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

Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age2-17
SponsorAscendis Pharma A/S

About this trial

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.

Eligibility criteria

Qualifiers

Chronological age between ≥2 and <18 years, at start of screening.

Naïve to growth hormone and growth hormone promoting therapies.

Prepubertal.

Able to stand without assistance.

Disqualifiers

Advanced bone age X-ray by central reading defined as >20% above chronological age in months (Greulich 1959).

Closed epiphyses as defined as bone age of ≥14.0 years in females or ≥16.0 years in males.

Current clinical diagnosis of diabetic retinopathy

Untreated moderate or severe sleep apnea as determined by formal (local) read of an inpatient or at-home sleep study.

Trial design

Treatments tested in this trial

  • Lonapegsomatropin [SKYTROFA®]
  • Somatropin Pen Injector

Treatment groups

186 Participants
are divided into 2 treatment groups

Sponsors and collaborators