincinnati, Ohio, United States
Kepler Universitätsklinikum GmbH - Med Campus IV (vorm.LFKK)
🇦🇹Linz, Upper Austria, Austria
Landeskrankenhaus Villach
🇦🇹Villach, Austria
CPQuali Pesquisa Clínica Ltda
🇧🇷São Paulo, Sao Paulo, Brazil
Centre Hospitalier Régional Universitaire d' Angers
🇫🇷ANGERS cedex 09, France
Scroll for more (28 remaining)Novo Nordisk Clinical Trial Call Center🇺🇸Cincinnati, Ohio, United States