Trials / Recruiting
RecruitingNCT07221851
Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children and Adolescents With Short Stature or Growth Failure Due to Growth Hormone Sufficient Disorders
A Pivotal, Parallel-Arm, Phase 3, Open-Label, Active-controlled, Global, Multicenter, Randomized Basket Trial Investigating the Efficacy and Safety of Once-weekly Lonapegsomatropin Compared to Daily Somatropin in Prepubertal Children and Adolescents With Growth Failure or Short Stature Due to Growth Hormone Sufficient Disorders - Turner Syndrome, SHOX Deficiency, Small for Gestational Age, and Idiopathic Short Stature
- Status
- Recruiting
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 186 (estimated)
- Sponsor
- Ascendis Pharma A/S · Industry
- Sex
- All
- Age
- 2 Years – 17 Years
- Healthy volunteers
- Not accepted
Summary
This basket trial will enroll prepubertal children and adolescents with clinically diagnosed and genetically confirmed (if applicable) TS, SHOX-D, SGA, or ISS between ages of ≥2 and \<18 years with open growth plates. The purpose of the study is to see how well treatment with once-weekly lonapegsomatropin works compared to treatment with daily somatropin. Approximately 186 participants will be distributed equally (1:1), to receive either lonapegsomatropin for 2 years or somatropin for 1 year followed by lonapegsomatropin for 1 year. This trial will be conducted in the United States, France, Germany, Italy, Romania, Spain and South Korea.
Conditions
- Turner Syndrome
- Short Stature Homeobox Gene Mutation
- Idiopathic Short Stature
- Small for Gestational Age at Delivery
Interventions
| Type | Name | Description |
|---|---|---|
| COMBINATION_PRODUCT | Lonapegsomatropin [SKYTROFA®] | Subcutaneous injection once weekly |
| COMBINATION_PRODUCT | Somatropin Pen Injector | Subcutaneous injection once daily |
Timeline
- Start date
- 2025-12-12
- Primary completion
- 2028-02-01
- Completion
- 2029-03-01
- First posted
- 2025-10-28
- Last updated
- 2026-04-08
Locations
7 sites across 1 country: United States
Regulatory
- FDA-regulated drug study
- FDA-regulated device study
Source: ClinicalTrials.gov record NCT07221851. Inclusion in this directory is not an endorsement.