Weekly Somapacitan vs Daily Norditropin® for Growth Hormone Deficiency

(REAL4 Trial)

No longer recruiting at 262 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests two treatments for children lacking sufficient growth hormone for proper growth. One group will take a daily medicine, Norditropin®, while the other will receive somapacitan, a new treatment administered as a weekly injection. The trial aims to determine if somapacitan is as effective as the daily option and to assess its safety. Children diagnosed with growth hormone deficiency who have not previously received growth hormone therapy may be suitable candidates. Participants will be randomly assigned to one of the treatment groups and monitored for four years. As a Phase 3 trial, this study represents the final step before FDA approval, providing an opportunity to contribute to important research.

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.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Studies have shown that somapacitan, administered once a week, is generally well-tolerated by patients. Research indicates that children who switched from daily growth hormone injections to weekly somapacitan experienced similar safety outcomes. The side effects were comparable for those who continued with somapacitan and those who switched from daily treatments.

In another study, children using somapacitan for up to three years continued to grow well and had safety results similar to those on daily treatments. This suggests that somapacitan is a safe option for treating growth hormone deficiency in children.

Norditropin® is already approved for daily use in children with growth hormone deficiency, demonstrating its safety. In this study, participants will receive either somapacitan or Norditropin® to compare the two treatments. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Somapacitan is unique because it offers a once-weekly injection for growth hormone deficiency, compared to the daily injections required with Norditropin® and other standard treatments. This less frequent dosing could significantly improve convenience and quality of life for patients, reducing the burden of daily medication administration. Researchers are excited because Somapacitan maintains effectiveness with fewer doses, which could enhance adherence to treatment and ultimately lead to better health outcomes.

What evidence suggests that this trial's treatments could be effective for growth hormone deficiency?

This trial will compare the effectiveness of weekly Somapacitan with daily Norditropin® for children with growth hormone deficiency. Research has shown that Somapacitan, administered once a week, yields promising results. Specifically, studies found that children who switched from daily growth hormone shots to Somapacitan grew about 3.4 inches per year on average over two years. This finding suggests that Somapacitan supports growth as effectively as the daily treatment. Additionally, families have reported that the weekly dose is less burdensome than daily injections. Overall, Somapacitan appears to be an effective and more convenient option for children requiring growth hormone therapy. ^{1 2 3 5 6}

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 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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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

Published Research Related to This Trial

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]

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]

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 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]

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. 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.

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

NCT02616562 | Investigating Efficacy and Safety of Once- ... The aim of the trial is to investigate efficacy and safety of once-weekly NNC0195-0092 treatment compared to daily growth hormone treatment (Norditropin® ...

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/NCT02229851

NCT02229851 | Trial to Compare the Efficacy and Safety of ... This study is conducted globally. The purpose is to demonstrate the efficacy of once weekly dosing of NNC0195-0092 (somapacitan) compared to placebo and ...

6. novomedlink.com novomedlink.com/growth-related-disorders/products/treatments/sogroya/efficacy-safety/pediatric-ghd.html

Pediatric Growth Hormone Deficiency | Sogroya® ... Pediatric patients showed sustained growth with once-weekly Sogroya® for up to 3 years · Sogroya® demonstrated similar efficacy to daily somatropin with fewer ...