RecruitingTurner SyndromeShort Stature Homeobox Gene MutationIdiopathic Short Stature

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

Eligible age

2–17 yrs

Accepts

All genders

Locations

9 states

Healthy volunteers

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About this study

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.

Sponsor: Ascendis Pharma A/S

You may qualify if…

✓ 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 stature defined as:

You may not qualify if…

✕ 1. Advanced bone age X-ray by central reading defined as \>20% above chronological age in months (Greulich 1959).
✕ 2. Closed epiphyses as defined as bone age of ≥14.0 years in females or ≥16.0 years in males.
✕ 3. Current clinical diagnosis of diabetic retinopathy
✕ 4. Any diagnosis or presence at screening of the following:
✕ 1. Untreated moderate or severe sleep apnea as determined by formal (local) read of an inpatient or at-home sleep study.
✕ 2. Prader Willi syndrome with severe obesity, history of severe upper airway obstruction, or severe respiratory impairment.
✕ 5. Signs/symptoms of intracranial hypertension, active proliferative retinopathy.
✕ 6. Uncontrolled hypo- or hyperthyroidism.