Phase 3: Lonapegsomatropin [SKYTROFA®] for Turner Syndrome
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.
Official registry: https://clinicaltrials.gov/study/NCT07221851 · Ascendis Pharma A/S · enrollment listed as 186
What this phase means
Phase 3: this treatment already looked useful in smaller studies. Now it is tested in a large group, often against standard care or a dummy treatment, to see if it should be approved.
Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.
What the registry says happens
If you join, the listed intervention(s) are Lonapegsomatropin [SKYTROFA®], or Somatropin Pen Injector. The registry lists allocation as randomized. 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.
They plan to enroll about 186 people at 50 sites. Taking part typically means study visits and measurements plus check-ins.
Official title (unrewritten)
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
Interventions: Lonapegsomatropin [SKYTROFA®], Somatropin Pen Injector
Primary outcome (registry wording): Annualized Height Velocity (AHV) (cm/year)
Who it is for (from inclusion criteria)
- Ages 2 Years to 17 Years
- Chronological age between ≥2 and \<18 years, at start of screening
- Naïve to growth hormone and growth hormone promoting therapies
- Able to stand without assistance
- 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):
- 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
- Impaired growth or short stature defined as:
Who the registry excludes
- 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
- Any diagnosis or presence at screening of the following:
- Untreated moderate or severe sleep apnea as determined by formal (local) read of an inpatient or at-home sleep study
- Prader Willi syndrome with severe obesity, history of severe upper airway obstruction, or severe respiratory impairment
- Signs/symptoms of intracranial hypertension, active proliferative retinopathy
Public study contact (from the registry)
Ascendis Registry Inquiries
asnd_registryinquiries@ascendispharma.com
Published on ClinicalTrials.gov. Contact them as a possible participant. They decide eligibility.
Sites listed on ClinicalTrials.gov
50 listed- Ascendis Pharma Investigational Site — Phoenix, Arizona (recruiting)
- Ascendis Pharma Investigational Site — Palo Alto, California (recruiting)
- Ascendis Pharma Investigational Site — Sacramento, California (recruiting)
- Ascendis Pharma Investigational Site — Aurora, Colorado (recruiting)
- Ascendis Pharma Investigational Site — Centennial, Colorado (recruiting)
- Ascendis Pharma Investigational Site — Wilmington, Delaware (recruiting)
- Ascendis Pharma Investigational Site — Washington D.C., District of Columbia (recruiting)
- Ascendis Pharma Investigational Site — Orlando, Florida (recruiting)
- Ascendis Pharma Investigational Site — St. Petersburg, Florida (recruiting)
- Ascendis Pharma Investigational Site — Atlanta, Georgia (recruiting)
- Ascendis Pharma Investigational Site — Idaho Falls, Idaho (recruiting)
- Ascendis Pharma Investigational Site — New Orleans, Louisiana (recruiting)
+ 24 sites outside the US on the same record
FAQ about this NCT record
Is NCT07221851 still recruiting?
The sponsor posted status “Recruiting” on ClinicalTrials.gov at last ingest. Sites can pause independently. Confirm with the official record and the listed contact.
What phase is NCT07221851?
Phase 3. Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.
Can PlainTrials tell me if I qualify for NCT07221851?
No. The inclusion and exclusion lists on this page are readable extracts of the public eligibility block. Only the study team can screen you.
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