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Study on the Effects of Vosoritide for Children with Idiopathic Short Stature

Verified siteRegistered drugNo 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 a condition known as Idiopathic Short Stature (ISS) in children. This condition is characterized by a height significantly shorter than average for a child's age and sex, without a known cause. The study will use a treatment called vosoritide, which is a modified form of a natural protein in the body. Vosoritide is administered as a solution for injection and is being tested to see if it can help improve growth in children with ISS.

The purpose of the study is to evaluate the effects of different doses of vosoritide compared to a placebo over a period of six months. In the long-term phase, the study will compare the effects of vosoritide with human growth hormone (hGH) on children's height and growth patterns. The study will involve regular assessments of the children's growth, including changes in height and growth rates, over a period of up to four years.

Participants in the study will receive either vosoritide or a placebo through subcutaneous injections, which means the injection is given under the skin. The study aims to find the most effective dose of vosoritide and to understand its long-term benefits and safety for children with ISS. Regular monitoring will be conducted to track any side effects and to measure changes in growth and development.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Upon joining the study, participants will be randomly assigned to receive one of three doses of vosoritide or a placebo. This is part of the dose-finding phase, which aims to evaluate the effect of different doses on growth after six months of treatment.
Step 2
Dose administration
Participants will receive vosoritide through a subcutaneous injection, which means the medication is injected under the skin. The specific dose and frequency will be determined by the study team. The treatment will continue for six months during the dose-finding phase.
Step 3
Long-term treatment phase
After the initial six months, participants will enter the long-term phase of the study. During this phase, the effect of the therapeutic dose of vosoritide will be compared to human growth hormone (hGH) on height and height Z-score over a longer period.
Step 4
Regular assessments
Throughout the study, participants will undergo regular assessments to monitor changes in height and other health parameters. These assessments will help determine the effectiveness and safety of the treatment.
Step 5
End of study
The study is expected to conclude by July 24, 2040. At the end of the study, participants will have a final assessment to evaluate the overall impact of the treatment on their growth and health.

Who can join the trial?

3 criteria

The patient must have a height that is significantly shorter than average for their age and sex. This is measured using a special chart from the Centers for Disease Control and Prevention (CDC), and the height must be at or below a certain level, called a height Z-score of ≤ -2.25 SDs.
The patient must be at Tanner Stage 1, which means they are in the early stage of physical development during puberty. If the patient is too young to determine this stage, it may not be required.
The patient must be at least 3 years old but younger than 10 years old if they are female, or younger than 11 years old if they are male, at the time of signing the informed consent form.

Who cannot join the trial?

7 criteria

Patients with any medical condition other than Idiopathic Short Stature (ISS) cannot participate. Idiopathic Short Stature means being shorter than most people of the same age and gender without a known cause.
Patients who are not within the specified age range for the study cannot participate. The study is open to children and adolescents.
Patients who are not willing or able to follow the study procedures cannot participate.
Patients who have participated in another clinical trial recently may not be eligible.
Patients with any condition that the study doctors believe would make it unsafe for them to participate cannot join the study.
Patients who are pregnant or breastfeeding cannot participate.
Patients who are taking certain medications that might interfere with the study treatment cannot participate.

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Verified sites

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

Verified
Assistance Publique Hopitaux De Marseille
Marseille, France
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Bicetre Hospital
Le Kremlin-Bicetre, France
Verified
Centre Hospitalier Regional D'Angers
Angers, France

Trial locations

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Investigated drugs

Vosoritide
is a medication being studied for its potential to help children with idiopathic short stature grow taller. It works by targeting specific pathways in the body that are involved in bone growth. The goal of using vosoritide in this trial is to see if it can increase the growth rate in children over a period of time, helping them achieve a height closer to the average for their age.
Human Growth Hormone (hGH)
is a therapy that has been used for many years to help children with growth issues. It is a natural hormone that the body produces to stimulate growth and cell reproduction. In this trial, hGH is used as a comparison to see how effective vosoritide is in promoting growth over the long term. The study aims to determine if vosoritide can be a better or complementary option to hGH for increasing height in children with idiopathic short stature.

Vosoritide

What is already known about the treatment

Vosoritide – Vosoritide is administered as an injection under the skin, typically in the upper arm, thigh, or abdomen. It is currently being studied in clinical trials for its potential to treat children with Idiopathic Short Stature (ISS), a condition where children are significantly shorter than their peers without a known cause. Vosoritide works by targeting a specific pathway in the body that affects bone growth, helping to promote increased height. It is classified as a growth modulator, and ongoing research is evaluating its effectiveness compared to other treatments like human growth hormone (hGH).

Investigated diseases

Idiopathic Short Stature (ISS) – Idiopathic Short Stature is a condition characterized by a height significantly below the average for a child's age and sex, without any identifiable medical cause. Children with ISS grow at a normal rate but remain shorter than their peers. The growth pattern is consistent with familial short stature or constitutional growth delay, but without a clear familial or developmental reason. The condition is diagnosed when a child's height is more than two standard deviations below the mean for their age and sex. ISS is not associated with any other health problems or syndromes. The growth rate may improve during puberty, but the final adult height is often shorter than average.