Lonapegsomatropin vs Somatropin for Turner Syndrome

Recruiting at 19 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

48 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

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.

What data supports the effectiveness of the drug Lonapegsomatropin for Turner Syndrome?

Research shows that various forms of recombinant human growth hormone (like Somatropin and its biosimilars) are effective in increasing height in children with Turner Syndrome. Studies have demonstrated significant improvements in growth rates and other growth-related factors, indicating that these treatments are generally safe and effective for managing short stature in Turner Syndrome. ^{1 2 3 4 5}

Is Lonapegsomatropin or Somatropin safe for humans?

Studies on Somatropin, including its various forms like Omnitrope and Saizen, show that it is generally safe for humans, with most side effects being mild, such as muscle and joint pain. Serious side effects are rare, and the safety profile is well-documented in both children and adults with growth hormone deficiencies. ^{6 7 8 9 10}

How does the drug Lonapegsomatropin differ from Somatropin in treating Turner Syndrome?

Lonapegsomatropin is a newer formulation that may offer a different dosing schedule or administration method compared to traditional Somatropin, which is available under various brand names and typically requires regular injections. ^{11 12 13 14 15}

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

References

1. Switzerland pubmed.ncbi.nlm.nih.gov

Safety and Effectiveness of Recombinant Human Growth Hormone in Children with Turner Syndrome: Data from the PATRO Children Study. [2022]

2. England pubmed.ncbi.nlm.nih.gov

Switching to Omnitrope(®) from Other Recombinant Human Growth Hormone Therapies: A Retrospective Study in an Integrated Healthcare System. [2022]

3. Sweden pubmed.ncbi.nlm.nih.gov

Knemometry as a predictor of response to somatrem in Turner's syndrome. [2019]

4. England pubmed.ncbi.nlm.nih.gov

Recombinant growth hormone therapy in children with Turner Syndrome in Korea: a phase III Randomized Trial. [2022]

5. Germany pubmed.ncbi.nlm.nih.gov

Efficacy and safety of Valtropin in the treatment of short stature in girls with Turner's syndrome. [2019]

6. Italy 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. [2018]

7. Italy 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. [2021]

8. England pubmed.ncbi.nlm.nih.gov

Bioequivalence studies of omnitrope, the first biosimilar/rhGH follow-on protein: two comparative phase 1 randomized studies and population pharmacokinetic analysis. [2011]

9. Israel pubmed.ncbi.nlm.nih.gov

The GHMonitorSM Registry: an update of the last three years. [2008]

10. United States pubmed.ncbi.nlm.nih.gov

Weekly Lonapegsomatropin in Treatment-Naïve Children With Growth Hormone Deficiency: The Phase 3 heiGHt Trial. [2022]

11. United States pubmed.ncbi.nlm.nih.gov

Patient and Healthcare Provider Perspectives of First-Generation Somatostatin Analogs in the Management of Neuroendocrine Tumors and Acromegaly: A Systematic Literature Review. [2021]

12. England pubmed.ncbi.nlm.nih.gov

Progress in the formulation and delivery of somatostatin analogs for acromegaly. [2018]

13. Singapore pubmed.ncbi.nlm.nih.gov

Clinical experience of octreotide in the treatment of acromegaly. [2016]

14. England pubmed.ncbi.nlm.nih.gov

Octreotide LAR: safety and tolerability issues. [2019]

15. Australia pubmed.ncbi.nlm.nih.gov

Treatment of severe orthostatic hypotension with the somatostatin analogue octreotide. [2019]