QED Therapeutics starts observational study
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."


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