Achondroplasia

Achondroplasia affects approximately 1 out every 20,000 newborns, and treatment advances for children with this bone growth disorder have been limited.16,17 Children with achondroplasia reach an adult height of 4 feet 10 inches or shorter and may also experience serious, often debilitating health complications.17

FGFR genetic alterations can cause more than 99% of achondroplasia cases.18



Learn more about PROPEL and PROPEL 2 Studies of infigratinib*


  1. Infigratinib is not currently FDA approved for this indication in the U.S. and is not approved for use by any other health authority.

QED Therapeutics is currently looking to enroll children in PROPEL, a prospective clinical assessment study in children with achondroplasia. The study aims to learn more about the overall health, growth, and possible medical complications in children with achondroplasia.

200 boys and girls will participate in the study at locations worldwide. No medication will be administered; however, children successfully enrolled in PROPEL for 6 months or more may be eligible for subsequent treatment trials sponsored by QED Therapeutics.

To be considered for inclusion in the PROPEL Study, children must:

  • Be 2.5 to 10 years of age at study entry
  • Have a diagnosis of achondroplasia
  • Receive agreement from parents or caregivers to have their children participate and follow all study visit schedules and procedures
  • Other study requirements will be reviewed in detail by the study staff

Participation in the PROPEL Study is voluntary.

To participate in or learn more about PROPEL or PROPEL 2, please contact us at: PROPELstudyinfo@QEDTx.com or visit ClinicalTrials.gov.

PROPEL 2 is currently enrolling, and is an open-label, dose-escalation and dose-expansion study of infigratinib,* an FGFR1-3-selective tyrosine kinase inhibitor, in children with achondroplasia.

  1. Infigratinib is not currently FDA approved in this indication in the U.S. and is not approved for use by any other health authority.

FGFR=fibroblast growth factor receptor.

Logo for the PROPEL assessment study

References

  1. Touat M et al. Clin Cancer Res. 2015;21(12):2684-2694.
  2. Turner N et al. Nat Rev Cancer. 2010;10:116-129.
  3. Katoh M. Nat Rev Clin Oncol. 2019;16:105-122.
  4. Babina IS et al. Nat Rev Cancer. 2017;17:318-332.
  5. TRUSELTIQ Prescribing Information. Brisbane, CA: QED Therapeutics, Inc.; [Month] 2021.
  6. American Cancer Society. https://www.cancer.org/cancer/bile-duct-cancer/about/key-statistics. Accessed March 27, 2021.
  7. Lamarca A et al. J Clin Med. 2020;9(9):2854.
  8. Arai Y et al. Hepatology. 2014;59(4):1427-1434.
  9. Kongpetch S et al. JCO Global Oncol. 2020;6:628-638.
  1. Ross JS et al. Cancer. 2016;122(5):702-711.
  2. Sfakianos JP et al. Eur Urol. 2015;68(6):970-977.
  3. Moss TJ et al. Eur Urol. 2017;72(4):641-649.
  4. Bagrodia A et al. J Urol. 2016;195(6):1684-1689.
  5. Bagrodia A et al. Eur Urol Focus. 2019;5(3):365-368.
  6. Audenet F et al. Clin Cancer Res. 2019;25(3):967-976.
  7. Komla-Ebri D et al. J Clin Invest. 2016;126(5):1871-1884.
  8. National Institutes of Health. https://ghr.nlm.nih.gov/condition/achondroplasia. Accessed April 1, 2021.
  9. Legare JM et al. https://www.ncbi.nlm.nih.gov/books/NBK1152/. Accessed April 1, 2021.

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