Not recruitingRare disease

Study of Vosoritide for Children with Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome Not Responding to Growth Hormone

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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 effects of a treatment called vosoritide in children with certain growth-related conditions. These conditions include Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome. These are genetic conditions that can affect growth and development in children. The study will compare the effects of vosoritide with those of continued treatment with human growth hormone (hGH), which is a common treatment for these conditions.

The purpose of the study is to evaluate how different doses of vosoritide affect growth in children who have not responded adequately to hGH. The study will involve children who have been receiving hGH for at least a year. Participants will be randomly assigned to receive either vosoritide or continue with hGH. The study will last for several months, during which the growth of the children will be monitored and compared.

Throughout the study, researchers will observe changes in growth and monitor for any side effects. The study aims to provide insights into whether vosoritide can be an effective alternative or addition to hGH for improving growth in children with these specific conditions. The study will also track other health indicators to ensure the safety and effectiveness of the treatment. The results will help determine if vosoritide can be a beneficial treatment option for children with these growth challenges.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial assessment
Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes verifying age, genetic diagnosis, height, and previous treatment with human growth hormone (hGH).
Step 2
Baseline growth phase
Participants continue their current hGH treatment. This phase ensures a consistent baseline for growth measurements before any changes in treatment.
Step 3
Randomization
Participants are randomly assigned to either continue with hGH or switch to one of three doses of vosoritide. This process is essential for comparing the effects of the treatments.
Step 4
Treatment phase
Participants receive their assigned treatment for six months. Vosoritide is administered via subcutaneous injection. The specific dosage and frequency depend on the randomization outcome.
Step 5
Evaluation at six months
After six months, growth is assessed to determine the change from baseline. This includes measuring height and calculating the height Z-score.
Step 6
Extended treatment phase
Participants continue their assigned treatment for up to 24 months. Regular assessments are conducted to monitor growth and any potential side effects.
Step 7
Final assessment
At the end of the study, a comprehensive evaluation is performed. This includes measuring final adult height and comparing it to baseline measurements.

Who can join the trial?

7 criteria

Participants must be at least 3 years old and younger than 10 years old for females, or younger than 11 years old for males, at the time of signing the informed consent form.
Participants must have a genetically confirmed diagnosis of **Turner syndrome**, **SHOX deficiency**, or **Noonan syndrome**. These are specific genetic conditions that affect growth and development.
Participants must have a height measurement that is significantly below the average for their age and sex, specifically a height **Z-score** of -2.00 or lower. A Z-score is a way to measure how much a person's height differs from the average height of people of the same age and sex.
Participants must be at **Tanner Stage 1** at the time of signing the informed consent form. Tanner Stages are a way to describe physical development during puberty, and Stage 1 means the beginning stage before puberty starts.
Participants must have been receiving continuous **hGH** (human growth hormone) treatment for at least 1 year before joining the study. This treatment is used to help with growth in children with certain conditions.
Participants must be willing to continue receiving hGH treatment during the initial phase of the study and for 2 years after being randomly assigned to the hGH group, if applicable.
Participants must have shown an inadequate response to previous hGH treatment, meaning the treatment did not work as well as expected.

Who cannot join the trial?

4 criteria

Individuals who do not have **Turner Syndrome**, **Short Stature Homeobox-Containing Gene Deficiency**, or **Noonan Syndrome** cannot participate. These are specific genetic conditions that affect growth and development.
Participants must be within a certain age range, which is not specified here, but typically means they must be children or adolescents.
Both **females** and **males** are eligible, so exclusion is not based on gender.
Participants must not be part of a **vulnerable population** that requires special protection, such as those unable to give informed consent.

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Verified sites

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

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Azienda Ospedaliera Universitaria Universita' Degli Studi Della Campania Luigi Vanvitelli
Naples, Italy
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Bicetre Hospital
Le Kremlin-Bicetre, France
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Centre Hospitalier Regional D'Angers
Angers, France

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Investigated drugs

Vosoritide
is a medication being studied for its potential to help children with certain growth conditions. It is being tested to see if it can improve growth in children with Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome, especially in those who have not responded well to human growth hormone treatments.
Human Growth Hormone (hGH)
is a treatment commonly used to help children grow taller. In this study, it is used as a comparison to see how effective vosoritide is in promoting growth in children with specific growth disorders.

Vosoritide

What is already known about the treatment

Vosoritide – Vosoritide is administered through subcutaneous injection, meaning it is injected under the skin. It is currently being studied in clinical trials for its effectiveness in treating conditions like Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome, particularly in children who do not respond adequately to human growth hormone. The medication works by targeting a specific pathway in the body that regulates bone growth, aiming to promote increased growth in children with these conditions. Vosoritide is classified as a C-type natriuretic peptide analog, which means it mimics natural substances in the body that help control growth and development.

Investigated diseases

Turner Syndrome
Turner Syndrome is a genetic disorder that affects females, characterized by the partial or complete absence of one of the X chromosomes. This condition can lead to a variety of developmental issues, including short stature and the lack of ovarian development. Individuals with Turner Syndrome may also experience heart defects, kidney problems, and certain learning disabilities. The syndrome is often identified during childhood or adolescence when growth delays become apparent. Hormonal imbalances are common, affecting physical development and reproductive health. The severity and range of symptoms can vary widely among those affected.
Short Stature Homeobox-Containing Gene Deficiency
This condition is a genetic disorder that results in significantly shorter stature due to mutations affecting the SHOX gene. The SHOX gene plays a crucial role in bone growth and development, particularly in the long bones of the arms and legs. Individuals with this deficiency may exhibit disproportionate short stature, with the limbs being more affected than the trunk. Other skeletal abnormalities, such as a curved spine or abnormal forearm development, may also be present. The condition can be inherited or occur as a new mutation. It is often diagnosed in childhood when growth patterns deviate from typical ranges.
Noonan Syndrome
Noonan Syndrome is a genetic disorder that affects multiple parts of the body, leading to distinctive facial features, heart defects, and short stature. It is caused by mutations in one of several genes that are involved in cell growth and development. Individuals with Noonan Syndrome may have a wide neck, low-set ears, and a broad forehead. Heart problems, such as valve disorders, are common and can vary in severity. Other features may include developmental delays, bleeding disorders, and skeletal abnormalities. The condition is present from birth, and its manifestations can differ significantly among those affected.