Weekly Somapacitan vs Daily Norditropin® for Growth Hormone Deficiency

(REAL4 Trial)

Recruiting at 227 trial locations

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Novo Nordisk A/S

Must not be taking: Systemic corticosteroids, Inhaled glucocorticoids

Disqualifiers: Inflammatory diseases, ADHD, Malignancy, 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

Compensation: Varies

Number of Visits: 19

Duration: 52 weeks

200 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing a new weekly growth hormone medicine, somapacitan, in children who lack enough growth hormone. The goal is to see if somapacitan works well and is safe. The study will last several years with regular clinic visits. Somapacitan is intended for once weekly administration for treatment of both adults and children with growth hormone deficiency.

Will I have to stop taking my current medications?

The trial requires that participants do not take certain medications that could affect growth, such as treatments for ADHD like methylphenidate. If you are on such medications, you may need to stop them to participate in the trial.

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

Somapacitan, a long-acting growth hormone, has been shown to be effective for adults with growth hormone deficiency, as it is approved by the FDA for once-weekly use, offering a convenient alternative to daily injections. Studies have demonstrated similar safety and effectiveness compared to daily growth hormone treatments, making it a promising option for patients. ^{1 2 3 4 5}

Is weekly Somapacitan safe for humans?

Research shows that weekly Somapacitan has been evaluated for safety in adults with growth hormone deficiency, and it has been found to have a similar safety profile to daily Norditropin. It has been approved by the FDA for use in adults, indicating it is generally considered safe for human use. ^{1 2 3 4 5}

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

Somapacitan is unique because it is administered weekly, unlike other growth hormone treatments like Norditropin® which require daily injections. This less frequent dosing can make it more convenient for patients. ^{6 7 8 9 10}

Research Team

Clinical Transparency (dept. 2834)

Principal Investigator

Novo Nordisk A/S

Eligibility Criteria

This trial is for prepubertal children with growth hormone deficiency, who are significantly shorter than average for their age and gender, have slow growth rates, and low levels of IGF-I. They must not have used growth hormone therapy before and should be diagnosed through specific tests. Children with certain diseases affecting growth or taking medications that influence height aren't eligible.

Inclusion Criteria

I am significantly shorter than average for my age and gender.

My child's growth rate is slower than expected for their age and gender.

My IGF-I levels are lower than normal for my age and gender.

Exclusion Criteria

My child has been on high-dose asthma medication for over a month in the past year.

I have had cancer before, including brain tumors.

I have been diagnosed with ADHD.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2 weeks

Treatment

Participants receive either somapacitan weekly or Norditropin® daily for 52 weeks

52 weeks

19 visits (in-person), 1 phone call

Extension

Participants completing the main trial period receive somapacitan weekly for 3 additional years

3 years

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 once a week) to Norditropin® (an existing daily treatment) in children lacking sufficient growth hormones. The effectiveness and safety of somapacitan will be evaluated over four years, involving 19 clinic visits and one phone call.

Participant Groups

Group I: Somapacitan weekly Experimental Treatment 1 Intervention

Participants will receive somapacitan weekly for 52 weeks (main trial period). Participants completing the main trial period in both the treatment arms ('Somapacitan weekly' and 'Norditropin® daily') will receive somapacitan weekly for 3 years (extension trial period).

Group II: Norditropin® daily Active Control 1 Intervention

Participants will receive Norditropin® daily for 52 weeks (main trial period).

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]

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]

The study found that once-daily 0.002% omidenepag isopropyl (OMDI) significantly reduced intraocular pressure (IOP) by an average of 2.96 mm Hg in patients with primary open angle glaucoma (POAG) or ocular hypertension (OHT) who did not respond well to latanoprost, indicating its efficacy as a treatment option.

Safety assessments showed no significant adverse events during the OMDI treatment, suggesting that it is a safe alternative for patients with POAG or OHT who are low or non-responders to existing therapies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Omidenepag Isopropyl in Latanoprost Low/Non-Responders with Primary Open-Angle Glaucoma or Ocular Hypertension: A Phase 3, Non-Randomized, Two-Phase, Open-Label Study. Panarelli, JF., Bowden, EC., Tepedino, ME., et al.[2023]

References

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

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

2. Spain pubmed.ncbi.nlm.nih.gov

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

3. 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]

4. 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]

5. 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]

6. China pubmed.ncbi.nlm.nih.gov

[Comparison of intraocular pressure reducing effects of three prostaglandin eyedrops in open-angle glaucoma]. [2018]

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

The effectiveness of latanoprost for the treatment of pediatric glaucoma. [2019]

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

Omidenepag Isopropyl in Latanoprost Low/Non-Responders with Primary Open-Angle Glaucoma or Ocular Hypertension: A Phase 3, Non-Randomized, Two-Phase, Open-Label Study. [2023]

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

24-Hour IOP control with once-daily bimatoprost, timolol gel-forming solution, or latanoprost: a 1-month, randomized, comparative clinical trial. [2022]

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

Latanoprost for the treatment of pediatric glaucoma. [2019]