Ongoing Clinical Trials for Short Stature
There are currently 3 clinical trials investigating treatments for short stature in children and adolescents. These studies are testing different growth hormone therapies and combination treatments across multiple European countries, aiming to improve height outcomes for children born small for gestational age or those with conditions such as Turner syndrome, Noonan syndrome, and idiopathic short stature.
Clinical trial locations
- Austria
- Belgium
- Bulgaria
- Croatia
- Finland
- France
- Germany
- Greece
- Italy
- Latvia
- Lithuania
- Netherlands
- Study Comparing Somapacitan and Somatropin for Growth in Children with Short Stature Due to Small for Gestational Age, Turner Syndrome, Noonan Syndrome, or Idiopathic Short Stature
- Study on the Safety and Effectiveness of Somapacitan for Children with Short Stature Due to Noonan Syndrome, Turner Syndrome, Small for Gestational Age, or Idiopathic Short Stature
- Poland
- Study Comparing Somapacitan and Somatropin for Growth in Children with Short Stature Due to Small for Gestational Age, Turner Syndrome, Noonan Syndrome, or Idiopathic Short Stature
- Study on the Safety and Effectiveness of Somapacitan for Children with Short Stature Due to Noonan Syndrome, Turner Syndrome, Small for Gestational Age, or Idiopathic Short Stature
- Portugal
- Slovenia
- Spain
- Study Comparing Somapacitan and Somatropin for Growth in Children with Short Stature Due to Small for Gestational Age, Turner Syndrome, Noonan Syndrome, or Idiopathic Short Stature
- Study on the Safety and Effectiveness of Somapacitan for Children with Short Stature Due to Noonan Syndrome, Turner Syndrome, Small for Gestational Age, or Idiopathic Short Stature
Study on the Effects of Somatropin and Letrozole in Boys with Short Predicted Height
This trial is examining a combination treatment approach for boys with predicted adult height below 164 cm. The study tests whether combining Omnitrope, a growth hormone, with Letrozole, an aromatase inhibitor, can lead to greater height gains than using growth hormone alone.
Who can participate: The study is looking for boys who are already in puberty, shown by having at least 4 ml of testicular volume. They must have a predicted adult height at or below -2.5 standard deviations (less than 164 cm) and a bone age between 11 and 13 years. Participants and their guardians must provide informed consent after understanding what the study involves.
Who cannot participate: Only boys are eligible for this study; girls cannot participate. Boys whose puberty has not yet started or is already advanced are excluded. Those whose predicted adult height is not considered short, or who belong to vulnerable populations requiring special protection, also cannot join.
Treatment approach: Omnitrope is given as an injection under the skin to support natural growth processes during puberty. Letrozole is taken as a tablet and works by reducing estrogen levels in the body, which may help increase the effectiveness of growth hormone therapy. The treatment lasts for four years, during which participants receive regular monitoring including height measurements, bone age assessments, and evaluation of any side effects. At the end of the treatment period, the final height is compared to the initial predicted height to evaluate the treatment’s effectiveness.
Study Comparing Somapacitan and Somatropin for Growth in Children with Short Stature Due to Small for Gestational Age, Turner Syndrome, Noonan Syndrome, or Idiopathic Short Stature
This large international trial is comparing a new once-weekly treatment called somapacitan with a daily treatment called Norditropin. The study includes children with various causes of short stature: being born small for gestational age, Turner syndrome, Noonan syndrome, or idiopathic short stature where the cause is unknown.
Who can participate: Children must not have received any previous growth-promoting treatments. For boys born small for gestational age, they must be at least 2 years and 26 weeks old but younger than 11 years, with testis volume below 4 mL. For girls born small for gestational age, they must be at least 2 years and 26 weeks old but younger than 10 years, without breast development. Girls with Turner syndrome must have a confirmed diagnosis and meet similar age requirements without breast development. Children with Noonan syndrome must have a clinical diagnosis, with boys meeting the same age and testis volume requirements as boys with small for gestational age, and girls meeting the same age and development requirements as girls with small for gestational age. For idiopathic short stature, similar age requirements apply, and bone age must be within specific ranges: 12 years or younger for boys and 11 years or younger for girls, with no more than 2 years difference from actual age.
Who cannot participate: Children without one of the specified conditions cannot join. Those outside the specified age ranges, taking medications that could affect growth, or with previous growth hormone treatment are excluded. Children with allergies to the study medication, other medical conditions that might interfere with the study, or who cannot comply with study procedures are also ineligible.
Treatment approach: Somapacitan is given once weekly through an injection under the skin using a pre-filled pen device in doses of 5 mg, 10 mg, or 15 mg. Norditropin is administered daily, also through subcutaneous injection using a pre-filled pen. Both medications work by mimicking or supplementing natural growth hormone to stimulate growth and development. Throughout the study, children’s growth is monitored regularly by measuring height velocity, changes in bone age, and various health indicators including fasting blood sugar and glycated hemoglobin levels. The goal is to confirm that once-weekly somapacitan is as effective as daily Norditropin in promoting growth.
Study on the Safety and Effectiveness of Somapacitan for Children with Short Stature Due to Noonan Syndrome, Turner Syndrome, Small for Gestational Age, or Idiopathic Short Stature
This trial focuses on evaluating the long-term safety and effectiveness of somapacitan in children with various growth conditions. The study monitors children over an extended period, up to 156 weeks, to thoroughly assess both growth outcomes and any potential side effects.
Who can participate: Children born small for gestational age must have been born smaller than usual according to national standards. Girls with Turner syndrome must have a confirmed diagnosis and be within a specific age range at screening, with open growth plates in their bones. Children with Noonan syndrome must have a diagnosis according to local clinical practice, with both boys and girls meeting age requirements at screening and having open growth plates. Children with idiopathic short stature must also be within specific age ranges at screening and have open growth plates.
Who cannot participate: Patients with a history of significant medical conditions that could interfere with the study are excluded. Those who received any growth hormone treatment within the last 6 months, have known allergies to the study medication or its ingredients, are currently in another clinical trial, or have a history of cancer or serious illness cannot participate. Patients unable to comply with study procedures or deemed unsuitable by the investigator are also excluded.
Treatment approach: Somapacitan is administered once weekly as an injection under the skin at a dose of 15 mg in 1.5 ml solution, using a special pen-injector designed for single patient use. The medication works by mimicking natural growth hormone in the body to stimulate growth in children with growth hormone deficiency or those needing additional growth support. After the initial visit involving physical examination and assessment of eligibility, children receive weekly injections with regular follow-up visits to monitor growth progress and check for side effects. A comprehensive mid-study evaluation occurs at approximately 26 weeks, with continued monitoring until 156 weeks. Throughout the study, researchers track the number of adverse events and measure changes in height to provide valuable information about the medication’s benefits and safety profile.
Summary
The three ongoing clinical trials for short stature are testing innovative treatment approaches across Europe. Two of the studies focus on somapacitan, a once-weekly growth hormone treatment that offers a more convenient dosing schedule compared to traditional daily injections. The third study explores combination therapy with growth hormone and an aromatase inhibitor specifically for boys.
The trials span 16 European countries, with the largest study available in countries including Germany, Italy, France, Spain, Netherlands, Poland, Belgium, and others. The research encompasses children with multiple causes of short stature, including being born small for gestational age, genetic conditions like Turner syndrome and Noonan syndrome, and cases where the cause is unknown.
All studies emphasize the importance of participants having open growth plates and no previous growth hormone treatment. The research aims to provide evidence on both effectiveness and long-term safety, with some studies monitoring participants for up to four years. These trials represent important advances in understanding how different treatment approaches can help children with growth challenges achieve better height outcomes.
