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

Our clinical program includes multiple achondroplasia clinical trials that are designed to better understand FGFR3-driven skeletal dysplasias and to evaluate an oral investigational therapy option for these conditions.

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

The PROPEL clinical program:

A holistic approach for
achondroplasia research

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 the investigational drug until their growth is complete.5-9

Three side-by-side cards with 'PROPEL' logos. The left card titled 'Purpose of study' outlines that PROPEL's focus is to understand achondroplasia in children and adolescents. The middle card lists 'Outcomes being studied,' including growth, medical symptoms like middle ear infections, sleep apnea, and spinal surgery, and well-being factors like quality of life, pain, and functional abilities. The right card, with 'Purpose of studies,' explains PROPEL2 and PROPEL3, which aim to find a safe and effective dose and assess the treatment’s impact on children with achondroplasia. A vertical layout of the PROPEL studies with 'PROPEL' logo at the top, followed by purpose descriptions. The first section highlights PROPEL's aim to understand achondroplasia in children. The middle section describes outcomes being studied, such as growth, medical complications, and well-being. The bottom section covers PROPEL2 and PROPEL3 with a focus on finding a safe dose and evaluating treatment impacts on children and adolescents with achondroplasia.

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.9

A horizontal chart depicting the flow through PROPEL, PROPEL2, PROPEL3, and an open-label extension. Each phase is marked by boxes with 'Enrolling' or 'Enrollment completed' tags, along with study descriptions for each phase. The flowchart emphasizes study progression from initial enrollment to treatment phases and optional extended treatment. A vertical flowchart detailing the stages of the PROPEL studies. The top section introduces PROPEL, enrolling children with achondroplasia, focusing on growth, medical symptoms, and quality of life. The chart branches to PROPEL2 and PROPEL3, each with descriptions of study goals, including evaluating safe dosages and monitoring treatment impact. At the bottom is an open-label extension study for participants to continue treatment under observation.

Infigratinib is not currently approved for the treatment of achondroplasia by the U.S. Food and Drug Administration (FDA) or any other health authority.

PROPEL 3 study design

PROPEL 3 is the next step in our process of developing a potential 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 the investigational treatment on well-being, pain, and functional abilities.9 Future studies will look at infigratinib in other FGFR3-driven conditions, such as hypochondroplasia.

A flowchart outlining the phases in the PROPEL studies, moving from observation to treatment and extended treatment. Arrows direct the flow, indicating an observation period followed by randomized treatment. The treatment phase offers infogratinib or placebo for up to 12 months, with an extended treatment option for eligible participants. A flowchart outlining the phases in the PROPEL studies, moving from observation to treatment and extended treatment. Arrows direct the flow, indicating an observation period followed by randomized treatment. The treatment phase offers infogratinib or placebo for up to 12 months, with an extended treatment option for eligible participants.

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

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 Children’s Hospital - London Health Sciences Center
  • 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 Fondazione IRCCS Ca’ Granda - Ospedale Maggiore Policlino
  • 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

References