We are committed to pursuing research to address the needs of the achondroplasia community

Our clinical program includes multiple studies that are grounded in learning more about achondroplasia and evaluating an oral treatment option for this skeletal dysplasia

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What is being studied?

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

Genetic variants that cause FGFR3 overactivity can lead to skeletal conditions such as achondroplasia

Learn more about the process required to develop a new treatment option

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The PROPEL clinical study program

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

The study program section title mobile image clinical study program

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:

  • Children with achondroplasia 2.5 years to <17 years in age (at screening)
  • Eligible children may participate in PROPEL for a minimum of 6 months to a maximum of 2 years

Goals of the observational study are:

  • To learn more about overall health, growth, and possible medical complications in children with achondroplasia
study program section title mobile image

Eligibility and study duration:

  • Children with achondroplasia ages 3 to 11 years who completed at least 6 months in the PROPEL study
  • Eligible children may receive the investigational treatment for up to 18 months in PROPEL 2
  • After completion of the PROPEL 2 study, eligible children may rollover to the PROPEL Open Label Extension

Goals of the observational study are:

  • To find the most appropriate dose of the investigational treatment to use in children with achondroplasia
  • To learn more about the safety and what side effects may occur with the treatment under investigation
  • To learn how it may affect growth, symptoms, medical complications, and well-being in children with achondroplasia
study program section title mobile image

Eligibility and study duration:

  • Children who complete PROPEL 2 may be given the option to continue to receive the investigational treatment until they complete their growth

Goals of the observational study are:

  • To further explore the effect of this investigational treatment on overall health, growth, and possible medical complications
study program table header image study program table header image study program table header image

Eligibility and study duration:

  • Children with achondroplasia 2.5 years to <17 years (at screening)
  • Eligible children may participate in PROPEL for a minimum of 6 months to a maximum of 2 years

Eligibility and study duration:

  • Children with achondroplasia ages 3 to 11 years who completed at least 6 months in the PROPEL study
  • Eligible children may receive the investigational treatment for up to 18 months in PROPEL 2
  • After completion of the PROPEL 2 study, eligible children may rollover to the PROPEL Open Label Extension

Eligibility and study duration:

  • Children who complete PROPEL 2 may be given the option to continue to receive the investigational treatment until they complete their growth

Goals of the observational study are:

  • To learn more about overall health, growth, and possible medical complications in children with achondroplasia

Goals of the study with infigratinib are:

  • To find the most appropriate dose of the investigational treatment to use in children with achondroplasia
  • To learn more about the safety and what side effects may occur with the treatment under investigation
  • To learn how it may affect growth, symptoms, medical complications, and well-being in children with achondroplasia

Goals of the study with infigratinib are:

  • To further explore the effect of this investigational treatment on overall health, growth, and possible medical complications

We are planning for a Phase 3 study in children with achondroplasia

This is the next step in the process of developing a new treatment option

Outcomes being studied in the PROPEL clinical program:

study program table header image

Information about achondroplasia is collected without treatment

What is being studied?

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)

Growth check icon

Medical symptoms and complications (middle ear infections, sleep apnea, spinal stenosis, spinal surgery)

Growth check icon

Well-being measurements (health-related pain and functional independence questionnaires)

Growth check icon

How the body interacts with the treatment under investigation after oral dosing as it moves through the body (pharmacokinetics)

No treatment is used

Study program table header image

Children with achondroplasia are given different doses of the investigational treatment

What is being studied?

What is the most appropriate dose of the investigational treatment?

Growth check icon

Safety and tolerability (side effects)

Growth check icon

Growth measurements (height, limb length)

Growth check icon

Medical symptoms and complications (middle ear infections, sleep apnea, spinal stenosis, spinal surgery)

Growth check icon

Well-being measurements (health-related pain and functional independence questionnaires)

Growth check icon

How the body interacts with the treatment under investigation after oral dosing as it moves through the body (pharmacokinetics)

Growth check icon
Study program table header image

Children who complete PROPEL 2 may be given the option to continue to receive the investigational treatment until they complete their growth

What is being studied?

What is the most appropriate dose of the investigational treatment?

The dose will be selected in PROPEL 2

Safety and tolerability (side effects)

Growth check icon

Growth measurements (height, limb length)

Growth check icon

Medical symptoms and complications (middle ear infections, sleep apnea, spinal stenosis, spinal surgery)

Growth check icon

Well-being measurements (health-related pain and functional independence questionnaires)

Growth check icon

How the body interacts with the treatment under investigation after oral dosing as it moves through the body (pharmacokinetics)

Not assessed

Nothing Here
What is being studied?
What is being studied card image

Information about achondroplasia is collected without treatment

What is being studied card image

Children with achondroplasia are given different doses of the investigational treatment

What is being studied card image

Children who complete PROPEL 2 may be given the option to continue to receive the investigational treatment until they complete their growth

What is being studied?

What is the most appropriate dose of the investigational treatment?

No treatment is used

Growth check icon

The dose will be selected in PROPEL 2

Safety and tolerability (side effects)

No treatment is used

Growth check icon Growth check icon

Growth measurements (height, limb length)

Growth check icon Growth check icon Growth check icon

Medical symptoms and complications (middle ear infections, sleep apnea, spinal stenosis, spinal surgery)

Growth check icon Growth check icon Growth check icon

Well-being measurements (health-related pain and functional independence questionnaires)

Growth check icon Growth check icon Growth check icon

How the body interacts with the treatment under investigation after oral dosing as it moves through the body (pharmacokinetics)

No treatment is used

Growth check icon

Not assessed

We are committed to sharing what we learn to help with care decisions and to support individuals with achondroplasia

News and updates

References section title icon References section title icon open

References

  1. Guagnano V et al. Discovery of 3-(2,6-Dichloro-3,5-dimethoxy-phenyl)-1-{6-[4-(4-ethyl-piperazin-1-yl)-phenylamino]-pyrimidin-4-yl}-1-methyl-urea (NVP-BGJ398), a potent and selective inhibitor of the fibroblast growth factor receptor family of receptor tyrosine kinase. J Med Chem. 2011;54(20):7066-7083.
  2. Dobscha K et al. FGFR-selective tyrosine kinase inhibitors, such as infigratinib, show potency and selectivity for FGFR3 at pharmacologically relevant doses for the potential treatment of achondroplasia. Poster accepted at: Endocrine Society 2020. Conference was cancelled amid concerns about COVID-19.
  3. Demuynck B et al. Low dose, daily or intermittent administration of infigratinib (BGJ398), a selective FGFR inhibitor, as treatment for achondroplasia in a preclinical mouse model. Poster presented at: American Society of Human Genetics, October 15-19, 2019; Houston, TX.
  4. Demuynck B et al. Support for a new therapeutic approach of using a low-dose FGFR tyrosine kinase inhibitor (infigratinib) for achondroplasia. Poster accepted at: Endocrine Society 2020. Conference was cancelled amid concerns about COVID-19.
  5. Savarirayan R et al. Infigratinib in children with achondroplasia: the PROPEL and PROPEL 2 studies. Ther Adv Musculoskelet Dis. 2022;14:1759720X221084848.
  6. Prospective clinical assessment study in children with achondroplasia (ACH). ClinicalTrials.gov identifier: NCT04035811. Updated April 8, 2022. Accessed November 11, 2022. https://clinicaltrials.gov/ct2/show/record/NCT04035811.
  7. Study of infigratinib in children with achondroplasia. ClinicalTrials.gov identifier: NCT04265651. Updated April 7, 2022. Accessed November 11, 2022. https://clinicaltrials.gov/ct2/show/NCT04265651.
  8. Extension study of infigratinib in children with achondroplasia (ACH). ClinicalTrials.gov identifier: NCT05145010. Updated April 7, 2022. Accessed November 11, 2022. https://clinicaltrials.gov/ct2/show/NCT05145010.