Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)
This study is looking at a new medicine called infigratinib for very young children (under 3 years old) who have achondroplasia. Achondroplasia is a condition that affects bone growth. The main aim is to find out the safest and most effective dose of infigratinib and to see if it helps these children grow better. Some children taking part will receive the active medicine, while others will receive a dummy pill (placebo) to help doctors understand the effects of the treatment. The medicine comes as small mini-tablets or sprinkle capsules that can be mixed with food. This research involves different stages to carefully check the medicine's effects.
At a glance
What is this study about?
Achondroplasia is a condition that affects how bones grow, leading to shorter limbs. This research study is testing a new medicine called infigratinib, which aims to help improve bone growth in very young children with achondroplasia. The study focuses on children under 3 years old because early treatment might have the biggest impact on their development.
The study is called 'PROPEL Infant & Toddler' and is being done over several stages. Initially, a small number of children will receive different doses of infigratinib to help researchers find the best and safest amount to use. After that, a larger group of children will take part, where some will receive the active medicine and others will receive a dummy pill (called a placebo). This comparison helps doctors understand if the medicine is truly making a difference. The medicine will be given by mouth, either as tiny tablets or sprinkle capsules that can be mixed with food.
Throughout the study, doctors will carefully watch the children to see how well the medicine works and if there are any side effects. This kind of research is really important because it helps scientists learn more about new treatments and how they might improve the lives of children with achondroplasia. By taking part, families contribute to future medical knowledge and potential new treatment options.
Key takeaways
- This study is testing a new medicine, infigratinib, for very young children with achondroplasia.
- It aims to find the safest and most effective dose to help with bone growth.
- Some children will receive the active medicine, while others will get a dummy pill.
- The medicine is given by mouth as tiny tablets or sprinkle capsules.
- Regular hospital visits and health checks will be needed to monitor your child.
- Your child's participation could help future children with achondroplasia.
Who may be eligible?
To be part of this study, your child needs to have achondroplasia, which must be confirmed by a genetic test. They also need to be very young, specifically between birth and 2 years and 8 months old when they first join. Their parent or legal guardian must be happy to sign a consent form, attend all study appointments, and follow the study's requirements, including giving the child medication as instructed.
Your child also needs to be able to swallow medicine by mouth that's suitable for their age. If your child is under one year old, they should already be taking the recommended vitamin D supplements. It's important that your child doesn't have any other conditions that might affect their growth or any serious health problems that could make the study unsafe or difficult.
For example, children won't be able to join if they have other genetic conditions that affect growth, were born very early or with a very low birth weight, have severe reflux, or have certain issues with their neck bones. Also, if they've broken a long bone or had a spinal fracture in the last six months, they won't be able to take part.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Is your child between birth and 2 years and 8 months old?
- Has your child been diagnosed with achondroplasia using a genetic test?
- Can your child swallow age-appropriate medicine by mouth?
- Are you able to bring your child to all study appointments and give them medicine as directed?
- Does your child currently have any other serious health conditions or recent bone breaks?
What does participation involve?
If your child takes part in this study, they will receive the study medicine (infigratinib) or a dummy pill (placebo) every day. This medicine comes as tiny mini-tablets or sprinkle capsules that can be mixed with food and swallowed. Throughout the study, your child will have regular check-ups at the hospital or clinic. These visits will involve various assessments, such as growth measurements, blood tests, and possibly scans like MRI, to see how the medicine is working and to monitor their health.
The study has different parts: an initial open-label part where everyone gets the medicine to find the right dose, then a part where some get the medicine and others get the dummy pill, and finally an extended part where all children may receive the active medicine. You will need to attend all scheduled appointments and help your child take their medicine correctly. The total length of your child's participation will depend on which part of the study they are in, but it will involve ongoing follow-up for a period of time.
Potential risks and benefits
Locations (13)
- UCSF Benioff Children's HospitalVerified postcodeOakland, United States· Recruiting
- Johns Hopkins UniversityVerified postcodeBaltimore, United States· Recruiting
- University of Wisconsin Madison - Waisman Center Bone Dysplasia ClinicVerified postcodeMadison, United States· Recruiting
- Murdoch Children's Research InstituteVerified postcodeParkville, Australia· Recruiting
- Children's Hospital of Eastern Ontario Research InstituteVerified postcodeOttawa, Canada· Recruiting
- Université de Montréal - Centre Hospitalier Universitaire Sainte-JustineVerified postcodeMontreal, Canada· Recruiting
- Paediatric Clinical Research Unit at Oslo University HospitalVerified postcodeOslo, Norway· Recruiting
- KK Women's and Children's HospitalVerified postcodeSingapore, Singapore· Recruiting
- Unidad de Cirugia Artroscopica (UCA)Verified postcodeVitoria-Gasteiz, Spain· Not yet recruiting
- NHS Greater Glasgow and ClydeVerified postcodeGlasgow, United Kingdom· Not yet recruiting
- Guy's and Saint Thomas' NHS Foundation TrustVerified postcodeLondon, United Kingdom· Not yet recruiting
- Manchester University NHS Foundation TrustVerified postcodeManchester, United Kingdom· Not yet recruiting
Common questions
What is achondroplasia?
Achondroplasia is a genetic condition that affects bone growth, leading to shorter arms and legs, and other specific features.
What is infigratinib?
Infigratinib is a new medicine being tested to see if it can help improve bone growth in children with achondroplasia.
Will my child definitely get the medicine?
In some parts of the study, children will get the active medicine. In other parts, some children will get the active medicine and others will get a dummy pill (placebo).
Is the medicine easy to give?
The medicine comes as mini-tablets or sprinkle capsules that can be mixed with food, making it easier for very young children to take.
How long will the study last?
The study has several stages, and your child's participation could last for some time, with regular check-ups to monitor their progress and safety.
How to find out more
QED Therapeutics Inc.
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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