QED - Propel study Q&A
QED is developing a medicine which may improve the overall health and quality of life of people with achondroplasia. This investigational medicine is called infigratinib (formerly called BGJ398) and may be an option for children to take while they are still growing. Infigratinib is a small tablet taken by mouth that works by reducing the activity of proteins called fibroblast growth factor receptors, or FGFRs. Because increased activity in one of the FGFRs – FGFR3 – leads to the changes in bone development associated with achondroplasia, limiting its activity may have benefits for children with achondroplasia.
Other conditions can occur as a result of different changes to the FGFRs, which is why infigratinib is also being studied (at higher doses) as a potential treatment for rare forms of cancer in adults. While the exact way infigratinib works to treat cancer is different from how it works in achondroplasia, these studies may give us an understanding of infigratinib’s safety and side effects in adults with cancer.
Studies using low doses of infigratinib in animals have shown an ability to improve irregular bone growth. Now, QED and its scientific partners are doing more research in animals to better understand how infigratinib may work in children with achondroplasia.
In the meantime, QED has started a research study called PROPEL that seeks to better understand the growth patterns and possible medical complications among children with achondroplasia by observing changes over time with routine medical care. Much will be learned through PROPEL even though no medicine will be administered in the study. For more information and a list of open sites, visit www.clinicaltrials.gov using identifier NCT04035811.
In 2020 QED plans to start the first clinical trial that studies the investigational medicine infigratinib in achondroplasia, which will explore the safety and potential effectiveness of infigratinib in children with achondroplasia. Children who have participated in the PROPEL study for at least six months may be eligible to participate in this or other future trials with infigratinib.
What is the PROPEL study?
PROPEL is an ongoing natural history study. “Natural history” is a term used to describe a study that observes changes in a person’s condition over time with routine medical care. PROPEL is a research study that seeks to better understand the growth patterns and possible medical complications among children with achondroplasia. No medicine will be administered to children who participate in this study.
The PROPEL study opened in July 2019 and will enroll approximately 200 children from around the world over multiple years. More information can be found at www.clinicaltrials.gov using the identifier NCT04035811. Children who participate in PROPEL may later be able to join future QED clinical trials studying a new investigational medicine for children with achondroplasia called infigratinib (previously known as BGJ398).
If you are interested in enrolling your child in the PROPEL study, talk to your child’s doctor. Additional information, including a list of participating doctors, can be found by visiting www.clinicaltrials.gov and searching for the identifier NCT04035811.
The PROPEL study will be conducted around the world, but not necessarily in every country. For the most up-to-date information and a list of participating doctors, visit www.clinicaltrials.gov and search for the identifier NCT04035811.
Your child may still be able to participate in PROPEL even if your child’s current doctor is not. For more information, email This email address is being protected from spambots. You need JavaScript enabled to view it. and someone from QED will help you explore the next steps.
Children participating in PROPEL will undergo regular doctor visits to check on their growth and overall health. A few visits will be scheduled within the first 6 months of participation, with regular check-ups every 6 months thereafter.
Study visits during PROPEL will provide the information QED hopes to learn about the growth and development of children with achondroplasia, including how quickly a child grows and more about the overall health and experience of a young person with achondroplasia. Each visit will include a physical exam, height measurements of multiple body parts, a blood sample, and questionnaires to understand your child’s – and your – quality of life.
Participation in the PROPEL study occurs at no charge to the child or family and is completely voluntary.
Study participation is anonymous and voluntary. You and your child may withdraw from participation in the study at any time. Otherwise, participants remain in the study for a minimum of six months and a maximum of two years. If your child has participated in the PROPEL study for at least six months, he or she may be eligible to participate in a later clinical trial that uses the investigational medicine infigratinib.
The first clinical trial that studies the investigational medicine infigratinib in children with achondroplasia is planned to begin in 2020. If your child has participated in the PROPEL study for at least six months, he or she may be eligible to participate in a clinical trial using the investigational medicine infigratinib. Ask your doctor to learn more and to explore whether your child may be eligible to participate in a clinical trial with the investigational medicine infigratinib.
Note: The safety and efficacy of infigratinib has not been established. There is no guarantee that infigratinib will receive health authority approval or become commercially available in any country for the uses being investigated.
Other conditions can occur as a result of different changes to the FGFRs, which is why infigratinib is also being studied (at higher doses) as a potential treatment for rare forms of cancer in adults. While the exact way infigratinib works to treat cancer is different from how it works in achondroplasia, these studies may give us an understanding of infigratinib’s safety and side effects in adults with cancer.
Studies using low doses of infigratinib in animals have shown an ability to improve irregular bone growth. Now, QED and its scientific partners are doing more research in animals to better understand how infigratinib may work in children with achondroplasia.
In the meantime, QED has started a research study called PROPEL that seeks to better understand the growth patterns and possible medical complications among children with achondroplasia by observing changes over time with routine medical care. Much will be learned through PROPEL even though no medicine will be administered in the study. For more information and a list of open sites, visit www.clinicaltrials.gov using identifier NCT04035811.
In 2020 QED plans to start the first clinical trial that studies the investigational medicine infigratinib in achondroplasia, which will explore the safety and potential effectiveness of infigratinib in children with achondroplasia. Children who have participated in the PROPEL study for at least six months may be eligible to participate in this or other future trials with infigratinib.
Q&A about the PROPEL study
What is the PROPEL study?
PROPEL is an ongoing natural history study. “Natural history” is a term used to describe a study that observes changes in a person’s condition over time with routine medical care. PROPEL is a research study that seeks to better understand the growth patterns and possible medical complications among children with achondroplasia. No medicine will be administered to children who participate in this study. The PROPEL study opened in July 2019 and will enroll approximately 200 children from around the world over multiple years. More information can be found at www.clinicaltrials.gov using the identifier NCT04035811. Children who participate in PROPEL may later be able to join future QED clinical trials studying a new investigational medicine for children with achondroplasia called infigratinib (previously known as BGJ398).
How can your child be enrolled in the PROPEL study?
If you are interested in enrolling your child in the PROPEL study, talk to your child’s doctor. Additional information, including a list of participating doctors, can be found by visiting www.clinicaltrials.gov and searching for the identifier NCT04035811.
Is QED conducting a study for children with achondroplasia in your living area?
The PROPEL study will be conducted around the world, but not necessarily in every country. For the most up-to-date information and a list of participating doctors, visit www.clinicaltrials.gov and search for the identifier NCT04035811.
Your child’s doctor is not included in the list of participating doctors for PROPEL. Can your child still participate?
Your child may still be able to participate in PROPEL even if your child’s current doctor is not. For more information, email This email address is being protected from spambots. You need JavaScript enabled to view it. and someone from QED will help you explore the next steps.
How often will you need to bring your child to the doctor for assessments in the PROPEL study?
Children participating in PROPEL will undergo regular doctor visits to check on their growth and overall health. A few visits will be scheduled within the first 6 months of participation, with regular check-ups every 6 months thereafter.
What happens at the study visits during PROPEL?
Study visits during PROPEL will provide the information QED hopes to learn about the growth and development of children with achondroplasia, including how quickly a child grows and more about the overall health and experience of a young person with achondroplasia. Each visit will include a physical exam, height measurements of multiple body parts, a blood sample, and questionnaires to understand your child’s – and your – quality of life.
Is there a cost for your child to participate in the PROPEL study?
Participation in the PROPEL study occurs at no charge to the child or family and is completely voluntary.
How long will my child participate in the PROPEL study?
Study participation is anonymous and voluntary. You and your child may withdraw from participation in the study at any time. Otherwise, participants remain in the study for a minimum of six months and a maximum of two years. If your child has participated in the PROPEL study for at least six months, he or she may be eligible to participate in a later clinical trial that uses the investigational medicine infigratinib.
When will QED start a trial with the investigational medicine infigratinib?
The first clinical trial that studies the investigational medicine infigratinib in children with achondroplasia is planned to begin in 2020. If your child has participated in the PROPEL study for at least six months, he or she may be eligible to participate in a clinical trial using the investigational medicine infigratinib. Ask your doctor to learn more and to explore whether your child may be eligible to participate in a clinical trial with the investigational medicine infigratinib.
Note: The safety and efficacy of infigratinib has not been established. There is no guarantee that infigratinib will receive health authority approval or become commercially available in any country for the uses being investigated.
