Somapacitan vs. Norditropin® for Small for Gestational Age (SGA) Children

Recruiting at 142 trial locations

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: Novo Nordisk A/S

Must not be taking: Systemic corticosteroids, Inhaled glucocorticoids

Disqualifiers: Hormonal deficiencies, ADHD, Malignancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 26 weeks

62 Participants Needed

Trial Summary

What is the purpose of this trial?

The study compares 2 medicines used for the treatment of children who are born small and who stayed small: somapacitan given once a week (a new medicine) and Norditropin® given once a day (the medicine doctors can already prescribe). Participants will either get somapacitan or Norditropin® - which treatment is decided by chance. Both participants and the study doctor will know which treatment the participants get. The study will last for 5 years. Participants will take either an injection once every week or once every day.

Will I have to stop taking my current medications?

The trial does not specify if you must stop taking your current medications, but it excludes children who need certain treatments that affect growth, like some ADHD medications or high doses of inhaled steroids. It's best to discuss your specific medications with the study doctor.

What data supports the effectiveness of the drug somapacitan for children born small for gestational age (SGA)?

Research shows that somapacitan, a long-acting growth hormone, is effective and safe for children with growth hormone deficiency, offering the convenience of once-weekly injections compared to daily ones. This could improve treatment adherence and outcomes for children born small for gestational age. ^{1 2 3 4 5}

Is somapacitan safe for use in humans?

Somapacitan has been studied for safety in both adults and children with growth hormone deficiency, showing similar safety profiles to daily growth hormone treatments like Norditropin®. In studies, it was generally well-tolerated, with no significant differences in safety compared to daily treatments. ^{1 2 3 4 6}

How does the drug somapacitan differ from other treatments for small for gestational age (SGA) children?

Somapacitan is unique because it is a long-acting growth hormone that only needs to be administered once a week, compared to the daily injections required by other growth hormone treatments like Norditropin. This can make the treatment more convenient and potentially improve adherence for children and their caregivers. ^{1 2 3 4 6}

Research Team

Clinical Reporting Anchor and Disclosure 1452

Principal Investigator

Novo Nordisk A/S

Eligibility Criteria

This trial is for pre-pubertal boys aged 2.5-11 years and girls aged 2.5-10 years who were born small, have stayed small, and haven't had growth hormone or IGF-I treatment. Kids with hormonal deficiencies, significant health issues affecting growth, recent steroid treatments, high-dose inhaled steroids, or other growth-affecting drugs can't join.

Inclusion Criteria

I am a boy aged 2.5 to 11 years with small testes.

I am a girl aged 2.5-10, born small for my age, with slow growth and no hormone treatments.

Exclusion Criteria

You have a medical condition that could affect your growth or make it difficult to measure your height accurately.

I have had cancer before, including brain tumors.

I am not taking medications like methylphenidate that affect growth.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either Somapacitan once a week or Norditropin® once a day for 26 weeks

26 weeks

Extension Period I

Continuation of the same treatment for an additional 26 weeks

26 weeks

Extension Period II

Participants switch to Somapacitan with dosage determined based on main phase data

Duration not specified

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Norditropin®
• somapacitan

Trial Overview The study compares somapacitan (a new medicine given weekly) to Norditropin® (an existing daily treatment) in children born small for gestational age. It's a randomized trial where both doctors and participants know which treatment is received over the course of one year with follow-up.

Participant Groups

Group I: Somapacitan 0.24 mg/kg/week Experimental Treatment 1 Intervention

Same treatment in main period (26 weeks) and extension period I (26 weeks). Dosage of Somapacitan during extension period II will be determined based on the data from main phase.

Group II: Somapacitan 0.20 mg/kg/week Experimental Treatment 1 Intervention

Same treatment in main period (26 weeks) and extension period I (26 weeks). Dosage of Somapacitan during extension period II will be determined based on the data from main phase.

Group III: Somapacitan 0.16 mg/kg/week Experimental Treatment 1 Intervention

Same treatment in main period (26 weeks) and extension period I (26 weeks). Dosage of Somapacitan during extension period II will be determined based on the data from main phase.

Group IV: Norditropin® 0.035 mg/kg/day Active Control 1 Intervention

Same treatment in main period (26 weeks) and extension period I (26 weeks). Participants will switch to Somapacitan during extension period II. Dosage of Somapacitan during extension period II will be determined based on the data from main phase.

Group V: Norditropin® 0.067 mg/kg/day Active Control 1 Intervention

Same treatment in main period (26 weeks) and extension period I (26 weeks). Participants will switch to Somapacitan during extension period II. Dosage of Somapacitan during extension period II will be determined based on the data from main phase.

Norditropin® is already approved in United States, European Union for the following indications:

Approved in United States as Norditropin for:

• Pediatric patients with growth failure due to inadequate secretion of endogenous growth hormone (GH)
• Short stature associated with Noonan syndrome
• Short stature associated with Turner syndrome
• Short stature born small for gestational age (SGA) with no catch-up growth by age 2 to 4 years
• Idiopathic Short Stature (ISS)
• Growth failure due to Prader-Willi syndrome (PWS)
• Replacement of endogenous GH in adults with growth hormone deficiency (GHD)

Approved in European Union as Norditropin for:

• Pediatric patients with growth failure due to inadequate secretion of endogenous growth hormone (GH)
• Short stature associated with Noonan syndrome
• Short stature associated with Turner syndrome
• Short stature born small for gestational age (SGA) with no catch-up growth by age 2 to 4 years
• Idiopathic Short Stature (ISS)
• Growth failure due to Prader-Willi syndrome (PWS)
• Replacement of endogenous GH in adults with growth hormone deficiency (GHD)

Who Is Running the Clinical Trial?

Novo Nordisk A/S

Lead Sponsor

Trials

1,578

Recruited

3,813,000+

Lars Fruergaard Jørgensen

Novo Nordisk A/S

Chief Executive Officer since 2017

MSc in Finance and Business Administration, Aarhus School of Business, Aarhus University, Denmark

Martin Holst Lange

Novo Nordisk A/S

Chief Medical Officer since 2021

MD from University of Copenhagen

Findings from Research

In a 26-week phase 3 trial involving 92 adults with growth hormone deficiency, once-weekly somapacitan was found to be well tolerated, with no significant safety issues or severe injection site reactions reported.

Patients using somapacitan reported significantly higher treatment satisfaction regarding convenience compared to those using the once-daily Norditropin, indicating a preference for the less frequent dosing schedule.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and convenience of once-weekly somapacitan in adult GH deficiency: a 26-week randomized, controlled trial. Johannsson, G., Feldt-Rasmussen, U., Håkonsson, IH., et al.[2019]

Somapacitan, a once-weekly growth hormone treatment for children with growth hormone deficiency, demonstrated similar efficacy and safety compared to daily growth hormone injections over a 4-year period, with 50 patients participating in the study.

Patients and their parents reported a significantly reduced treatment burden when switching to somapacitan, with 81.8% of parents preferring it over daily injections, highlighting the potential for improved quality of life.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Weekly Somapacitan in GH Deficiency: 4-Year Efficacy, Safety, and Treatment/Disease Burden Results From REAL 3. Sävendahl, L., Battelino, T., Højby Rasmussen, M., et al.[2023]

Norditropin(R) SimpleXx(TM), a liquid formulation of human growth hormone, significantly improved the ease of injection for 64% of children compared to the traditional freeze-dried version, which may enhance patient compliance in long-term treatment.

The new auto-insertion device, PenMate(TM), was found to be safe and easy to use, with 88% of children preferring it for future treatments, indicating a positive impact on the overall treatment experience.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Norditropin SimpleXx: a liquid human growth hormone formulation, a pen system and an auto-insertion device. Müller, J., Skakkebaek, NE., Jacobsen, BB., et al.[2022]

References

1. England pubmed.ncbi.nlm.nih.gov

Somapacitan in children born small for gestational age: a multi-centre, open-label, controlled phase 2 study. [2023]

2. England pubmed.ncbi.nlm.nih.gov

Safety and convenience of once-weekly somapacitan in adult GH deficiency: a 26-week randomized, controlled trial. [2019]

3. United States pubmed.ncbi.nlm.nih.gov

Weekly Somapacitan in GH Deficiency: 4-Year Efficacy, Safety, and Treatment/Disease Burden Results From REAL 3. [2023]

4. Switzerland pubmed.ncbi.nlm.nih.gov

Norditropin SimpleXx: a liquid human growth hormone formulation, a pen system and an auto-insertion device. [2022]

5. Spain pubmed.ncbi.nlm.nih.gov

Somapacitan: a long-acting growth hormone derivative for treatment of growth hormone deficiency. [2022]

6. England pubmed.ncbi.nlm.nih.gov

Similar safety and efficacy in previously treated adults with growth hormone deficiency randomized to once-weekly somapacitan or daily growth hormone. [2022]