Our clinical program includes multiple studies that are grounded in learning more about achondroplasia and evaluating an oral treatment option for this skeletal dysplasia
Genetic variants that cause FGFR3 overactivity can lead to skeletal conditions such as achondroplasia. We are studying whether an investigational agent (infigratinib) has the potential to improve the irregular bone growth in achondroplasia by decreasing the overactivity of the FGFR3, thereby targeting the condition at its source.1-5
Infigratinib is not currently approved for the treatment of achondroplasia by the U.S. Food and Drug Administration (FDA) or any other health authority.
It is the only oral agent under investigation for achondroplasia and is currently being studied in the PROPEL clinical program (see below for study details).5-8
Our clinical trials are currently underway and may change over time. PROPEL is an observational study to collect information about achondroplasia.6 Participants who complete PROPEL may enroll in investigational studies of oral infigratinib sponsored by QED Therapeutics.7,8
Our clinical trials are currently underway and may change over time. PROPEL is an observational study to collect information about achondroplasia.6 Participants who complete PROPEL may enroll in investigational studies of oral infigratinib sponsored by QED Therapeutics.7,8
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Eligibility and study duration:
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Eligibility and study duration:
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Eligibility and study duration:
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Goals of the observational study are:
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Goals of the study with infigratinib are:
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Goals of the study with infigratinib are:
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This is the next step in the process of developing a new treatment option
Outcomes being studied in the PROPEL clinical program:
Information about achondroplasia is collected without treatment
What is the most appropriate dose of the investigational treatment?
No treatment is used
Safety and tolerability (side effects)
No treatment is used
Growth measurements (height, limb length)
Medical symptoms and complications (middle ear infections, sleep apnea, spinal stenosis, spinal surgery)
Well-being measurements (health-related pain and functional independence questionnaires)
How the body interacts with the treatment under investigation after oral dosing as it moves through the body (pharmacokinetics)
No treatment is used
Children with achondroplasia are given different doses of the investigational treatment
What is the most appropriate dose of the investigational treatment?
Safety and tolerability (side effects)
Growth measurements (height, limb length)
Medical symptoms and complications (middle ear infections, sleep apnea, spinal stenosis, spinal surgery)
Well-being measurements (health-related pain and functional independence questionnaires)
How the body interacts with the treatment under investigation after oral dosing as it moves through the body (pharmacokinetics)
Children who complete PROPEL 2 may be given the option to continue to receive the investigational treatment until they complete their growth
What is the most appropriate dose of the investigational treatment?
The dose will be selected in PROPEL 2
Safety and tolerability (side effects)
Growth measurements (height, limb length)
Medical symptoms and complications (middle ear infections, sleep apnea, spinal stenosis, spinal surgery)
Well-being measurements (health-related pain and functional independence questionnaires)
How the body interacts with the treatment under investigation after oral dosing as it moves through the body (pharmacokinetics)
Not assessed
What is being studied? |
![]() Information about achondroplasia is collected without treatment |
![]() Children with achondroplasia are given different doses of the investigational treatment |
![]() Children who complete PROPEL 2 may be given the option to continue to receive the investigational treatment until they complete their growth |
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What is being studied? |
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What is the most appropriate dose of the investigational treatment? |
No treatment is used |
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The dose will be selected in PROPEL 2 |
Safety and tolerability (side effects) |
No treatment is used |
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Growth measurements (height, limb length) |
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Medical symptoms and complications (middle ear infections, sleep apnea, spinal stenosis, spinal surgery) |
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Well-being measurements (health-related pain and functional independence questionnaires) |
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How the body interacts with the treatment under investigation after oral dosing as it moves through the body (pharmacokinetics) |
No treatment is used |
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Not assessed |