Ongoing Clinical Trials for Growth Hormone Deficiency
There are currently 3 ongoing clinical trials focused on growth hormone deficiency, a condition where the body does not produce enough growth hormone necessary for normal growth and development in children. These trials are being conducted across multiple European countries including Austria, France, Germany, Italy, Latvia, Poland, Slovenia, and Spain, testing different approaches to growth hormone treatment including new weekly medications and comparing continuation versus stopping of therapy during puberty.
Clinical trial locations
- Austria
- France
- Germany
- Italy
- Latvia
- Poland
- Slovenia
- Spain
Long-Term Safety Study of LUM-201 (Ibutamoren Mesilate) in Children with Growth Hormone Deficiency
This trial is examining the long-term safety of LUM-201, a medication containing Ibutamoren Mesilate, in children with idiopathic growth hormone deficiency. The medication is taken orally in tablet form and works by stimulating the pituitary gland to release more growth hormone.
Who can participate: Children who have already completed at least 12 months of treatment with LUM-201 in a previous study and have shown positive results. To be eligible, participants must have demonstrated a growth rate of at least 6.7 centimeters per year after 12 months of treatment. A parent or caregiver must provide consent, and the child must also agree to participate if they are old enough to understand. The principal investigator must approve eligibility before the child can begin.
Who cannot participate: Children without idiopathic growth hormone deficiency, those outside the specified age range, or those with other medical conditions that might interfere with the study cannot join. Children taking medications that affect growth hormone levels or who have had previous treatments that could influence results are also excluded. Additionally, children who cannot follow study procedures or attend all required visits are not eligible.
What the trial focuses on: The main goal is to monitor the long-term safety of LUM-201 over an extended period, with the study expected to continue until November 2028. Participants will have regular check-ups every six months to assess growth hormone levels, insulin-like growth factor 1 levels, and changes in height and weight. Bone age compared to chronological age will be evaluated annually. Healthcare professionals will track various health indicators and monitor for any side effects or changes in health throughout the study.
Study on Growth Hormone Deficiency: Comparing Effects of Continuing vs. Stopping Somatropin Treatment in Pubertal Children with Isolated Growth Hormone Deficiency
This study is examining what happens when pubertal children who have shown improvement in their condition either continue or stop taking somatropin, a laboratory-made form of human growth hormone. Multiple brands will be used including NutropinAq, Saizen, Omnitrope, Humatrope, Norditropin FlexPro, and Genotropin MiniQuick, all administered as injections.
Who can participate: Children aged 8-15 years for girls and 9-17 years for boys who have reversed isolated growth hormone deficiency and are in established puberty. Girls must be in Tanner stages B2/3, while boys should have testes size between 6-12 milliliters. Participants must have stopped taking growth hormone medication for at least 6 weeks before retesting and must show a reversal of deficiency with a peak stimulated growth hormone level of 6.7 micrograms per liter or higher. A normal brain MRI scan showing a small anterior pituitary gland is required. Written informed consent from both the child and their guardians is necessary.
Who cannot participate: Children with any other medical condition that might interfere with the study, those currently taking medications affecting growth hormone levels, or those with previous growth hormone treatment history are excluded. Children with a history of cancer, tumors, uncontrolled diabetes, severe liver or kidney disease, or known allergies to study medications cannot participate. Pregnant or breastfeeding patients and those who have participated in another clinical trial within 30 days are also excluded.
What the trial focuses on: The study compares the effects of continuing versus stopping growth hormone treatment in children who have reached puberty and have shown reversal of their deficiency. Participants will be randomly divided into two groups: one continuing treatment and one stopping. The main goal is to assess how these different approaches affect final adult height. The study will monitor growth and development through height measurements, bone health assessments, and blood marker evaluations, continuing until early 2026.
Study on the Effects of Somapacitan and Somatropin in Children with Growth Hormone Deficiency
This trial is comparing two different growth hormone treatments: Somapacitan, administered once weekly, and Norditropin, given daily. Both are delivered as injections under the skin using pen-injector devices. The study aims to determine which treatment is more effective in helping children with growth hormone deficiency grow and develop properly.
Who can participate: Children who have not yet started puberty are eligible. Boys should be at least 2 years and 26 weeks old but younger than 11 years, with testis volume less than 4 milliliters. Girls should be at least 2 years and 26 weeks old but younger than 10 years without breast development. Participants must have confirmed growth hormone deficiency verified by two different tests within the past year. Their height must be at least 2 standard deviations below average for their age and gender, with a growth rate slower than the 25th percentile measured over 6 to 18 months. Low levels of Insulin-like Growth Factor-I at the study start are required, and children should not have received previous growth hormone or IGF-I treatment.
Who cannot participate: Children without growth hormone deficiency, those outside the specific age range, or those with other medical conditions that might interfere with the study are excluded. Children currently taking medications affecting growth or interacting with the study treatment, those with previous growth hormone treatment, or those with allergies to the study medication are not eligible. Children unable to follow study procedures or attend required visits cannot participate.
What the trial focuses on: The study monitors how well children grow in height and examines changes in bone age and blood markers. Participants are randomly assigned to receive either weekly Somapacitan or daily Norditropin. Somapacitan is provided in pre-filled pens with dosages of 5 mg, 10 mg, or 15 mg per 1.5 mL solution. The primary endpoint is the change in height velocity from the start of the trial to week 52, with long-term monitoring continuing up to week 208. Regular follow-up visits include height measurements, bone age assessments, and blood tests. Safety is closely monitored throughout, including checks for changes in blood sugar levels and other health indicators.
Summary
The three ongoing trials represent different approaches to managing growth hormone deficiency in children. One trial focuses on long-term safety monitoring of an oral medication (LUM-201) in Poland, expected to continue until 2028. Another addresses an important clinical question about whether to continue or stop treatment during puberty in Austria. The largest trial, conducted across eight European countries (Germany, Slovenia, Latvia, France, Italy, Spain, Poland, and Austria), compares the convenience and effectiveness of weekly versus daily injections.
These trials collectively address various aspects of treatment: safety monitoring, optimal treatment duration during developmental stages, and more convenient dosing schedules. The geographic spread of trials, particularly the multi-country study on Somapacitan, suggests significant European interest in improving treatment options for children with this condition. Each trial uses different medications or approaches, reflecting the ongoing search for better ways to help affected children achieve normal growth and development.
