Lonapegsomatropin vs Somatropin for Turner Syndrome

Recruiting in Palo Alto (17 mi)

+19 other locations

Age: < 18

Sex: Female

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Ascendis Pharma Endocrinology Division A/S

No Placebo Group

Prior Safety Data

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.

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.

+9 more

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.

+14 more

Participant Groups

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.

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