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Compares treatment options for Turner Syndrome
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 [SKYTROFA®]?
Lonapegsomatropin [SKYTROFA®] is an investigational medicine, given as an once-weekly injection under the skin, being studied as a potential treatment for 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 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.
- 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 of TS, SHOX-D, SGA, or ISS with impaired growth or short stature, according to the following disease-specific criteria: TS or SHOX-D...
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
- 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.
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 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: (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. 46,XX...
- 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
- 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.
- 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...
- Exclusion Criteria only applicable to TS:
- 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...
- Exclusion Criteria only applicable to ISS:
- 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's taking place
- Palo Alto, California, United States
- Sacramento, California, United States
- Aurora, Colorado, United States
- Centennial, Colorado, 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
- Minneapolis, Minnesota, United States
- Saint Paul, Minnesota, United States
- Lake Success, New York, United States
- Oklahoma City, Oklahoma, United States
- Edinburg, Texas, United States
- Fort Worth, Texas, United States
- San Antonio, Texas, United States
- Le Kremlin-Bicêtre, France
- Toulouse, France
- Hanover, Germany
- Naples, Italy
+ 6 more site(s).
Compensation & support
Compensation mentioned.
ClinicalTrials.gov doesn't provide a reliable structured field for payment or travel support - confirm details with the study team.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team - confirm the details with them.
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 - 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?
Study sites include Palo Alto, California, United States; Sacramento, California, United States; Aurora, Colorado, United States; Centennial, Colorado, United States; Orlando, Florida, United States; St. Petersburg, Florida, United States and 20 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.