Therachon starts a Natural History Study in children with achondroplasia

"Dreambird" is the name of the natural history study that Therachon announced on the 21st June 2018.

The “Dreambird” study, or TA 46-002, will prospectively assess the burden of complications of achondroplasia in approximately 200 children across multiple sites in Europe, Canada, and the United States. The study will pave the way for future interventional studies with TA 46 and also support the development of key biomarkers of bone tissue growth.1

Because it will be a prospective study, it will watch for outcomes, such as the development of the disease during the study period and will relate this to other factors such as suspected risk or protection factor(s).3

The first center announced that will hold this study is Antwerp University Hospitaledead by Professor Geert Mortier, clinical geneticist. The list of the other clinical centers for this study is still under evaluation by the company.

What is the reason to conduct a Natural History Study?

To know the disease once it is an essential element in the scientific foundation of any clinical development program, especially in rare diseases, that in general, are poorly understood. It is also important and essential the role of Natural History studies in rare disease drug development (more for the Investigational New Drug phase) to facilitate efficient clinical development.2 

Christian Meyer,  Therachon´s Chief Medical Officer said that “Knowledge of the natural history of achondroplasia and identification of molecular biomarkers of bone growth are essential to build the scientific foundation for the forthcoming clinical development program of TA-46,” and that “We are grateful to the children and their families who will participate in this study.”1

Key information

Enrollment in the natural history study is a pre-requisite for participation in the forthcoming interventional studies with TA 46, or phase 2 studies.1

This means that for children to be able to participate in the TA 46 clinical trial, is mandatory to participate in this observational study before.

About the TA 46

Therachon´s molecule for the treatment of achondroplasia, the TA 46 is being developed as a weekly subcutaneous drug for children and adolescents living with the condition.

 

 

Sources:

1. Business Wire

2. Importance of Natural History Studies in Rare Diseases - FDA

3. Boston University - School of Public Health - Prospective studies