What is the mechanism of action of this treatment?

Turner Syndrome patients often experience short stature, and growth hormone therapy is a key treatment to address this issue. Lonapegsomatropin, a long-acting growth hormone, works by stimulating bone and tissue growth, thereby increasing height. Administered weekly, it offers a more convenient alternative to daily somatropin injections, potentially improving adherence and outcomes. This treatment is crucial for enhancing the physical development and quality of life in Turner Syndrome patients.

What side effects are associated with this type of intervention?

Common side effects of growth hormone treatments, including long-acting formulations like lonapegsomatropin, can include injection site reactions, headaches, and joint pain. Other potential side effects are swelling due to fluid retention, increased blood sugar levels, and, rarely, development of antibodies against the growth hormone. It is important for patients to discuss these potential side effects with their healthcare provider to manage and monitor any adverse reactions effectively.

What prior FDA approvals exist?

The FDA has previously approved somatropin, a daily growth hormone injection, for the treatment of Turner Syndrome. Somatropin helps to increase growth in individuals with this condition. The current trial is investigating lonapegsomatropin, a long-acting growth hormone administered once a week, which could offer a more convenient alternative to daily somatropin injections.

What other types of research exist?

A promising alternative to Lonapegsomatropin for Turner Syndrome patients is daily somatropin, which has been used effectively for growth hormone therapy. Other potential alternatives could include newer formulations of growth hormone treatments that may emerge from ongoing clinical trials or recent advancements in the field. It is essential to consult with a healthcare provider to discuss the most current and suitable treatment options available in 2024.

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Growth Hormone

Lonapegsomatropin vs Somatropin for Turner Syndrome

Phase 2

Waitlist Available

Research Sponsored by Ascendis Pharma Endocrinology Division A/S

Eligibility Criteria Checklist

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

Must have

AHV< 6 cm/year or <25ᵗʰ percentile over a time span of 6-18 months for children of 2 years and older

Biochemically euthyroid (including when on thyroid hormone supplementation)

Must not have

Diagnosis of diabetes mellitus

Known history or presence of malignancy

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 26 weeks, 52 weeks and 104 weeks

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new growth hormone treatment for young children with Turner syndrome to help them grow. The new treatment lasts longer in the body, so it doesn't need to be given as frequently. Growth hormone therapy has been used for many years to treat Turner syndrome, showing varied results in improving height.

Who is the study for?

This trial is for prepubertal children aged 1-10 with Turner syndrome, confirmed by genetic testing. They must be new to growth hormone therapy and have impaired growth as per specific criteria. Children can't join if they have Y-chromosomal material without gonadectomy, diabetes, certain medical conditions affecting growth, or a history of malignancy.

What is being tested?

The study compares different doses of Lonapegsomatropin (a long-acting weekly injection) against daily injections of Somatropin in treating Turner syndrome's growth impairment over 104 weeks. It will include about 48 participants across the United States who are randomly assigned to one of four groups.

What are the potential side effects?

Potential side effects may include reactions at the injection site, headaches, muscle pain, and possible development or worsening of scoliosis. Long-term use might affect glucose metabolism leading to increased 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's height velocity is less than 6 cm/year or below the 25th percentile for their age.

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My thyroid function is normal, with or without medication.

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I have never been treated with growth hormone or growth hormone secretagogues.

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I have been diagnosed with TS through a genetic test.

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I have not yet gone through puberty.

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I am between 1 and 10 years old.

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My bone age matches my actual age, within a 20% range.

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My height is below the 10th percentile for my age and sex.

Exclusion Criteria

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

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I have been diagnosed with diabetes.

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I have a history of cancer.

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I am pregnant, planning to become pregnant, or am breastfeeding.

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I have Turner Syndrome with Y-chromosome material and no history of gonad removal.

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I haven't had major surgery, serious injuries, or severe illness in the last 6 months.

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My high blood pressure is not well-managed.

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I have not taken any corticosteroids by mouth, IV, or muscle injection in the last 90 days.

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I do not have major health issues or reasons I can't take human growth hormone.

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My kidney function is not normal.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 26 weeks, 52 weeks and 104 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~26 weeks, 52 weeks and 104 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 26 weeks, 52 weeks and 104 weeks for reporting.

Treatment Details

Study Objectives

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

Secondary study objectives

Annualized Height Velocity (AHV) (cm/year)

Change from baseline in Bone age (calculated years)

Change from baseline in height standard deviation score (SDS)

+1 more

Other study objectives

Insulin-like growth factor 1 (IGF-1) standard deviation score (SDS)

Awards & Highlights

No Placebo-Only Group

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

Trial Design

4Treatment groups

Experimental Treatment

Active Control

Group I: Lonapegsomatropin at 0.36 mg hGH/kg/weekExperimental Treatment1 Intervention

Lonapegsomatropin at 0.36 mg hGH/kg/week administered once-weekly by subcutaneous injection

Group II: Lonapegsomatropin at 0.30 mg hGH/kg/weekExperimental Treatment1 Intervention

Lonapegsomatropin at 0.30 mg hGH/kg/week administered once-weekly by subcutaneous injection

Group III: Lonapegsomatropin at 0.24 mg hGH/kg/weekExperimental Treatment1 Intervention

Lonapegsomatropin at 0.24 mg hGH/kg/week administered once-weekly by subcutaneous injection

Group IV: Somatropin at 0.05 mg/kg/dayActive Control1 Intervention

Somatropin at 0.05 mg/kg/day administered once-daily by subcutaneous injection

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Lonapegsomatropin

2020

Completed Phase 3

~270

0 / 17Eligibility 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 patients often experience short stature, and growth hormone therapy is a key treatment to address this issue. Lonapegsomatropin, a long-acting growth hormone, works by stimulating bone and tissue growth, thereby increasing height. Administered weekly, it offers a more convenient alternative to daily somatropin injections, potentially improving adherence and outcomes. This treatment is crucial for enhancing the physical development and quality of life in Turner Syndrome patients.