A Study Comparing the Effect and Safety of Once Weekly Dosing of Somapacitan With Daily Norditropin® as Well as Evaluating Long-term Safety of Somapacitan in a Basket Study Design in Children With Short Stature Either Born Small for Gestational Age or With Turner Syndrome, Noonan Syndrome, or Idiopathic Short Stature

Novo Nordisk A/S375 sites in 6 countries412 target enrollmentAugust 10, 2022

ConditionsSGA, Turner Syndrome, Noonan Syndrome, ISS

InterventionsSomapacitan Norditropin®

DrugsSomapacitan Norditropin®

Overview

Phase: Phase 3
Intervention: Somapacitan
Conditions: SGA, Turner Syndrome, Noonan Syndrome, ISS
Sponsor: Novo Nordisk A/S
Enrollment: 412
Locations: 375
Primary Endpoint: Height velocity reported separately for small for gestational age (SGA), Turner syndrome (TS), Noonan syndrome (NS) and idiopathic short stature (ISS)
Status: Active, Not Recruiting
Last Updated: 8 days ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

The study compares two medicines for treatment of children born small and who stay small, or with Turner Syndrome, Noonan Syndrome, or idiopathic short stature. The purpose of the study is to see how well treatment with somapacitan works compared to treatment with Norditropin®. Somapacitan is a new medicine, and Norditropin® is a medicine doctors can already prescribe in some countries. The study will last for upto 5.5 years. The participants will either get somapacitan once a week up to 5.5 years or Norditropin® once a day for 1 year followed by somapacitan once a week for up to 4.5 years. Which treatment the participants get is decided by chance.

Registry

clinicaltrials.gov

Start Date

August 10, 2022

End Date

October 29, 2027

Last Updated

8 days ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Novo Nordisk A/S

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Informed consent of parent or legally acceptable representative of participant and child assent, as age appropriate must be obtained before any study-related activities. Study-related activities are any procedures that are carried out as part of the study, including activities to determine suitability for the study.
•No prior exposure to growth promoting therapy, including but not limited to growth hormone, IGF-I and ghrelin analogues.
•Applicable to children with SGA:
•Born small for gestational age (birth length below -2 SDS OR birth weight below -2 SDS OR both) (according to national standards).
•Prepubertal children:
•Age above or equal to 2 years and 26 weeks and below 11.0 years at screening.
•Testis volume below 4 mL
•Age above or equal to 2 years and 26 weeks and below 10.0 years at screening.
•Tanner stage 1 for breast development: No palpable glandular breast tissue)
•Impaired height defined as at least 2.5 standard deviations below the mean height for chronological age and sex at screening according to the standards of Centers for Disease Control and Prevention.

Exclusion Criteria

•Known or suspected hypersensitivity to study intervention(s) or related products.
•Previous randomization into same sub-study in this study.
•Receipt of any investigational medicinal product within 3 months before screening or participation in another clinical study at the time of randomization.
•Children with suspected or confirmed growth hormone deficiency according to local practice.
•laboratory of
•fasting plasma glucose above or equal to 126 mg/dL (7.0 mmol/L) or
•HbA1c above or equal to 6.5%.
•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 greater than 400 µg/day of inhaled budesonide or equivalent (i.e., 250 µg/day for fluticasone propionate) for longer than 4 consecutive weeks within the last 12 months prior to screening.
•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 (ADHD).

Arms & Interventions

Somapacitan

Participants will receive Somapacitan for 273 weeks

Intervention: Somapacitan

Norditropin®

Participants will receive Norditropin® for 52 weeks (main treatment period) and Somapacitan for 221 weeks (extension period)

Intervention: Norditropin®

Outcomes

Primary Outcomes

Height velocity reported separately for small for gestational age (SGA), Turner syndrome (TS), Noonan syndrome (NS) and idiopathic short stature (ISS)

Time Frame: From baseline (week 0) to visit 7 (week 52)

Measured in centimeter per year (cm/year)

Secondary Outcomes

Change in bone age reported separately for SGA, TS and NS(From baseline (week 0) to visit 7 (week 52))
Change in insulin-like growth factor 1 (IGF-1) SDS reported separately for SGA, TS, NA and ISS(From baseline (week 0) to visit 7 (week 52).)
Change in glycated haemoglobin (HbA1c) reported separately for SGA, TS, NS and ISS(From screening (visit 1) to visit 15 (week 156))
Change in Height Velocity SDS reported separately for SGA, TS, NS and ISS(From baseline (week 0) to visit 7 (week 52))
Change in fasting plasma glucose reported separately for SGA, TS, NS and ISS(From screening (visit 1) to visit 15 (week 156))
Change in homeostatic model assessment-B (HOMA-B) reported separately for SGA, TS, NS and ISS(From screening (visit 1) to visit 15 (week 156))
Change in homeostatic model assessment of insulin resistance (HOMA-IR) reported separately for SGA, TS, NS and ISS(From screening (visit 1) to visit 15 (week 156))
Change in Height standard deviation scores (SDS) reported separately for SGA, TS, NS and ISS(From baseline (week 0) to visit 7 (week 52))
Change in bone age for ISS(From screening (visit 1) to visit 7 (week 52))
Change in insulin-like growth factor binding protein-3 (IGFBP-3) SDS reported separately for SGA, TS, NA and ISS(From baseline (week 0) to visit 7 (week 52).)
Change in homeostatic model assessment of insulin resistance (HOMAIR) reported separately for SGA, TS, NS and ISS(From screening (visit 1) to visit 7 (week 52))
Weekly average somapacitan concentration (Cavg) based on population PK analysis(From visit 3 (week 4) to visit 7 (week 52))
Change in fasting plasma glucose reported separately for SGA, TS, NS and ISS(From screening (visit 1) to visit 7 (week 52))
Change in homeostatic model assessment-B (HOMA-B) reported separately for SGA, TS, NS and ISS(From screening (visit 1) to visit 7 (week 52))
Change in glycated haemoglobin (HbA1c) reported separately for SGA, TS, NS and ISS(From screening (visit 1) to visit 7 (week 52))