Ascendis Pharma A/S
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.
Inclusion Criteria: 1. Chronological age between ≥2 and \<18 years, at start of screening. 2. Naïve to growth hormone and growth hormone promoting therapies. 3. Prepubertal. 4. Able to stand without assistance. 5. Diagnosis of TS, SHOX-D, SGA, or ISS with impaired growth or short stature, according to the following disease-specific criteria: TS or SHOX-D (Léri-Weill dyschondrosteosis): 1. Diagnosis confirmed by a genetic test. NOTE: Historical test results are acceptable for proof of diagnosis. For karyotypes, a minimum of 20 cells must be counted. 2. Impaired growth or short statur…
Subcutaneous injection once weekly
Subcutaneous injection once daily