Somapacitan for Growth Hormone Deficiency in Children

(REAL 9 Trial)

Not currently recruiting at 40 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety and effectiveness of a new growth hormone medicine, somapacitan, for children with certain growth challenges. It focuses on children born small, those with Turner syndrome, Noonan syndrome, or idiopathic short stature (unexplained short height). Participants will use the medicine once a week for about three years. Children who have not received growth hormone treatment before and are significantly shorter than average for their age might be suitable candidates. As a Phase 3 trial, this study represents the final step before FDA approval, giving participants the opportunity to contribute to the potential availability of a new treatment.

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.

Is there any evidence suggesting that somapacitan is likely to be safe for children?

Research shows that patients generally tolerate somapacitan well. In earlier studies, somapacitan did not interfere with sugar processing in the body, even with use for up to 86 weeks.

Another study found that children born smaller than average tolerated somapacitan well. They experienced no safety concerns or issues with how the medicine felt in their bodies, indicating they handled the treatment well.

Overall, the safety of somapacitan appears consistent across different studies, showing no serious side effects. However, treatments can affect people differently, so discussing any concerns with a healthcare provider is important. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard of care for growth hormone deficiency, which often involves daily injections, Somapacitan is unique because it offers a weekly injection option. This longer-acting growth hormone analog provides a more convenient dosing schedule, potentially improving adherence and quality of life for children and their families. Researchers are particularly excited about Somapacitan because it maintains consistent hormone levels throughout the week, which could enhance treatment outcomes and reduce the burden of frequent injections.

What evidence suggests that somapacitan might be an effective treatment for growth hormone deficiency in children?

Research has shown that somapacitan, which participants in this trial will receive, effectively treats growth hormone deficiency in children. In earlier studies, children who switched to somapacitan grew about 3.4 inches each year for two years. This growth mirrors that seen with daily growth hormone treatments. Administered once a week, somapacitan may be easier than daily injections. The treatment has shown promising results, providing a convenient option for children and their caregivers. ^{1 2 3 6 7}

Who Is on the Research Team?

Clinical Transparency (dept. 2834)

Principal Investigator

Novo Nordisk A/S

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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

Published Research Related to This Trial

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 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]

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, 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 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]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10505532/

Weekly Somapacitan in GH Deficiency: 4-Year Efficacy, ... Observer-reported outcomes showed that patients and parents/guardians seem to have experienced a reduced treatment burden when switching from daily GH to ...

2. ncbi.nlm.nih.gov ncbi.nlm.nih.gov/books/NBK599837/

Somapacitan (Sogroya) - NCBI Bookshelf - NIH CDEC concluded that somapacitan may meet some of the needs identified by patients and their caregivers, including comparable growth results to existing ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT02616562

NCT02616562 | Investigating Efficacy and Safety of Once- ... 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 ...

4. sogroya.com sogroya.com/results-and-safety/for-children.html

Growth Hormone Treatment for Children Children switching to Sogroya® grew ~3.4 inches per year (8.7 cm), on average, at 2 years. These results are from the REAL4 main trial phase ( ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT03811535

NCT03811535 | A Research Study in Children With a Low ... 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 ...

6. academic.oup.com academic.oup.com/jcem/article/110/4/1086/7756773

Somapacitan in Children Born SGA: 52-Week Efficacy, Safety ... Somapacitan was well-tolerated at all doses without safety or local tolerability issues identified. Safety and tolerability of somapacitan were consistent with ...

7. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40400262/

Efficacy, safety, and insulin-like growth factor I of weekly ... This study aims to evaluate the efficacy and tolerability of somapacitan after 3 years of treatment and 2 years after switch from daily GH in children with GHD.