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

Smiling family portrait with mother, father, and a young child with achondroplasia

What is being studied?

Genetic variants that cause fibroblast growth factor receptor 3 (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 directly impacting FGFR3 overactivity, the underlying cause of the condition.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).5-9

The PROPEL clinical program is studying a wide range of outcomes to better understand the holistic impact of treating achondroplasia

The PROPEL clinical program: A holistic approach to understanding achondroplasia

The PROPEL clinical program is studying a wide range of outcomes related to growth, medical challenges, and quality of life in children and adolescents with achondroplasia. PROPEL is an observational study, in which no treatment is given. PROPEL 2 and PROPEL 3 are interventional studies, in which participants receive infigratinib and/or placebo. A placebo is a treatment with no active properties, such as a sugar pill. Participants from PROPEL 2 and PROPEL 3 have the option to join the PROPEL open-label extension study, where they may continue to receive treatment until their growth is complete.5-9

Infographic presenting Propel clinical studies. The left side states that the purpose of the study is to understand achondroplasia in children and adolescents. The middle section lists the outcomes being studied, such as growth (height and body proportions), medical symptoms and complications (middle ear infections, sleep apnea, spinal stenosis, and spinal surgery), and well-being (health-related quality of life, pain, and functional abilities). The right side highlights the purpose of studies: to find a safe and effective dose of treatment and understand how treatment may impact children and adolescents with achondroplasia during development. Infographic presenting Propel clinical studies. The left side states that the purpose of the study is to understand achondroplasia in children and adolescents. The middle section lists the outcomes being studied, such as growth (height and body proportions), medical symptoms and complications (middle ear infections, sleep apnea, spinal stenosis, and spinal surgery), and well-being (health-related quality of life, pain, and functional abilities). The right side highlights the purpose of studies: to find a safe and effective dose of treatment and understand how treatment may impact children and adolescents with achondroplasia during development. Infographic presenting Propel clinical studies. The left side states that the purpose of the study is to understand achondroplasia in children and adolescents. The middle section lists the outcomes being studied, such as growth (height and body proportions), medical symptoms and complications (middle ear infections, sleep apnea, spinal stenosis, and spinal surgery), and well-being (health-related quality of life, pain, and functional abilities). The right side highlights the purpose of studies: to find a safe and effective dose of treatment and understand how treatment may impact children and adolescents with achondroplasia during development.

Overview of the PROPEL studies

Participants who complete at least 6 months in the PROPEL observational study may enroll in PROPEL 3, the Phase 3 investigational study of oral infigratinib sponsored by QED Therapeutics, a BridgeBio company.9

Detailed flowchart and summary of the PROPEL clinical study series for achondroplasia. The series starts with PROPEL, NCT0435811, which is currently enrolling participants aged 2.5 to <17 years. PROPEL has a study duration of 6 months to 2 years and has a study goal to learn about overall health, growth, and potential medical complications in children and adolescents with achondroplasia. This leads into PROPEL2, NCT04265651, with enrollment completed and a study duration of 18 months. PROPEL 2 is focused on children aged 3 to 11 years, aiming to find the most appropriate dose of treatment, safety, and how treatment may affect growth, medical complications, and well-being. Parallel to this is PROPEL 3, NCT06164951, still enrolling, for children and adolescents aged 3 to <18 years, with goals to understand treatment effects on growth, medical complications, and well-being, with a study duration of 1 year. Both PROPEL 2 and PROPEL 3 funnel into PROPEL open-label extension, NCT0514510, enrolling children and adolescents aged 3 to 17 years who completed PROP2 or PROP3, with the goal to explore long-term effects of treatment on overall health, growth, and possible medical complications. The study duration for PROPEL 3 is until growth is complete. Detailed flowchart and summary of the PROPEL clinical study series for achondroplasia. The series starts with PROPEL, NCT0435811, which is currently enrolling participants aged 2.5 to <17 years. PROPEL has a study duration of 6 months to 2 years and has a study goal to learn about overall health, growth, and potential medical complications in children and adolescents with achondroplasia. This leads into PROPEL2, NCT04265651, with enrollment completed and a study duration of 18 months. PROPEL 2 is focused on children aged 3 to 11 years, aiming to find the most appropriate dose of treatment, safety, and how treatment may affect growth, medical complications, and well-being. Parallel to this is PROPEL 3, NCT06164951, still enrolling, for children and adolescents aged 3 to <18 years, with goals to understand treatment effects on growth, medical complications, and well-being, with a study duration of 1 year. Both PROPEL 2 and PROPEL 3 funnel into PROPEL open-label extension, NCT0514510, enrolling children and adolescents aged 3 to 17 years who completed PROP2 or PROP3, with the goal to explore long-term effects of treatment on overall health, growth, and possible medical complications. The study duration for PROPEL 3 is until growth is complete. Detailed flowchart and summary of the PROPEL clinical study series for achondroplasia. The series starts with PROPEL, NCT0435811, which is currently enrolling participants aged 2.5 to <17 years. PROPEL has a study duration of 6 months to 2 years and has a study goal to learn about overall health, growth, and potential medical complications in children and adolescents with achondroplasia. This leads into PROPEL2, NCT04265651, with enrollment completed and a study duration of 18 months. PROPEL 2 is focused on children aged 3 to 11 years, aiming to find the most appropriate dose of treatment, safety, and how treatment may affect growth, medical complications, and well-being. Parallel to this is PROPEL 3, NCT06164951, still enrolling, for children and adolescents aged 3 to <18 years, with goals to understand treatment effects on growth, medical complications, and well-being, with a study duration of 1 year. Both PROPEL 2 and PROPEL 3 funnel into PROPEL open-label extension, NCT0514510, enrolling children and adolescents aged 3 to 17 years who completed PROP2 or PROP3, with the goal to explore long-term effects of treatment on overall health, growth, and possible medical complications. The study duration for PROPEL 3 is until growth is complete.

PROPEL 3 study design

PROPEL 3 is the next step in our process of developing a new treatment option for FGFR3-driven skeletal dysplasias. PROPEL 3 is the first double-blinded study to compare the efficacy and safety of the oral investigational agent infigratinib with placebo in children and adolescents who have achondroplasia.9

Participants who complete at least 6 months in the PROPEL study may enroll in PROPEL 3. The primary goal of PROPEL 3 is to understand the effect of oral infigratinib on bone growth, as measured by annual height velocity (or AHV). This study will also look at the impact of treatment on well-being, pain, and functional abilities.9 Future studies will look at infigratinib in other FGFR3-driven conditions, such as hypochondroplasia.

Flowchart infographic detailing the stages of the PROPEL clinical study. The first stage is 'Observation' with a 26-week period where no treatment is given. This leads to the 'Treatment' phase, labeled 'PROPEL 3,' where participants are randomized to receive either Infigratinib at a dose of 0.25 mg/kg/day or a placebo, with treatment lasting up to 12 months. The final stage is 'Extended treatment' under the 'PROPEL Open Label Extension,' where eligible participants have the option to continue receiving Infigratinib until their final or near final height is reached. Flowchart infographic detailing the stages of the PROPEL clinical study. The first stage is 'Observation' with a 26-week period where no treatment is given. This leads to the 'Treatment' phase, labeled 'PROPEL 3,' where participants are randomized to receive either Infigratinib at a dose of 0.25 mg/kg/day or a placebo, with treatment lasting up to 12 months. The final stage is 'Extended treatment' under the 'PROPEL Open Label Extension,' where eligible participants have the option to continue receiving Infigratinib until their final or near final height is reached. Flowchart infographic detailing the stages of the PROPEL clinical study. The first stage is 'Observation' with a 26-week period where no treatment is given. This leads to the 'Treatment' phase, labeled 'PROPEL 3,' where participants are randomized to receive either Infigratinib at a dose of 0.25 mg/kg/day or a placebo, with treatment lasting up to 12 months. The final stage is 'Extended treatment' under the 'PROPEL Open Label Extension,' where eligible participants have the option to continue receiving Infigratinib until their final or near final height is reached.

Learn more about enrolling into PROPEL, the run-in study for PROPEL 3, the Phase 3 study

Learn more about the study design for PROPEL 3, the Phase 3 study

Participating Site Locations

Participating site locations

United States

  • California Benioff Children’s Hospital Oakland
  • Colorado Children’s Hospital Colorado
  • Maryland Johns Hopkins School of Medicine
  • Missouri University of Missouri
  • Ohio Cincinnati Children’s Hospital Medical Center
  • Tennessee Vanderbilt University Medical Center
  • Wisconsin UW Madison - Waisman Center Bone Dysplasia Clinic

For more details, visit clinicaltrials.gov

Global

  • Argentina Hospital de Pediatría SAMIC Prof. Dr. Juan P. Garrahan
  • Australia Royal Children’s Hospital Melbourne
  • Canada Stollery Children’s Hospital University of Montreal University of Ottawa
  • France Hôpital Femme Mère Enfant Centre Hospitalier Universitaire de Toulouse Hôpital Necker-Enfants Malades
  • Germany Otto-von-Guericke-University Magdeburg Medical Faculty
  • Italy Rare Disease Unit Fondazione Policlinico A Gemelli IRCCS
  • Norway Oslo Universitetssykehus Haukeland Universitetssjukehus
  • Singapore KK Women’s and Children’s Hospital
  • Spain Vithas Hospital San José Hospital Universitario La Paz Hospital Universitario Virgen de la Victoria
  • United Kingdom Manchester University NHS Foundation Trust NHS Greater Glasgow and Clyde Birmingham Women’s and Children’s NHS Foundation Trust Guy’s and Saint Thomas’ NHS Foundation Trust Sheffield Children’s NHS Foundation Trust University Hospitals Bristol and Weston NHS Foundation Trust

For more details, visit clinicaltrials.gov

The process of developing a new treatment option

An infographic outlining the six phases of drug development. Phase 1 is 'Discovery,' where identification and validation of the treatment approach occur. Phase 2 is 'Preclinical Studies,' which involves confirming the effects of the treatment in laboratory studies. Phase 3 encompasses 'Phase 1/2 Studies,' assessing treatment effects, side effects, and dosages in a small group of participants. Phase 4, 'Phase 3 Studies,' confirms results in a larger group of participants. Phase 5 is 'Health Authority Review,' where the treatment is reviewed and approved by health authorities. Finally, Phase 6 is 'Approval Granted,' where treatment availability is monitored, and education on the condition and treatment is provided. Below these phases, the infographic emphasizes community involvement throughout the development process with strategies like listening to community needs, inviting dialogue on the condition and treatment, ensuring community participation, sharing results transparently, and gathering advice on treatment use in the community.

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

News and updates

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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.
  9. A study to evaluate the efficacy and safety of infigratinib in children and adolescents with achondroplasia (PROPEL 3). ClinicalTrials.gov identifier: NCT06164951. Updated December 21, 2023. Accessed January 12, 2024. https://clinicaltrials.gov/study/NCT06164951?cond=achondroplasia&term=PROPEL3&rank=2.