A Phase 2, Randomized, Multicenter, Study of Vosoritide in Children with Noonan Syndrome with Inadequate Growth During or After Human Growth Hormone Treatment
Noonan syndrome is a genetic condition that often causes children to grow much more slowly than their peers. While standard human growth hormone therapy helps many, some children do not gain enough height. This Phase 2 clinical trial is testing an experimental medicine called vosoritide (also known as Voxzogo). Vosoritide works differently from standard growth hormone by directly stimulating bone growth plates. Researchers want to find out whether daily injections of vosoritide can safely improve yearly growth rates over six months and beyond. Doctors will closely monitor participants' height, heart health, bone strength, and daily well-being. By taking part, families help doctors learn whether this medicine could become a helpful future treatment option for young people with Noonan syndrome who need extra support to grow.
במבט חטוף
על מה המחקר הזה?
Noonan syndrome is a genetic condition that affects how different parts of the body develop. One of the most common challenges is slow physical growth, which often leaves children much shorter than other young people their age. Many children receive standard human growth hormone injections to help them grow, but this treatment does not always work well enough for everyone.
This study is exploring an alternative medicine called vosoritide (marketed as Voxzogo). Vosoritide is designed to work directly on the growth plates inside bones to encourage natural bone lengthening. Doctors want to see if this treatment can restart or improve growth in children and young people whose growth has stayed slow despite trying standard growth hormone therapies.
During the trial, doctors will track how fast participants grow over six months and check if the treatment remains safe and effective until they finish growing. They will also look closely at bone density, body proportions, heart health, and how the treatment affects everyday physical activity and quality of life.
נקודות עיקריות
- Tests a medicine called vosoritide to improve growth in Noonan syndrome.
- Aimed at young people whose growth was not sufficient with standard growth hormone.
- Given as a daily injection under the skin, usually managed at home.
- Includes detailed checks on height, bone development, and heart health.
- Participation is entirely voluntary with no impact on standard NHS care.
מי עשוי להיות זכאי?
This study is designed for individuals diagnosed with Noonan syndrome who have had inadequate growth during or after standard human growth hormone treatment. Even though some trial registries list adult ages, this trial focuses on young people whose bones are still developing and have room to grow.
Both boys and girls can take part. To be suitable, participants must have open growth plates in their bones so that growth is still possible. They must also be able to attend regular clinic visits for check-ups and monitoring.
People might not be eligible if their bones have already stopped growing, if they have certain serious heart problems that need urgent medical care, or if they have other medical conditions that make daily injections or trial assessments unsafe. A study doctor will review each person's full medical history to confirm if joining is safe.
האם מחקר זה יכול להתאים לך?
ענה על השאלות המהירות הללו כדי לבדוק אם אתה עשוי להיות זכאי. זהו מדריך בלבד — צוות המחקר מקבל את ההחלטה הסופית.
- Has a confirmed diagnosis of Noonan syndrome.
- Has had slow or inadequate growth during or after human growth hormone therapy.
- Still has open growth plates with potential for further bone growth.
- Is willing and able to receive a daily injection under the skin.
- Can attend regular hospital visits for scans, blood tests, and measurements.
מה כרוך בהשתתפות?
Taking part involves receiving daily injections of vosoritide under the skin, which can usually be given at home after training from the nursing team. Participants will need to visit the hospital clinic regularly for check-ups. At these visits, doctors will measure height, arm span, and body proportions very carefully.
The team will also carry out routine health checks, including blood and urine tests, bone scans (DXA scans), and X-rays of the legs to check the growth plates. Because Noonan syndrome can affect the heart, regular heart scans (such as echocardiograms) will be done to ensure participants remain safe. Families will also be asked to fill out short questionnaires about daily activities and quality of life. The study monitors growth over six months initially, with longer-term follow-up to see how growth develops over time.
סיכונים ותועלות פוטנציאליים
מיקומים (4)
- —לא מאומתצרפת
- —לא מאומתספרד
- —לא מאומתגרמניה
- —לא מאומתאיטליה
שאלות נפוצות
כיצד ניתן ללמוד רפואה?
Vosoritide is given as an injection just under the skin once a day. The clinic team will teach families how to do this safely at home.
Does this replace standard growth hormone?
This study looks at vosoritide for young people who did not grow well enough on growth hormone, testing whether this new medicine works better for them.
Will taking part guarantee extra height?
No. Because this is a clinical trial, doctors are still learning how effective vosoritide is for Noonan syndrome, and individual results can vary.
Are the hospital visits painful?
Most visits involve gentle checks like measuring height, taking scans, and heart checks. Some visits will include standard blood tests, which may cause brief discomfort.
What happens if we want to stop the trial?
You can leave the study at any time for any reason. Doing so will never affect the standard medical care you or your child receive.
כיצד לגלות עוד
תמיד התייעץ עם רופא המשפחה או המומחה שלך לפני שתחליט להשתתף במחקר.
מעוניינים להשתתף?
דיון
דיון קהילתי
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