Somapacitan for Growth Hormone Deficiency in Children

(REAL 9 Trial)

Recruiting at 34 trial locations

Overseen By Novo Nordisk

Age: < 65

Sex: Any

Trial Phase: Phase 3

Sponsor: Novo Nordisk A/S

Must not be taking: Systemic corticosteroids

Disqualifiers: Diabetes, Malignancy, Skeletal dysplasia, others

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

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 26 weeks

48 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing somapacitan, a new growth hormone medicine, in children who have low levels of growth hormone. These children include those born small for their age or with conditions like Turner syndrome, Noonan syndrome, or idiopathic short stature. Somapacitan is given as an injection to help them grow better. Somapacitan is a long-acting growth hormone derivative developed for periodic administration, already approved for use in adult GH deficiency in the USA and Japan.

Do I need to stop my current medications to join the trial?

The trial protocol does not specify if you need to stop taking your current medications. However, if you are on systemic corticosteroids or high-dose inhaled glucocorticoids, you may not be eligible. It's best to discuss your specific medications with the study doctor.

Do I have to stop taking my current medications for the trial?

The trial information does not specify if you need to stop taking your current medications. However, if you are on certain medications like systemic corticosteroids or high doses of inhaled glucocorticoids, you may not be eligible to participate. It's best to discuss your specific medications with the study doctor.

What data supports the idea that Somapacitan for Growth Hormone Deficiency in Children is an effective drug?

The available research shows that Somapacitan, a once-weekly drug, is effective for treating children with Growth Hormone Deficiency. Studies indicate that it is well-tolerated and provides effective growth hormone replacement. Specifically, the REAL4 trial demonstrated that children receiving Somapacitan experienced positive outcomes over a two-year period, and it was effective when children switched from daily growth hormone treatments to this weekly option. This suggests that Somapacitan is a convenient and effective alternative to daily treatments. ^{1 2 3 4 5}

What data supports the effectiveness of the drug Somapacitan for treating growth hormone deficiency in children?

Research shows that Somapacitan, a long-acting growth hormone, is effective and well-tolerated in children with growth hormone deficiency, with studies indicating successful results from once-weekly dosing. ^{1 2 3 4 5}

What safety data is available for Somapacitan in treating growth hormone deficiency?

Somapacitan has been evaluated in several studies for its safety and efficacy. It has been found to be well-tolerated in children with growth hormone deficiency in the Phase 3 REAL4 trial. Additionally, Phase I trials have shown it to be well-tolerated in both children and adults, including those with growth hormone deficiency. These studies support its use as a once-weekly treatment. ^{1 2 4 5 6}

Is Somapacitan safe for use in humans?

Somapacitan has been well-tolerated in both children and adults with growth hormone deficiency, according to several studies. It has been tested in various trials and found to be safe for use with a once-weekly dosing schedule. ^{1 2 4 5 6}

Is the drug Somapacitan a promising treatment for growth hormone deficiency in children?

Yes, Somapacitan is a promising drug for treating growth hormone deficiency in children. It is a long-acting growth hormone that can be given once a week instead of daily, making it more convenient for children and their families. Studies show it is effective and well-tolerated, which means it works well and is safe for use. ^{1 3 4 5 7}

How is the drug Somapacitan different from other treatments for growth hormone deficiency in children?

Somapacitan is unique because it is a long-acting growth hormone that only needs to be administered once a week, unlike other treatments that require daily injections. This can reduce the treatment burden for children and their families. ^{1 3 4 5 7}

Research Team

Clinical Transparency (dept. 2834)

Principal Investigator

Novo Nordisk A/S

Eligibility Criteria

This trial is for children aged 10-18 with Turner Syndrome, Noonan Syndrome, or born small for gestational age. They must have open growth plates and a specific height deficit. Children can't join if they have significant health issues affecting growth, diabetes, cancer history, or need certain steroid treatments.

Inclusion Criteria

My child is small for their age with growth plates still open and a bone age under 14 (if female) or under 16 (if male).

My child has Turner Syndrome confirmed by specific genetic tests and hasn't started growth hormone treatment.

My child has open growth plates and is under the age limit for their gender.

Exclusion Criteria

My child has diabetes or high blood sugar levels.

My child is suspected or confirmed to have growth hormone deficiency.

My child has a condition that could affect their growth.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive somapacitan once a week for 26 weeks to evaluate safety and efficacy

26 weeks

Weekly visits for injection training and monitoring

Extension

Participants continue to receive somapacitan for an additional 130 weeks to assess long-term safety and efficacy

130 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Somapacitan

Trial Overview The study tests Somapacitan—a new once-weekly injectable growth hormone treatment—over three years to see how safe it is and how well it helps these children grow. Participants will learn to self-inject the medication at home.

Participant Groups

Group I: Somapacitan Experimental Treatment 1 Intervention

Participants will receive Somapacitan for 26-week main phase followed by 130-week extension phase.

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

Approved in European Union as Sogroya for:

• Growth hormone deficiency in adults
• Growth hormone deficiency in children aged 3 years and above, and adolescents

Approved in United States as Sogroya for:

• Replacement of endogenous growth hormone in adults with growth hormone deficiency

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

Somapacitan, a long-acting growth hormone derivative, demonstrated sustained efficacy in promoting height velocity in children with growth hormone deficiency over a 2-year period, with similar growth rates observed after switching from daily growth hormone treatment.

The treatment was well tolerated with no safety issues reported, and a significant majority (90%) of patients and caregivers preferred the once-weekly somapacitan regimen over daily injections, indicating a favorable patient experience.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effective GH Replacement With Somapacitan in Children With GHD: REAL4 2-year Results and After Switch From Daily GH. Miller, BS., Blair, JC., Rasmussen, MH., et al.[2023]

Somapacitan, a once-weekly growth hormone treatment, showed similar efficacy to daily growth hormone (GH) in increasing height velocity in children with growth hormone deficiency over a 52-week period, confirming its noninferiority.

Both somapacitan and daily GH had comparable safety profiles, with low rates of injection-site reactions, and somapacitan was associated with a greater reduction in treatment burden for patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Weekly Somapacitan is Effective and Well Tolerated in Children With GH Deficiency: The Randomized Phase 3 REAL4 Trial. Miller, BS., Blair, JC., Rasmussen, MH., et al.[2023]

Somapacitan, a growth hormone derivative designed for weekly use, demonstrated effective pharmacokinetic properties across rats, minipigs, and monkeys, with sustained IGF-I levels for up to 10 days after a single dose.

In hypophysectomised rats, somapacitan led to a significant dose-dependent increase in body weight and IGF-I levels, indicating its potential efficacy in promoting growth in individuals with growth hormone deficiency.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Nonclinical pharmacokinetic and pharmacodynamic characterisation of somapacitan: A reversible non-covalent albumin-binding growth hormone. Thygesen, P., Andersen, HS., Behrens, C., et al.[2018]

Somapacitan, a long-acting growth hormone derivative, has been shown to be well-tolerated and effective in increasing IGF-I levels in both children and adults with growth hormone deficiency (GHD) based on pharmacokinetic/pharmacodynamic modeling from three phase I trials.

The study supports a once-weekly dosing regimen for somapacitan, demonstrating that it maintains elevated IGF-I levels throughout the week without significant accumulation, making it a practical treatment option for GHD.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pharmacokinetics and Pharmacodynamics of Once-Weekly Somapacitan in Children and Adults: Supporting Dosing Rationales with a Model-Based Analysis of Three Phase I Trials. Juul, RV., Rasmussen, MH., Agersø, H., et al.[2020]

Somapacitan, a long-acting growth hormone for treating growth hormone deficiency, allows for effective monitoring of insulin-like growth factor-I (IGF-I) levels through reliable linear models that predict mean and peak IGF-I levels based on blood samples taken after dosing.

The study simulated 39,200 IGF-I profiles from 49 patients (26 adults and 23 children) and found that samples taken on day 2 and day 4 after dosing provided the most accurate estimates for peak and mean IGF-I levels, respectively, indicating a robust method for monitoring treatment efficacy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Optimal Monitoring of Weekly IGF-I Levels During Growth Hormone Therapy With Once-Weekly Somapacitan. Juul Kildemoes, R., Højby Rasmussen, M., Agersø, H., et al.[2021]

Somapacitan, a long-acting growth hormone derivative, shows increased exposure in patients with kidney or hepatic impairment, indicating that dosage adjustments may be necessary for these individuals.

In trials involving 44 subjects with kidney impairment and 34 with hepatic impairment, no new safety issues were identified, suggesting that somapacitan is well-tolerated even in patients with compromised organ function.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effect of Kidney or Hepatic Impairment on the Pharmacokinetics and Pharmacodynamics of Somapacitan: Two Open-Label, Parallel-Group Trials. Bentz Damholt, B., Dombernowsky, SL., Dahl Bendtsen, M., et al.[2021]

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]

References

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

Effective GH Replacement With Somapacitan in Children With GHD: REAL4 2-year Results and After Switch From Daily GH. [2023]

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

Weekly Somapacitan is Effective and Well Tolerated in Children With GH Deficiency: The Randomized Phase 3 REAL4 Trial. [2023]

3. Scotland pubmed.ncbi.nlm.nih.gov

Nonclinical pharmacokinetic and pharmacodynamic characterisation of somapacitan: A reversible non-covalent albumin-binding growth hormone. [2018]

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

Pharmacokinetics and Pharmacodynamics of Once-Weekly Somapacitan in Children and Adults: Supporting Dosing Rationales with a Model-Based Analysis of Three Phase I Trials. [2020]

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

Optimal Monitoring of Weekly IGF-I Levels During Growth Hormone Therapy With Once-Weekly Somapacitan. [2021]

6. Switzerland pubmed.ncbi.nlm.nih.gov

Effect of Kidney or Hepatic Impairment on the Pharmacokinetics and Pharmacodynamics of Somapacitan: Two Open-Label, Parallel-Group Trials. [2021]

7. 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]