This web site uses cookies. Do you accept the use of external cookies? Yes No Know more x
Survey about achondroplasia
We have designed a questionnaire with the purpose to better know the population connected to achondroplasia.
The questionnaire is optional and anonymous. The data obtained will be used for statistical purposes and to better understand the natural history of achondroplasia. Please consider answering.
If you would like to take the survey later, you can click on the link at the top "Register at BA"
The PROPEL trial is the Prospective Clinical Assessment Study in Children with Achondroplasia, officially started on the 29th July 2019 and is actively recruiting at the first study center, the Murdoch Children's Research Institute, Parkville, in Australia. You can read about QED Therapeutics and infigratinib here. We posted the first article posted about Infigratinib or BGJ398 in May 2016, that you can read an explanation here, after the publication of the study "Tyrosine kinase inhibitor NVP-BGJ398 functionally improves FGFR3-related dwarfism in mouse model."

QED logo
Then, Beyond Achondroplasia first published about QED Tx, a new company dedicating efforts to achondroplasia in August 2018 and one year later, the company is now starting this observational study, showing that QED is in a fast process towards starting an interventional study with infigratinib in 2020. Information about the PROPEL trial can be found at
What is an Observational study?

Is conducted to identify and evaluate causes or risk factors of diseases or health-related events in which the investigator do not intervene and rather simply “observe” the natural progression of the disease or condition. 1

What is a Prospective study?

This type of study focuses on the observation of outcomes, such as the development or progression of one disease during the study period and relates this to other factors. The study usually involves taking a cohort (groups) of subjects and watching them over a long period. The outcome of interest should be common.2

prospective retrospective study
Figure 1. Temporal Design of Observational Studies. Credits: Song J & Cheung K, 2010 

The PROPEL trial has the following information:

Study Type  : Observational
Estimated Enrollment  : 200 participants (at a global level, to be completed in many study centers)
Observational Model: Case-Only
Time Perspective: Prospective
Official Title: Prospective Clinical Assessment Study in Children With Achondroplasia: The PROPEL Trial
Estimated Study Start Date  : July 2019
Estimated Primary Completion Date  : June 2026
Estimated Study Completion Date  : June 2026

Soon other study centers will follow the Murdoch Children's Research Institute. For any question regarding recruitment, contact QED Medical Director at This email address is being protected from spambots. You need JavaScript enabled to view it.

1. Song J, Cheung K, 2010 - Observational studies: cohort and case-control studies
2. Stats direct 2019

To view this site you need Internet Explorer 8 (or a higher version)
or another internet browser.

Thank You.