Short Stature Homeobox Gene Mutation

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Review clinical trials related to Short Stature Homeobox Gene Mutation. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Recruiting

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

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.

Participants needed: 186
Trial details
Phase: Phase 3Age: 2-17Biological sex: AllType: InterventionalSponsor: Ascendis Pharma A/SUpdated: Jun 23, 2026Locations: 25
Eligibility criteria

Chronological age between ≥2 and <18 years, at start of screening. [+6]

Advanced bone age X-ray by central reading defined as >20% above chronological a... [+19]