Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia
This research is specifically designed for children who have hypochondroplasia. This is a condition that affects bone growth, leading to shorter limbs. The study is a continuation of previous research and aims to understand how safe and effective a medicine called vosoritide is over a longer period when taken daily. Doctors will watch how participants grow and check for any side effects. This information will help us learn more about how vosoritide can help children with hypochondroplasia in the long run. Participants must have already completed an earlier study on this medication.
במבט חטוף
על מה המחקר הזה?
This study is a long-term look at a medicine called vosoritide for children who have hypochondroplasia. Hypochondroplasia is a condition that affects how bones grow, particularly in the arms and legs, making them shorter than usual. The main goal of this study is to see how safe vosoritide is and how well it works over a longer period of time when given every day.
Children taking part in this study have already been involved in an earlier study about vosoritide. Because these conditions are rare, it's really important to gather as much information as possible over time to properly understand how new treatments work and what their effects are. By taking part, families are helping doctors and scientists learn more about new ways to support children with hypochondroplasia now and in the future.
This research is important because it builds on what we already know. It helps to ensure that if vosoritide is approved for wider use, doctors will have a complete picture of its long-term benefits and any potential concerns. This means families can be more confident in the treatment options available for their children.
נקודות עיקריות
- Study for children with hypochondroplasia who previously participated in a vosoritide trial.
- Aims to understand the long-term safety and effectiveness of daily vosoritide.
- Participation involves daily injections and regular clinic visits for checks.
- Opportunity to contribute to understanding a new treatment for hypochondroplasia.
- Voluntary participation with the right to withdraw at any time.
מי עשוי להיות זכאי?
To join this study, children must have already completed one of two previous studies (called 111-303 or 111-212) on vosoritide. They also need to have open growth plates in their bones, which means they are still growing. Their parents or guardians must be willing to give permission and ensure their child follows all study instructions.
If a girl is 10 years old or older, or has started her periods, she will need to have pregnancy tests during the study. If she is sexually active, she must agree to use effective birth control. Parents or guardians must be able to give daily injections to the child and be willing to be trained on how to do this.
There are also some reasons why someone couldn't join. For example, if they stopped treatment in a previous study, their growth has slowed down a lot, or their growth plates have closed. Other reasons include planning to have surgery that affects bones or taking certain other medications. If a child has cancer, a serious allergy to the medication, or if a participant is pregnant or breastfeeding, they cannot join. The study also can't accept anyone with a health condition that would make it difficult or unsafe to take part.
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 my child already completed one of the previous vosoritide studies (111-303 or 111-212)?
- Are my child's growth plates still open (meaning they are still growing)?
- Am I, as a parent or guardian, willing and able to give daily injections and attend appointments?
- Is my child free from certain other health conditions, including cancer, or plans for bone surgery?
- If my child is female and 10+ or has started periods, are we willing to undergo pregnancy tests and use birth control if sexually active?
מה כרוך בהשתתפות?
If you decide to take part in this study, your child will receive daily injections of vosoritide, which you, as a parent or guardian, will be trained to give. You'll need to make sure your child attends regular check-ups at the study clinic. These visits will involve various tests and measurements to see how your child is growing and to check for any side effects. For example, they might have X-rays of their hand to check their bone growth. If applicable, girls will have pregnancy tests at the start and during the study. You'll also be asked to help your child complete any necessary procedures to the best of their ability. The total duration of participation will be explained in detail by the study team, but it is a long-term study following on from previous research.
סיכונים ותועלות פוטנציאליים
Locations (30)
- Phoenix Children's Hospital - Thomas Campusמיקוד מאומתפניקס, ארצות הברית
- Benioff Children's Hospital - Oaklandמיקוד מאומתאוקלנד, ארצות הברית
- Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children)מיקוד מאומתוילמינגטון, ארצות הברית
- המרכז הרפואי הלאומי לילדיםמיקוד מאומתוושינגטון די.סי., ארצות הברית
- בית החולים לילדים אן ורוברט ה. לורי בשיקגומיקוד מאומתשיקגו, ארצות הברית
- The Johns Hopkins University School of Medicineמיקוד מאומתבולטימור, ארצות הברית
- University of Minnesota Masonic Children's Hospitalמיקוד מאומתמיניאפוליס, ארצות הברית
- University of Missouriמיקוד מאומתColumbia, United States
- Cook Children's Endocrinologyמיקוד מאומתFort Worth, United States
- Medical College of Wisconsinמיקוד מאומתמילווקי, ארצות הברית
- Murdoch Children's Research Institute of The Royal Children's Hospitalמיקוד מאומתParkville, Australia
- University of Alberta Stollery Children's Hospitalמיקוד מאומתאדמונטון, קנדה
שאלות נפוצות
What is hypochondroplasia?
Hypochondroplasia is a genetic condition that affects bone growth, leading to shorter arms, legs, and overall stature.
What is vosoritide?
Vosoritide is a medicine designed to help improve bone growth in children with certain types of growth conditions, like hypochondroplasia.
Will my child definitely grow more by taking part?
While the medicine aims to help with growth, there's no guarantee of a specific outcome. The study is designed to gather more information on its long-term effects.
What if we change our minds during the study?
You are free to withdraw your child from the study at any time, for any reason, without affecting their regular medical care.
Are there any costs involved for us to participate?
The study should cover the costs related to the study medicine and appointments. Specific details will be provided by the study team.
כיצד לגלות עוד
תמיד התייעץ עם רופא המשפחה או המומחה שלך לפני שתחליט להשתתף במחקר.
מעוניינים להשתתף?
דיון
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