A new step forward for TherAchon: the Dreambird study on Achondroplasia

TherAchon is a Biotechnology company that was established in 2014 and focuses on rare diseases. It is developing a drug for achondroplasia in particular: TA-46, which is planned to go onto phase I clinical trials in 2019 [1].

 

In order to prepare for the clinical trial with TA-46, TherAchon needs to capture many parameters on achondroplasia, for example, establish how much children with achondroplasia grow each year, or in other words understanding the baseline growth.

 

Image adapted from Brain for the cure

 

TherAchon has announced at the end of June that they have started a Natural History study on achondroplasia, called Dreambird.

The Dreambird study will follow-up 200 participants from several sites in Europe, USA and Canada which will evaluate and measure children growth, disease progression, the potential risk of complications related to ACH and protection factors, among other biological parameters for a specific period [2]. Which parameters will be measured, exactly, have not been disclosed yet, but the full details of this study will be available in the Clinical Trials website very soon.

Is important to emphasize that, because the Dreambird study is an observational study, the experimental drug TA-46 will not be administered at any time in this study.

Nevertheless, since one of the objectives of this study, besides providing a better understanding of pediatric achondroplasia, is to establish the baseline growth rates and to identify biomarkers for achondroplasia, it will be mandatory for children to participate in the Dreambird study before being enrolled in the future interventional studies, namely: the phase 2 and 3 of the TA-46 clinical trial.

The age of children to be enrolled in the Dreambird study is from 0 to 10 years old.