A Trial Comparing the Effect and Safety of Once Weekly Dosing of Somapacitan With Daily Norditropin® in Children With Growth Hormone Deficiency
Overview
- Phase
- Phase 3
- Intervention
- Somapacitan
- Conditions
- Growth Hormone Deficiency in Children
- Sponsor
- Novo Nordisk A/S
- Enrollment
- 200
- Locations
- 267
- Primary Endpoint
- Height Velocity: In-trial Observation Period
- Status
- Completed
- Last Updated
- 3 months ago
Overview
Brief Summary
The study compares 2 medicines for children who do not have enough hormone to grow: somapacitan given once a week (a new medicine) and Norditropin® given once a day (the medicine doctors can already prescribe). Researchers will test to see how well somapacitan works. The study will also test if somapacitan is safe. Participants will either get somapacitan or Norditropin® - which treatment participants get, is decided by chance. Both participants and the study doctor will know which treatment participants get. The study will last for 4 years. Participants will attend 19 clinic visits and have 1 phone call with the study doctor.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Prepubertal children: a) Boys: Age more than or equal to 2 years and 26 weeks and less than 11.0 years at screening. Testis volume less than 4 ml. b) Girls: Age more than or equal to 2 years and 26 weeks and less than 10.0 years at screening. Tanner stage 1 for breast development (no palpable glandular breast tissue)
- •Confirmed diagnosis of growth hormone deficiency determined by two different growth hormone stimulation tests performed within 12 months prior to randomisation, defined as a peak growth hormone level of less than or equal to 10.0 ng/ml using the World Health Organisation (WHO) International Somatropin 98/574 standard
- •Impaired height defined as at least 2.0 standard deviations below the mean height for chronological age and gender at screening according to the standards of Center for Disease Control and Prevention
- •Impaired height velocity, defined as annualised height velocity below the 25th percentile for chronological age and gender according to the standards of Prader calculated over a time span of minimum 6 months and maximum 18 months prior to screening
- •Insulin-like Growth Factor-I (IGF-I) less than -1.0 SDS at screening, compared to age and gender normalized range measured at central laboratory
- •No prior exposure to growth hormone therapy or IGF-I treatment
Exclusion Criteria
- •Any known or suspected clinically significant abnormality likely to affect growth or the ability to evaluate growth with standing height measurements
- •Current inflammatory diseases requiring systemic corticosteroid treatment for longer than 2 consecutive weeks within the last 3 months prior to screening
- •Children requiring inhaled glucocorticoid therapy at a dose of greater than 400 μg/day of inhaled budesonide or equivalents for longer than 4 consecutive weeks within the last 12 months prior to screening
- •Diagnosis of attention deficit hyperactivity disorder
- •Concomitant administration of other treatments that may have an effect on growth, e.g. but not limited to methylphenidate for treatment of attention deficit hyperactivity disorder
- •Prior history or presence of malignancy including intracranial tumours
Arms & Interventions
Somapacitan weekly
Participants will receive somapacitan weekly for 52 weeks (main trial period). Participants completing the main trial period in both the treatment arms ('Somapacitan weekly' and 'Norditropin® daily') will receive somapacitan weekly for 3 years (extension trial period).
Intervention: Somapacitan
Norditropin® daily
Participants will receive Norditropin® daily for 52 weeks (main trial period).
Intervention: Norditropin®
Outcomes
Primary Outcomes
Height Velocity: In-trial Observation Period
Time Frame: From baseline (week 0) to visit 7 (week 52)
Height velocity (HV) was derived from height measurements taken at baseline and Week 52 visit as: HV = (height at 52 weeks visit - height at baseline)/(time from baseline to 52 weeks visit in years). Data is reported for 'in-trial' observation period. In-trial observation period: from first administration and up until visit 7 or last trial contact, whichever comes first.
Height Velocity: On-treatment Observation Period
Time Frame: From baseline (week 0) to visit 7 (week 52)
Height velocity was derived from height measurements taken at baseline and Week 52 visit as: HV = (height at 52 weeks visit - height at baseline)/(time from baseline to 52 weeks visit in years). Data is reported for 'on-treatment' observation period. On-treatment observation period: from first administration and up until last trial contact, visit 7 or 14 days after last administration, whichever comes first.
Secondary Outcomes
- Change in HbA1c at Week 104(Baseline (week -2), week 104)
- Change in Homeostatic Model Assessment Steady State Beta Cell Function (HOMA-B) at Week 52(Baseline (week -2), week 52)
- Change in Bone Age(Baseline (week -2), week 52)
- Change in Height Velocity Standard Deviation Score (HV SDS)(Baseline (week 0), week 52)
- Change in Fasting Plasma Glucose (FPG) at Week 52(Baseline (week -2), week 52)
- Change in Height Standard Deviation Score (HSDS)(Baseline (week 0), week 52)
- Change in FPG at Week 156(Baseline (week -2), week 156)
- Change in HOMA-B at Week 104(Baseline (week -2), week 104)
- Change in HOMA-B at Week 208(Baseline (week -2), week 208)
- Change in Homeostatic Model Assessment Insulin Resistance (HOMA-IR) at Week 52(Baseline (week -2), week 52)
- Change in HOMA-IR at Week 104(Baseline (week -2), week 104)
- Change in HOMA-IR at Week 156(Baseline (week -2), week 156)
- Change in FPG at Week 104(Baseline (week -2), week 104)
- Change in FPG at Week 208(Baseline (week -2), week 208)
- Change in HOMA-B at Week 156(Baseline (week -2), week 156)
- Change in HOMA-IR at Week 208(Baseline (week -2), week 208)
- Change in Glycated Haemoglobin (HbA1c) at Week 52(Baseline (week -2), week 52)
- Change in HbA1c at Week 156(Baseline (week -2), week 156)
- Change in HbA1c at Week 208(Baseline (week -2), week 208)
- Change in Insulin-like Growth Factor I (IGF-I) Standard Deviation Score (SDS) at Week 52(Baseline (week 0), week 52)
- Change in IGF-I SDS at Week 104(Baseline (week 0), week 104)
- Change in IGF-I SDS at Week 156(Baseline (week 0), week 156)
- Change in IGF-I SDS at Week 208(Baseline (week 0), week 208)
- Change in Insulin-like Growth Factor Binding Protein 3 (IGFBP-3) Standard Deviation Score (SDS) at Week 52(Baseline (week 0), week 52)
- Change in IGFBP-3 SDS at Week 104(Baseline (week 0), week 104)
- Change in IGFBP-3 SDS at Week 156(Baseline (week 0), week 156)
- Change in IGFBP-3 SDS at Week 208(Baseline (week 0), week 208)