Somapacitan for Growth Hormone Deficiency in Children

(REAL 9 Trial)

Recruiting in Palo Alto (17 mi)

+34 other locations

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Novo Nordisk A/S

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Approved in 2 Jurisdictions

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.

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 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 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}

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.

Treatment Details

Interventions

• Somapacitan (Growth Hormone Medicine)

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.

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

Univ of AL at Birmingham_BRM Birmingham, AL

Sutter Valley Med Fdt Ped Endo Sacramento, CA

Rocky Mt Ped and Endo Centennial, CO

Childrens National Medical Ctr Washington, United States

More Trial Locations

Who Is Running the Clinical Trial?

Novo Nordisk A/S

Lead Sponsor

Trials

1578

Patients Recruited

3,813,000+

Findings from Research

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]

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]

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]

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]

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]

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]

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]