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
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
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 2 Years to 17 Years
- Sex
- All sexes
- Healthy volunteers
- Not accepted
- Compensation
- Not stated
Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.
What to expect
No one can promise a trial will help you personally — that is what the research is trying to find out.
What participation involves
- Time periods mentioned: 2 years, 1 year
Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.
Show the study's own words
- "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."
- Compensation
- Not stated
- Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
- Participants are monitored closely by the research team throughout the study.
- Some participants may receive access to an investigational treatment, which is still being studied.
- Your participation helps researchers learn whether this approach could help future patients.
About this study
In plain language
This study compares weekly lonapegsomatropin with daily somatropin to see how well they help children grow and how safe they are. It is for children ages 2 to under 18 who have not started puberty and have certain causes of short height or poor growth, but do not lack growth hormone. Treatment lasts 2 years; children starting daily shots switch to weekly shots after 1 year.
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.
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- Be age 2 to under 18, have not started puberty, and have bones that are still able to grow.
- Have never used growth hormone or treatments that help the body make growth hormone.
- Be able to stand without help.
- Have Turner syndrome (TS), SHOX gene deficiency (SHOX-D), small size at birth without catching up in growth (SGA), or short height with no known cause (ISS).
- Meet the study’s height or growth-rate limits for the diagnosis. TS and SHOX-D must be confirmed by a genetic test.
- For ISS, tests must show normal growth hormone function. Girls must also have testing that shows 46,XX chromosomes.
- If taking medicine to replace other hormones, use a suitable, steady dose for at least 4 weeks before screening and during screening.
- A parent or legal guardian must give written permission. The child must also agree if required.
Who can't join
- Bones are too mature for the child’s age or have stopped growing.
- Have diabetes-related eye damage, certain abnormal blood vessel growth in the eye, or signs of high pressure around the brain.
- Have untreated moderate or severe sleep apnea (breathing pauses during sleep), or Prader-Willi syndrome with severe obesity or serious breathing problems.
- Have poorly controlled thyroid disease or diabetes, untreated or poorly controlled high blood pressure, or major liver or kidney problems.
- Have a history of gut inflammation, HIV, radiation exposure, certain bone growth disorders, growth hormone deficiency, or heart or chest surgery.
- Use medicines that may affect growth or growth hormone, including sex hormones or ADHD stimulants; or have used high-dose inhaled steroids for more than 28 days in a row within 12 months.
- Are pregnant, planning pregnancy, or breastfeeding; joined another study testing a drug within 90 days or are in one now; or have a condition that makes this study unsafe or hard to follow.
- Have certain diagnosis-specific findings: for TS, some chromosome findings or significant heart problems; for SGA or ISS, certain genetic conditions, syndromes, or bone problems that affect growth.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
Inclusion Criteria: 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: (i.) AHV \<25th percentile over a time span of 6-16 months prior to screening utilizing a historical height properly documented in a health care setting (self-measurement record is not accepted) OR (ii.) Height \<5th percentile for sex and age according to the Centers for Disease Control Growth Charts for the United States SGA without catch-up growth: c. Birth weight and/or birth length \< -2.0 SDS for gestational age according to the 2006 World Health Organization Child Growth Standards. For infants born premature, the Fenton Preterm Infant Growth Chart (Fenton 2013) should be used. d. Impaired growth or short stature defined as: (i.) AHV \<25th percentile over a time span of 6-16 months prior to screening properly documented in a health care setting (self-measurement record is not accepted) OR (ii.) Height \< -2.0 SDS for age and sex according to the 2000 Centers for Disease Control Growth Charts for the United States for children ≥ 3 years or height \< -2.5 SDS for age and sex according to the for children ≥ 2 years and \< 3 years ISS: e. Height \< -2.25 SDS for sex and age according to the Centers for Disease Control Growth Charts for the United States with no identifiable cause for short stature. f. Documented normal GH-IGF-1 axis, defined as either: (i.)IGF-1 SDS \>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 chromosome as determined by karyotype or microarray if female. For karyotypes, a minimum of 30 cells must be counted. 6. 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. 7. 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. Exclusion Criteria: 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. 7. Uncontrolled diabetes mellitus (defined as: HbA1c \>7.5% from central laboratory at screening). 8. 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 on growth. 9. Any significant hepatic or renal abnormality, such as abnormal renal function (defined as eGFR \<60 mL/min/1.73m2). 10. Undiagnosed or uncontrolled hypertension. 11. 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). 12. High dose inhaled glucocorticoid for more than 28 consecutive days total over the course of 12 months. 13. Female who is pregnant, plans to be pregnant, or breastfeeding. 14. Participation in another interventional clinical trial involving an investigational compound within 90 days prior to screening or in parallel to this trial. 15. 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 product or trial procedures. 16. Exclusion Criteria only applicable to TS: 1. Presence of Y chromosome material on genetic testing without history of gonadectomy. 2. Less than 10% of 45,X mosaicism. 3. Any known, clinically significant, congenital or acquired cardiovascular dysfunction that might interfere with growth. 17. 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, significant gene mutations, or medical syndromes with short stature, including but not limited to Turner syndrome, Laron syndrome, Noonan syndrome, Prader-Willi syndrome, abnormal SHOX-1 gene analysis or absence of GH receptors. (ii.)Congenital abnormalities (causing skeletal abnormalities), including but not limited to skeletal dysplasias. 18. Exclusion Criteria only applicable to ISS: 1. 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 limited to Turner syndrome, Laron syndrome, Noonan syndrome, Prader-Willi syndrome, abnormal SHOX-1 gene analysis or absence of gH receptors.
Study sites (23)
CA
- Ascendis Pharma Investigational Site · Palo Alto, CA 94304More trials in Palo Alto
- Ascendis Pharma Investigational Site · Sacramento, CA 95821More trials in Sacramento
CO
- Ascendis Pharma Investigational Site · Aurora, CO 80045More trials in Aurora
- Ascendis Pharma Investigational Site · Centennial, CO 80112More trials in Centennial
DC
- Ascendis Pharma Investigational Site · Washington D.C., DC 20010More trials in Washington D.C.
DE
- Ascendis Pharma Investigational Site · Wilmington, DE 19803More trials in Wilmington
FL
- Ascendis Pharma Investigational Site · Orlando, FL 32806More trials in Orlando
- Ascendis Pharma Investigational Site · St. Petersburg, FL 33701More trials in St. Petersburg
GA
- Ascendis Pharma Investigational Site · Atlanta, GA 30329More trials in Atlanta
ID
- Ascendis Pharma Investigational Site · Idaho Falls, ID 83404More trials in Idaho Falls
LA
- Ascendis Pharma Investigational Site · New Orleans, LA 70118More trials in New Orleans
MN
- Ascendis Pharma Investigational Site · Minneapolis, MN 55454More trials in Minneapolis
- Ascendis Pharma Investigational Site · Saint Paul, MN 55102More trials in Saint Paul
NY
- Ascendis Pharma Investigational Site · Lake Success, NY 11042More trials in Lake Success
OH
- Ascendis Pharma Investigational Site · Cincinnati, OH 45229More trials in Cincinnati
OK
- Ascendis Pharma Investigational Site · Oklahoma City, OK 73104More trials in Oklahoma City
TX
- Ascendis Pharma Investigational Site · Edinburg, TX 78539More trials in Edinburg
- Ascendis Pharma Investigational Site · Fort Worth, TX 76104More trials in Fort Worth
- Ascendis Pharma Investigational Site · San Antonio, TX 78229More trials in San Antonio
- Ascendis Pharma Investigational Site · San Antonio, TX 78232More trials in San Antonio
UT
- Ascendis Pharma Investigational Site · Salt Lake City, UT 84112More trials in Salt Lake City
VA
- Ascendis Pharma Investigational Site · Charlottesville, VA 22908More trials in Charlottesville
WA
- Ascendis Pharma Investigational Site · Seattle, WA 98105More trials in Seattle