Turner Syndrome study: Somatropin Pen Injector
Official title 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
ClinicalTrials.gov ID: NCT07221851
What this study is testing
What is Lonapegsomatropin?
An investigational once‑weekly injectable medicine being studied for growth in children with Turner Syndrome.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study is testing whether a weekly injection of lonapegsomatropin helps children grow as well as daily injections. It also looks at how safe the weekly treatment is.
- Who it's for
- You may be able to join if you are a child or teen aged 2 to 17 with Turner syndrome, a SHOX gene mutation, small for gestational age, or idiopathic short stature, and you have open growth plates.
- What's involved
- You would receive a weekly injection of lonapegsomatropin for two years, or a daily injection for one year followed by a weekly injection for another year. You will have regular doctor visits and growth measurements.
- Time commitment
- The study lasts two years, with visits every few weeks for injections and growth checks.
- Phase 3: a large, late-stage study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 17
You may be able to join if
- 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.
- Diagnosis confirmed by a genetic test. NOTE: Historical test results are acceptable for proof of diagnosis.
You likely can't join if
- 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.
- Prader Willi syndrome with severe obesity, history of severe upper airway obstruction, or severe respiratory impairment.
- Signs/symptoms of intracranial hypertension, active proliferative retinopathy.
See the full eligibility criteria
- 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.
- 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: (i.) AHV \ 0 at screening based on central laboratory OR (ii.)Historical documentation of normal peak GH upon stimulation test (as defined by local institution) g.
- If on hormone replacement therapies for any hormone deficiencies other than growth hormone (e.g., adrenal, thyroid), must be on adequate and stable doses for ≥4 weeks prior to and throughout screening.
- Written, signed informed consent provided by parent(s) or legal guardian(s) of the participant. Assent should be signed by participant as required by IRB/HREC/IEC.
- 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.
- Prader Willi syndrome with severe obesity, history of severe upper airway obstruction, or severe respiratory impairment.
- Signs/symptoms of intracranial hypertension, active proliferative retinopathy.
- Uncontrolled hypo- or hyperthyroidism.
- Uncontrolled diabetes mellitus (defined as: HbA1c \>7.5% from central laboratory at screening).
- Known history or diagnosis of any gastrointestinal inflammatory condition, HIV, radiation exposure, other skeletal dysplasias, growth hormone deficiency, and/or cardio-thoracic surgery due to their independent effects...
- Any significant hepatic or renal abnormality, such as abnormal renal function (defined as eGFR \<60 mL/min/1.73m2).
- Undiagnosed or uncontrolled hypertension.
- Receiving treatment with any agent that might influence growth or interfere with GH secretion or action including any sex steroids and stimulants for attention-deficit/hyperactivity disorder (ADHD).
- High dose inhaled glucocorticoid for more than 28 consecutive days total over the course of 12 months.
- Female who is pregnant, plans to be pregnant, or breastfeeding.
- Participation in another treatment clinical trial involving an investigational compound within 90 days prior to screening or in parallel to this trial.
- Any disease or condition that, in the judgement of the investigator, may make the participant unlikely to comply with the requirements of the protocol or any condition that presents undue risk from the investigational...
- Presence of Y chromosome material on genetic testing without history of gonadectomy.
- Less than 10% of 45,X mosaicism.
- Any known, clinically significant, congenital or acquired cardiovascular dysfunction that might interfere with growth.
- Exclusion Criteria only applicable to SGA: a. Any known clinically significant abnormality likely to affect growth or the ability to evaluate growth with standing height measurements: (i.)Chromosomal aneuploidy...
- Known history of any condition that causes disproportionate short stature (i.e. skeletal dysplasias), chromosomal aneuploidy, significant gene mutations, or medical syndromes with short stature, including but not...
The study team makes the final eligibility decision.
Where it is enrolling
- Phoenix, Arizona, United States
- Palo Alto, California, United States
- Sacramento, California, United States
- Aurora, Colorado, United States
- Centennial, Colorado, United States
- Wilmington, Delaware, United States
- Washington D.C., District of Columbia, United States
- Orlando, Florida, United States
- St. Petersburg, Florida, United States
- Atlanta, Georgia, United States
- Idaho Falls, Idaho, United States
- New Orleans, Louisiana, United States
- Boston, Massachusetts, United States
- Minneapolis, Minnesota, United States
- Saint Paul, Minnesota, United States
- Lake Success, New York, United States
- Chapel Hill, North Carolina, United States
- Cincinnati, Ohio, United States
- Oklahoma City, Oklahoma, United States
- Columbia, South Carolina, United States
+ 30 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years to 17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Phoenix, Arizona, United States; Palo Alto, California, United States; Sacramento, California, United States; Aurora, Colorado, United States; Centennial, Colorado, United States; Wilmington, Delaware, United States and 44 more locations. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.