Not recruitingRare disease

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

Verified siteInvestigationalNo placebo

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the safety and effectiveness of a medication called Somapacitan in children who have difficulty growing. The study includes children with conditions such as Turner syndrome, Noonan syndrome, being born small for gestational age, or having idiopathic short stature, which means short stature without a known cause. Somapacitan is given as a solution for injection under the skin, using a special pen-injector designed for single patient use.

The purpose of the study is to evaluate how safe Somapacitan is and how well it works in helping children grow. Participants in the study will receive the medication once a week. The study will monitor the number of any adverse events, which are unexpected medical problems, over a period of time. The study will also look at how the children's height changes during the study.

The study will take place over several weeks, with regular check-ups to monitor the children's growth and any side effects they might experience. The goal is to gather information on how Somapacitan affects growth in children with these specific conditions, providing valuable insights into its potential benefits and safety.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial visit
Upon joining the study, an initial visit is scheduled. During this visit, a healthcare professional will explain the study details, including its purpose and what to expect.
A physical examination will be conducted to assess overall health and eligibility for the study. This may include measuring height, weight, and other relevant health indicators.
Step 2
Medication administration
The study involves the administration of a medication called somapacitan. This medication is provided as a solution for injection and is administered subcutaneously, which means it is injected under the skin.
The dosage is 15 mg of somapacitan in 1.5 ml of solution. The injection is given once a week.
Step 3
Weekly follow-ups
Weekly follow-up visits are scheduled to monitor the response to the medication. During these visits, healthcare professionals will check for any side effects and measure growth progress.
These visits are important to ensure the safety and effectiveness of the treatment.
Step 4
Mid-study evaluation
At approximately 26 weeks into the study, a more comprehensive evaluation is conducted. This includes assessing growth and any changes in health status.
The number of any adverse events, which are unexpected medical problems, will be recorded and analyzed.
Step 5
Long-term follow-up
The study continues with regular follow-up visits until 156 weeks. These visits are similar to the weekly follow-ups and are crucial for long-term monitoring.
The focus remains on tracking growth and any potential side effects over the extended period.
Step 6
Final assessment
At the end of the study period, a final assessment is conducted. This includes a thorough evaluation of growth and overall health.
The results from the entire study period are reviewed to determine the safety and effectiveness of the treatment.

Who can join the trial?

11 criteria

Children with Small for Gestational Age (SGA) - Born smaller than usual for the number of weeks of pregnancy, according to national standards.
Children with Turner's Syndrome (TS) - Female participants must be within a specific age range at the time of screening.
Children with Turner's Syndrome (TS) - Must have open epiphyses, which means the growth plates in the bones are still open, allowing for growth.
Children with Noonan Syndrome (NS) - Must have a diagnosis of Noonan Syndrome according to local clinical practice.
Children with Noonan Syndrome (NS) - Male and female participants must be within a specific age range at the time of screening.
Children with Noonan Syndrome (NS) - Must have open epiphyses, meaning the growth plates in the bones are still open.
Children with Idiopathic Short Stature (ISS) - Male and female participants must be within a specific age range at the time of screening.
Children with Idiopathic Short Stature (ISS) - Must have open epiphyses, meaning the growth plates in the bones are still open.
Children with Small for Gestational Age (SGA) - Male and female participants must be within a specific age range at the time of screening.
Children with Small for Gestational Age (SGA) - Must have open epiphyses, meaning the growth plates in the bones are still open.
Children with Turner's Syndrome (TS) - Must have a diagnosis of Turner's Syndrome according to local clinical practice.

Who cannot join the trial?

7 criteria

Patients with a history of any other significant medical condition that could interfere with the study.
Patients who have had any other growth hormone treatment within the last 6 months.
Patients with known allergies to the study medication or its ingredients.
Patients who are currently participating in another clinical trial.
Patients with a history of cancer or any other serious illness.
Patients who are unable to comply with the study procedures.
Patients with any condition that, in the opinion of the investigator, makes the patient unsuitable for the study.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Responsive
Rotterdam, The Netherlands
Hospital Vall d’Hebron
Verified
Barcelona, Spain
Kliniczny Szpital Wojewodzki Nr 2 Im. Sw. Jadwigi Krolowej W Rzeszowie
Verified
Rzeszow, Poland

Trial locations

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Investigated drugs

Somapacitan is a medication being studied for its potential to help children grow taller. It is given once a week and is being tested in children who are shorter than usual for their age. This includes children who were born smaller than expected, or who have certain conditions like Turner syndrome, Noonan syndrome, or idiopathic short stature, which means their short height doesn't have a known cause. The study is looking at how safe this medication is for children and how well it works to help them grow.

Somapacitan

What is already known about the treatment

Somapacitan – Somapacitan is administered as an injection under the skin, typically given once a week. It is currently being studied for its safety and effectiveness in children with growth issues, such as those with Noonan syndrome, Turner syndrome, small size for their gestational age, or idiopathic short stature. The medication works by mimicking the natural growth hormone in the body, helping to stimulate growth in children who have a deficiency or need additional growth support. Somapacitan is classified as a growth hormone therapy, and it aims to improve height and growth patterns in affected children.

Investigated diseases

Noonan syndrome
Noonan syndrome is a genetic disorder that affects various parts of the body. It is characterized by distinctive facial features, short stature, and heart defects. Individuals with this condition may also experience developmental delays and learning difficulties. The syndrome can lead to a wide range of symptoms, which can vary significantly among affected individuals. As the person grows, some features may become more pronounced, while others may become less noticeable. The progression of symptoms can differ, with some individuals experiencing more severe manifestations than others.
Turner syndrome
Turner syndrome is a chromosomal disorder that affects females, characterized by the partial or complete absence of one of the X chromosomes. This condition often results in short stature and ovarian insufficiency, leading to infertility. Other common features include a webbed neck, low-set ears, and a broad chest. As individuals with Turner syndrome age, they may experience additional health issues such as heart defects and hearing loss. The severity and range of symptoms can vary widely among those affected. The progression of the condition is influenced by the specific genetic makeup and the presence of associated health issues.
Small for gestational age
Small for gestational age (SGA) refers to a condition where a newborn baby is smaller than the typical size for the number of weeks of pregnancy. This can occur due to various factors, including maternal health issues, placental problems, or genetic conditions. Babies born SGA may have a lower birth weight and may face challenges in growth and development. As the child grows, they may catch up in size, but some may continue to experience growth issues. The progression of growth in SGA children can vary, with some achieving normal growth patterns over time. Monitoring and support are often needed to ensure healthy development.
Idiopathic short stature
Idiopathic short stature (ISS) is a condition where a child is significantly shorter than average for their age and gender, with no identifiable medical cause. This condition is diagnosed when other potential causes of short stature, such as hormonal deficiencies or genetic disorders, have been ruled out. Children with ISS typically have normal body proportions and do not exhibit other symptoms associated with growth disorders. The growth rate in children with ISS is usually slower than average, but they may continue to grow at a steady pace. The progression of height in individuals with ISS can vary, with some achieving a final adult height within the normal range. The condition is often monitored to assess growth patterns over time.