What prior FDA approvals exist?

The FDA has approved several growth hormone therapies for conditions such as Small for Gestational Age (SGA), Turner Syndrome, Noonan Syndrome, and Intrauterine Growth Restriction (IUGR). Norditropin® (somatropin) is one such growth hormone therapy that is commonly prescribed. It is used to treat children with growth failure due to these conditions by promoting growth and development. Somapacitan, a newer growth hormone therapy, is currently being studied for its efficacy and safety compared to Norditropin® in treating these conditions.

What other types of research exist?

Promising novel alternatives to Somapacitan for treating conditions such as Small for Gestational Age, Turner Syndrome, Noonan Syndrome, and Intrauterine Growth Restriction include vosoritide, a C-type natriuretic peptide analogue, which has shown efficacy in children with achondroplasia. Additionally, advancements in gene therapy and targeted molecular treatments, such as FGFR3 inhibitors for achondroplasia, may offer new therapeutic options. Continuous research and clinical trials in these areas are essential to validate their efficacy and safety for broader use in 2024.

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Growth Hormone

Somapacitan vs Norditropin for Growth Disorders (REAL 8 Trial)

Q: What side effects are associated with this type of intervention?

Growth hormone therapy, such as treatments with Somapacitan and Norditropin®, is commonly used for conditions like Small for Gestational Age, Turner Syndrome, Noonan Syndrome, and Intrauterine Growth Restriction. Known side effects of growth hormone therapy include injection site reactions, headaches, muscle pain, joint stiffness, and an increased risk of developing diabetes. In rare cases, it can lead to intracranial hypertension, slipped capital femoral epiphysis, and progression of scoliosis. Regular monitoring by healthcare providers is essential to manage these potential side effects.

Phase 3

Recruiting

Research Sponsored by Novo Nordisk A/S

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Applicable to children with ISS: Prepubertal children: Boys: Age above or equal to 2 years and 26 weeks and below 11.0 years at screening. Testis volume below 4 mL

Prepubertal children: Boys: Age above or equal to 2 years and 26 weeks and below 11.0 years at screening. Testis volume below 4 mL

Must not have

Children requiring inhaled glucocorticoid therapy at a dose greater than 400 µg/day of inhaled budesonide or equivalent for longer than 4 consecutive weeks within the last 12 months prior to screening

Current treatment with sex hormones or aromatase inhibitors

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from screening (visit 1) to visit 15 (week 156)

Awards & highlights

All Individual Drugs Already Approved

No Placebo-Only Group

Pivotal Trial

Summary

This trial compares two growth hormone treatments for children with growth issues. Somapacitan is a new treatment, while Norditropin® is an existing treatment. The study aims to find out which treatment helps these children grow better. Somapacitan is a long-acting growth hormone derivative in development for treatment of GH deficiency.

Who is the study for?

This trial is for children who were born small and remain small, or have Turner Syndrome, Noonan Syndrome, or idiopathic short stature. They must not have had growth-promoting therapy before and meet specific criteria related to age, puberty status, BMI, and height. Children with certain medical conditions or on conflicting medications are excluded.

What is being tested?

The study compares somapacitan (a new medicine given once a week) with Norditropin® (an existing treatment taken daily). Over three years, participants will either receive somapacitan the whole time or start with Norditropin® for one year followed by somapacitan for two years. The assignment of treatments is random.

What are the potential side effects?

While the side effects are not detailed in this summary, common side effects of growth hormone therapies can include injection site reactions, headaches, muscle pain, joint stiffness and hypothyroidism. Long-term safety profiles will be closely monitored throughout the study.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My son is between 2.5 and 11 years old with small testes.

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My son is between 2.5 and 11 years old with small testes.

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I have not used growth-promoting treatments like growth hormone.

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For girls with ISS: 2 years and 26 weeks to 10 years old, no breast development, and specific bone age. For boys with ISS: up to 12 years old with specific bone age. Height at least 2.5 standard deviations below the average for age and sex. Also, a specific growth hormone test result is needed.

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I was born smaller than the average for my gestational age.

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My child has been diagnosed with Noonan Syndrome.

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My BMI is below the 95th percentile for my age, according to CDC charts.

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I have Turner Syndrome confirmed by a specific blood test.

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My thyroid hormone therapy has been stable for the last 3 months.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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

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I am currently taking sex hormones or aromatase inhibitors.

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I haven't taken steroids for inflammation for more than 2 weeks in the last 3 months.

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I have or had another type of cancer, brain tumor, or brain cyst.

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My child has NS or a related disorder and hasn't been stable on a gluten-free diet for the past year.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from screening (visit 1) to visit 15 (week 156)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from screening (visit 1) to visit 15 (week 156)

This trial's timeline: 3 weeks for screening, Varies for treatment, and from screening (visit 1) to visit 15 (week 156) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Height velocity reported separately for small for gestational age (SGA), Turner syndrome (TS), Noonan syndrome (NS) and idiopathic short stature (ISS)

Secondary study objectives

Change in Height Velocity SDS reported separately for SGA, TS, NS and ISS

Change in Height standard deviation scores (SDS) reported separately for SGA, TS, NS and ISS

Change in bone age for ISS

+9 more

Awards & Highlights

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Active Control

Group I: SomapacitanExperimental Treatment1 Intervention

Participants will receive Somapacitan for 156 weeks

Group II: Norditropin®Active Control1 Intervention

Participants will receive Norditropin® for 52 weeks (main phase) and Somapacitan for 104 weeks (extension phase)

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Somapacitan

FDA approved

0 / 14Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Growth hormone therapy, such as somapacitan, stimulates the liver to produce insulin-like growth factor 1 (IGF-1), which promotes bone growth and increases muscle mass. This mechanism is vital for patients with Small for Gestational Age (SGA), Turner Syndrome, Noonan Syndrome, and Intrauterine Growth Restriction (IUGR) as these conditions often lead to short stature and delayed growth. By enhancing growth and development, growth hormone therapy helps these patients achieve a height closer to the average for their age and sex, thereby improving their overall health and quality of life.