Lonapegsomatropin vs Somatropin for Turner Syndrome

Not currently recruiting at 20 trial locations

Overseen By Ascendis Pharma

Age: < 18

Sex: Female

Trial Phase: Phase 2

Sponsor: Ascendis Pharma Endocrinology Division A/S

Must be taking: Hormone replacements

Must not be taking: Corticosteroids, Anabolic agents

Disqualifiers: Diabetes, Cardiac dysfunction, Malignancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 104 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new long-acting growth hormone treatment called lonapegsomatropin for children with Turner syndrome, a condition affecting growth and development. The study aims to determine the best dose of lonapegsomatropin, administered once a week, and compare it to a daily growth hormone treatment called somatropin. Children diagnosed with Turner syndrome through a genetic test, who are not yet in puberty and have not previously received growth hormone treatment, might be suitable for this trial. Participants will help researchers assess whether the new treatment can improve growth more effectively or conveniently than the current daily option. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of participants.

Do I have to stop taking my current medications for the trial?

The trial protocol does not specify if you need to stop taking your current medications. However, if you are on hormone replacement therapies for hormone deficiencies other than growth hormone, you must be on stable doses for at least 4 weeks before and during the screening period.

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

Research has shown that lonapegsomatropin is generally safe and well tolerated. Previous studies found no issues that caused participants to stop the treatment, suggesting it is comfortable for most people. Similarly, somatropin, a commonly used growth hormone, is also safe for humans. Most of its side effects are mild, such as minor skin reactions at the injection site. Both lonapegsomatropin and somatropin have good safety records, making them reliable options for those considering joining a clinical trial. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about Lonapegsomatropin for Turner Syndrome because it offers a more convenient dosing schedule compared to current treatments like Somatropin. Unlike Somatropin, which requires daily injections, Lonapegsomatropin is administered just once a week, making it easier for patients to manage. This less frequent dosing could improve adherence and overall quality of life for those with Turner Syndrome. Additionally, Lonapegsomatropin uses a pegylated form of growth hormone, which may provide longer-lasting effects in the body.

What evidence suggests that this trial's treatments could be effective for Turner syndrome?

This trial will compare Lonapegsomatropin with Somatropin for treating Turner syndrome. Research has shown that Lonapegsomatropin, a long-lasting growth hormone, effectively treats growth hormone deficiencies. Studies have found it to be as safe and beneficial for hormone balance as daily treatments like Somatropin. Specifically, Lonapegsomatropin has successfully helped patients with Turner syndrome grow. One study confirmed it works as well as daily treatments, with no major safety differences. These findings suggest Lonapegsomatropin could be a promising option for boosting growth in people with Turner syndrome. ^{1 2 3 4 6}

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

Showing slow or abnormal growth based on at least one of the following:

I have been on stable hormone replacement therapy, except for growth hormone, for over 4 weeks.

My child's height velocity is less than 6 cm/year or below the 25th percentile for their age.

Exclusion Criteria

I am not taking any drugs that affect growth hormone actions.

I have been diagnosed with diabetes.

I have a history of cancer.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive lonapegsomatropin or somatropin for 104 weeks to assess safety, tolerability, and efficacy

104 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension (optional)

Participants may opt into continuation of treatment with lonapegsomatropin for an additional 78 weeks

78 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Lonapegsomatropin
• Somatropin

Trial Overview 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.

How Is the Trial Designed?

Group I: Lonapegsomatropin at 0.36 mg hGH/kg/week Experimental Treatment 1 Intervention

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

Group II: Lonapegsomatropin at 0.30 mg hGH/kg/week Experimental Treatment 1 Intervention

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

Group III: Lonapegsomatropin at 0.24 mg hGH/kg/week Experimental Treatment 1 Intervention

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

Group IV: Somatropin at 0.05 mg/kg/day Active Control 1 Intervention

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

Who Is Running the Clinical Trial?

Ascendis Pharma Endocrinology Division A/S

Lead Sponsor

Trials

7

Recruited

2,300+

Published Research Related to This Trial

In a study of 67 Italian patients with growth hormone deficiency, Omnitrope® was found to be effective in improving health markers like insulin-like growth factor-1 levels and body composition over an average treatment duration of 45.4 months.

While 55.2% of patients experienced adverse events, none were deemed related to Omnitrope®, indicating that it is generally safe for long-term use in adults with growth hormone deficiency.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-term safety and efficacy of Omnitrope&#174; in adults with growth hormone deficiency: Italian interim analysis of the PATRO Adults study. Ferone, D., Profka, E., Gasco, V., et al.[2018]

In a study of 291 children treated with the somatropin biosimilar Omnitrope® for an average of 33 months, the treatment showed acceptable safety with only 12% experiencing adverse events, primarily mild issues like headaches and abdominal pain.

The effectiveness of Omnitrope® was demonstrated by significant improvements in height standard deviation scores (HSDS) and height velocity standard deviation scores (HVSDS), indicating positive growth outcomes over a treatment period of up to 6.5 years.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and effectiveness of a somatropin biosimilar in children requiring growth hormone treatment: second analysis of the PATRO Children study Italian cohort. Iughetti, L., Antoniazzi, F., Giavoli, C., et al.[2021]

Citations

1. investors.ascendispharma.com investors.ascendispharma.com/news-releases/news-release-details/new-insights-trial-transcontm-hgh-lonapegsomatropin-turner

New InsiGHTS Trial of TransCon™ hGH ... Week 26 topline data from the New InsiGHTS Trial showed that once-weekly TransCon hGH provided comparable safety and endocrine benefits to those seen with the ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT07221851

NCT07221851 | Trial Investigating the Efficacy and Safety ... This basket trial will enroll prepubertal children and adolescents with clinically diagnosed and genetically confirmed (if applicable) TS, ...

3. withpower.com withpower.com/trial/phase-2-gonadal-dysgenesis-2023-ecacb

Lonapegsomatropin vs Somatropin for Turner Syndrome The effectiveness of Omnitrope® was demonstrated by significant improvements in height standard deviation scores (HSDS) and height velocity standard deviation ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11480202/

Efficacy and Safety of Somapacitan Relative to Somatrogon ... No efficacy and safety differences were identified in comparisons of once weekly somapacitan versus somatrogon and lonapegsomatropin, as well as daily GH.

5. hcplive.com hcplive.com/view/fda-approves-lonapegsomatropin-tcgd-skytrofa-formerly-transcon-hgh-for-adult-growth-hormone-deficiency

FDA Approves Lonapegsomatropin-tcgd (Skytrofa), ... The foresiGHt trial demonstrated significant efficacy and safety of lonapegsomatropin compared to placebo and daily somatropin.

6. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/nda/2021/761177Orig1s000MedR.pdf

761177Orig1s000 - accessdata.fda.gov Safety Analyses by Demographic Subgroups. The Applicant also presented the safety data as a subgroup analysis conducted on Safety Pool II,.