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Ongoing Clinical Trials for Noonan Syndrome

There are currently 3 ongoing clinical trials investigating treatments for children with Noonan syndrome. These studies focus on improving growth in children who experience short stature as part of this condition. The trials are testing medications called vosoritide and somapacitan, comparing them to existing growth hormone treatments. These studies are taking place across multiple European countries and involve children who have not responded adequately to standard growth hormone therapy or who have not yet received growth-promoting treatments.

Clinical trial locations

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

This trial is investigating vosoritide as a potential treatment for children with growth-related genetic conditions, including Noonan syndrome. The study is taking place in Germany, France, Spain, and Italy.

Who can participate:

Children eligible for this study must be between 3 and 10 years old for girls, or between 3 and 11 years old for boys. They must have a genetically confirmed diagnosis of Turner syndrome, SHOX deficiency, or Noonan syndrome. Participants must have a height Z-score of -2.00 or lower, meaning they are significantly shorter than average for their age and gender. They must be at Tanner Stage 1, which indicates they have not yet started puberty. Importantly, children must have been receiving continuous human growth hormone treatment for at least one year before joining the study, and must have shown an inadequate response to this treatment.

Who cannot participate:

Children who do not have one of the three specified genetic conditions cannot join the study. The trial is open to both boys and girls within the specified age ranges. Participants from vulnerable populations who cannot give informed consent are excluded.

Main focus of the study:

The study aims to evaluate whether vosoritide can improve growth in children who have not responded well to human growth hormone therapy. Researchers will compare the effects of different doses of vosoritide with continued growth hormone treatment over a period extending up to 24 months. The primary measure will be changes in height and height Z-scores. The study will also monitor safety by tracking any side effects that occur during treatment.

Investigational medications:

Vosoritide is the main medication being tested. It is a C-type natriuretic peptide analog that works by targeting a specific pathway involved in regulating bone growth. It is given as an injection under the skin. Human growth hormone continues to be used in the comparison group to measure how vosoritide performs against the current standard treatment.

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 trial is comparing a once-weekly treatment called somapacitan with a daily treatment called Norditropin for children with various causes of short stature, including Noonan syndrome. The study is being conducted across 16 European countries: Germany, Italy, Latvia, Croatia, Bulgaria, France, Greece, Belgium, Finland, Netherlands, Portugal, Poland, Lithuania, Slovenia, Spain, and Austria.

Who can participate:

Children must not have received any previous growth-promoting treatments. For boys with Noonan syndrome, they must be at least 2 years and 26 weeks old but younger than 11 years at the start of the study, and their testis volume must be below 4 mL. For girls with Noonan syndrome, they must be at least 2 years and 26 weeks old but younger than 10 years, and must not have started breast development. All children with Noonan syndrome must have a clinical diagnosis according to a specific scoring system. The study also includes children with other conditions such as Turner syndrome, being born small for gestational age, or idiopathic short stature, with similar age and developmental requirements.

Who cannot participate:

Children who are not diagnosed with one of the specified short stature conditions cannot participate. Those outside the specified age ranges, those taking medications that could affect growth, those who have had previous growth hormone therapy, those with allergies to the study medication, or those with other medical conditions that might interfere with the study are excluded. Children who cannot comply with study procedures are also not eligible.

Main focus of the study:

The primary goal is to determine whether once-weekly somapacitan is as effective as daily Norditropin in promoting growth in children with these conditions. The study will measure height velocity, which is the rate at which children grow over time. Researchers will also monitor bone age changes, blood glucose levels, and other health indicators to ensure the safety of the treatment. The study aims to provide valuable information on whether somapacitan can offer a more convenient alternative to daily injections while maintaining effectiveness.

Investigational medications:

Somapacitan is given once weekly via injection under the skin using a pre-filled pen device. It is available in doses of 5 mg, 10 mg, or 15 mg per 1.5 mL solution. Somapacitan works by mimicking natural growth hormone, stimulating growth and cell reproduction. Norditropin, the comparison medication, is given daily via injection and is an established treatment that replaces or supplements natural growth hormone.

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 is evaluating the safety and effectiveness of somapacitan in children with growth difficulties due to various conditions, including Noonan syndrome. The study is being conducted in Poland, Spain, and the Netherlands.

Who can participate:

Children with Noonan syndrome must have a diagnosis according to local clinical practice. Male and female participants must be within a specific age range at the time of screening. Importantly, participants must have open epiphyses, which means the growth plates in their bones are still open, allowing for continued growth. The study also includes children born small for gestational age and those with Turner syndrome or idiopathic short stature, all meeting similar requirements regarding open growth plates and appropriate age ranges.

Who cannot participate:

Children with a history of any significant medical condition that could interfere with the study are excluded. Those who have received any other growth hormone treatment within the last 6 months cannot participate. Children with known allergies to the study medication or its ingredients, those currently participating in another clinical trial, those with a history of cancer or other serious illness, and those unable to comply with study procedures are not eligible.

Main focus of the study:

The study aims to evaluate how safe somapacitan is and how well it works in helping children with these specific conditions grow. Participants receive the medication once a week via injection under the skin. The study monitors the number of any adverse events over an extended period, up to 156 weeks. Researchers will track changes in the children’s height during the study, with regular follow-up visits to monitor both growth progress and any side effects.

Investigational medications:

Somapacitan is administered as a 15 mg dose in 1.5 mL of solution, given once weekly as an injection under the skin using a special pen-injector designed for single patient use. The medication works by mimicking natural growth hormone in the body, helping to stimulate growth in children who have a deficiency or need additional growth support.

Summary

The three ongoing clinical trials for Noonan syndrome all focus on addressing short stature, one of the most common challenges faced by children with this condition. Two of the trials are investigating somapacitan, a once-weekly growth hormone treatment, while one trial is studying vosoritide, a medication with a different mechanism of action that targets bone growth pathways.

The trials show a concentration of study sites across Europe, with the largest trial operating in 16 countries. Germany, France, Italy, Spain, Poland, and the Netherlands appear in multiple trials, suggesting these countries have strong research infrastructure for pediatric growth disorders. The vosoritide study specifically targets children who have already tried standard growth hormone therapy without adequate results, representing an important research area for patients who need alternative treatment options.

All three studies require careful screening to ensure participants have the appropriate diagnosis, are in the correct age range, and still have potential for growth as indicated by open growth plates. The duration of these studies ranges from several months to up to three years, reflecting the time needed to properly assess changes in growth patterns and ensure long-term safety. These trials represent important steps in expanding treatment options for children with Noonan syndrome who experience growth challenges.

Ongoing Clinical Trials on Noonan syndrome

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

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    France Germany Italy 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

    Not recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    Austria Belgium Bulgaria Croatia Finland France +10

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