What is the mechanism of action of this treatment?

Turner Syndrome is commonly treated with growth hormone (GH) supplementation, such as somapacitan, which is administered weekly. GH stimulates the liver to produce insulin-like growth factor 1 (IGF-1), promoting bone growth and development. This treatment is essential for Turner Syndrome patients as it helps address short stature, a significant symptom of the condition, thereby enhancing their physical development and quality of life.

What side effects are associated with this type of intervention?

The most common side effects of growth hormone supplementation in the treatment of Turner Syndrome include injection site reactions, such as pain, redness, and swelling. Other potential side effects are headaches, muscle pain, joint stiffness, and an increased risk of developing glucose intolerance or diabetes. Rarely, patients may experience intracranial hypertension, which can cause severe headaches and vision problems. It is important for patients to be monitored regularly by their healthcare provider to manage and mitigate these side effects.

What prior FDA approvals exist?

The FDA has approved growth hormone therapy for the treatment of Turner Syndrome, which helps improve growth and increase final adult height. Recombinant human growth hormone (rhGH) is commonly used for this purpose. Examples of FDA-approved growth hormone treatments include somatropin, which is marketed under various brand names such as Genotropin, Humatrope, Norditropin, and others. These treatments are similar to somapacitan, which is being studied for its efficacy and safety in children with growth hormone deficiencies, including those with Turner Syndrome.

What other types of research exist?

While the provided context does not specify novel alternatives to Somapacitan for Turner Syndrome, other growth hormone therapies such as vosoritide (used in achondroplasia) and somatostatin analogs (used in acromegaly) may offer insights into alternative approaches. However, these alternatives are not specifically indicated for Turner Syndrome and should be discussed with a healthcare provider for suitability and efficacy.

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Growth Hormone Medicine

Somapacitan for Growth Hormone Deficiency in Children (REAL 9 Trial)

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 SGA: Open epiphyses; bone age < 14 years for females and < 16 years for males

Applicable to children with TS: Confirmed diagnosis of TS by 30-cell (or more) lymphocyte chromosomal analysis or confirmation of TS and TS mosaicism using comparative genomic hybridization (CGH)-array for GH treatment naïve participants

Must not have

Children diagnosed with diabetes mellitus or screening values from the central laboratory - Fasting plasma glucose above or equal to 126 mg/dL (7.0 mmol/L) or Glycated hemoglobin (HbA1c) above or equal to 6.5%

Applicable to children with SGA: Any known or suspected clinically significant abnormality likely to affect growth or the ability to evaluate growth with height

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from baseline (week 0) to week 156

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

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.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Possible side effects of Somapacitan are not detailed in the provided information but may be similar to other growth hormones: pain at injection site, headache, muscle pain, and possible impact on blood sugar levels.

Eligibility Criteria

Inclusion Criteria

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

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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).

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My child has Turner Syndrome confirmed by specific genetic tests and hasn't started growth hormone treatment.

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My child has open growth plates and is under the age limit for their gender.

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My child is significantly shorter than other kids their age and has not received growth hormone treatment.

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I am a child within the age range required for the study.

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I am a child with NS, aged 11-18 if male, or 10-18 if female.

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

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My child's growth plates are still open, and their bone age is under the limit for their gender.

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My child is significantly shorter than other kids their age and has not received growth hormone treatment.

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I am a girl aged between 10 and 18 years.

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My child's bone age is not more than 2 years behind their actual age and they haven't started GH treatment.

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I am a child with SGA, aged 11-18 if male, or 10-18 if female.

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My child has Noonan Syndrome, confirmed by clinical diagnosis or genetic test, and hasn't received growth hormone treatment.

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My child has Turner syndrome, is still growing, and meets the age criteria for bone development.

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My child is significantly shorter than average for their age and sex, and has not received growth hormone treatment.

Exclusion Criteria

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

Select...

My child has diabetes or high blood sugar levels.

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My child has a condition that could affect their growth.

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My child has a condition that could affect their growth.

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My child has a condition that could affect their growth.

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

Select...

I have or had cancer, including brain tumors.

Select...

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

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from baseline (week 0) to week 156

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from baseline (week 0) to week 156

This trial's timeline: 3 weeks for screening, Varies for treatment, and from baseline (week 0) to 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

Number of adverse events (AEs) 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 insulin-like growth factor 1 (IGF-1) SDS reported separately for SGA, TS, NS and ISS

+5 more

Awards & Highlights

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

1Treatment groups

Experimental Treatment

Group I: SomapacitanExperimental Treatment1 Intervention

Participants will receive Somapacitan for 26-week main phase followed by 130-week extension phase.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Somapacitan

2017

Completed Phase 1

~80

0 / 22Eligibility 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

Turner Syndrome is commonly treated with growth hormone (GH) supplementation, such as somapacitan, which is administered weekly. GH stimulates the liver to produce insulin-like growth factor 1 (IGF-1), promoting bone growth and development. This treatment is essential for Turner Syndrome patients as it helps address short stature, a significant symptom of the condition, thereby enhancing their physical development and quality of life.